William W Hauswirth

William W Hauswirth, PhD

Eminent Scholar

Department: MD-OPHTHALMOLOGY
Business Phone: (352) 359-4301
Business Email: hauswrth@eye.ufl.edu

Teaching Profile

Courses Taught
2018
GMS7980 Research for Doctoral Dissertation
2013,2015
GMS6709 Current Topics in Vision

Publications

2022
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene.
Gene therapy. 29(6):368-378 [DOI] 10.1038/s41434-022-00333-6. [PMID] 35383288.
2022
Neuroprotective Gene Therapy by Overexpression of the Transcription Factor MAX in Rat Models of Glaucomatous Neurodegeneration.
Investigative ophthalmology & visual science. 63(2) [DOI] 10.1167/iovs.63.2.5. [PMID] 35103748.
2022
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma.
Gene therapy. 29(3-4):147-156 [DOI] 10.1038/s41434-021-00281-7. [PMID] 34363035.
2021
CNGB1-related rod-cone dystrophy: A mutation review and update.
Human mutation. 42(6):641-666 [DOI] 10.1002/humu.24205. [PMID] 33847019.
2021
Disease mechanisms of X-linked cone dystrophy caused by missense mutations in the red and green cone opsins.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology. 35(10) [DOI] 10.1096/fj.202101066R. [PMID] 34547123.
2021
Examining Whether AOSLO-Based Foveal Cone Metrics in Achromatopsia and Albinism Are Representative of Foveal Cone Structure.
Translational vision science & technology. 10(6) [DOI] 10.1167/tvst.10.6.22. [PMID] 34111268.
2021
Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis.
Molecular therapy : the journal of the American Society of Gene Therapy. 29(12) [DOI] 10.1016/j.ymthe.2021.10.021. [PMID] 34715016.
2021
Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis.
Molecular therapy : the journal of the American Society of Gene Therapy. 29(8):2456-2468 [DOI] 10.1016/j.ymthe.2021.03.021. [PMID] 33781914.
2021
Optical Coherence Tomography Artifacts Are Associated With Adaptive Optics Scanning Light Ophthalmoscopy Success in Achromatopsia.
Translational vision science & technology. 10(1) [DOI] 10.1167/tvst.10.1.11. [PMID] 33510950.
2020
Clarin-1 expression in adult mouse and human retina highlights a role of Müller glia in Usher syndrome.
The Journal of pathology. 250(2):195-204 [DOI] 10.1002/path.5360. [PMID] 31625146.
2020
Effects of a combinatorial treatment with gene and cell therapy on retinal ganglion cell survival and axonal outgrowth after optic nerve injury.
Gene therapy. 27(1-2):27-39 [DOI] 10.1038/s41434-019-0089-0. [PMID] 31243393.
2020
Evaluation of Photoreceptor Transduction Efficacy of Capsid-Modified Adeno-Associated Viral Vectors Following Intravitreal and Subretinal Delivery in Sheep.
Human gene therapy. 31(13-14):719-729 [DOI] 10.1089/hum.2020.023. [PMID] 32486858.
2020
Interocular Symmetry of Foveal Cone Topography in Congenital Achromatopsia.
Current eye research. 45(10):1257-1264 [DOI] 10.1080/02713683.2020.1737138. [PMID] 32108519.
2020
Interplay between cell-adhesion molecules governs synaptic wiring of cone photoreceptors.
Proceedings of the National Academy of Sciences of the United States of America. 117(38):23914-23924 [DOI] 10.1073/pnas.2009940117. [PMID] 32879010.
2020
Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy.
Molecular therapy : the journal of the American Society of Gene Therapy. 28(1):266-278 [DOI] 10.1016/j.ymthe.2019.08.013. [PMID] 31604676.
2020
Prdm1 overexpression causes a photoreceptor fate-shift in nascent, but not mature, bipolar cells.
Developmental biology. 464(2):111-123 [DOI] 10.1016/j.ydbio.2020.06.003. [PMID] 32562755.
2020
XIAP Protects Retinal Ganglion Cells in the Mutant ND4 Mouse Model of Leber Hereditary Optic Neuropathy.
Investigative ophthalmology & visual science. 61(8) [DOI] 10.1167/iovs.61.8.49. [PMID] 32735323.
2019
Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model.
Proceedings of the National Academy of Sciences of the United States of America. 116(10):4496-4501 [DOI] 10.1073/pnas.1817537116. [PMID] 30782832.
2019
Dual ABCA4-AAV Vector Treatment Reduces Pathogenic Retinal A2E Accumulation in a Mouse Model of Autosomal Recessive Stargardt Disease.
Human gene therapy. 30(11):1361-1370 [DOI] 10.1089/hum.2019.132. [PMID] 31418294.
2019
Optimal Inhibition of Choroidal Neovascularization by scAAV2 with VMD2 Promoter-driven Active Rap1a in the RPE.
Scientific reports. 9(1) [DOI] 10.1038/s41598-019-52163-z. [PMID] 31673119.
2019
Rescue of M-cone Function in Aged Opn1mw-/- Mice, a Model for Late-Stage Blue Cone Monochromacy.
Investigative ophthalmology & visual science. 60(10):3644-3651 [DOI] 10.1167/iovs.19-27079. [PMID] 31469404.
2019
Retina transduction by rAAV2 after intravitreal injection: comparison between mouse and rat.
Gene therapy. 26(12):479-490 [DOI] 10.1038/s41434-019-0100-9. [PMID] 31562387.
2018
BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure.
Proceedings of the National Academy of Sciences of the United States of America. 115(12):E2839-E2848 [DOI] 10.1073/pnas.1720662115. [PMID] 29507198.
2018
Co-Expression of Wild-Type and Mutant S163R C1QTNF5 in Retinal Pigment Epithelium.
Advances in experimental medicine and biology. 1074:61-66 [DOI] 10.1007/978-3-319-75402-4_8. [PMID] 29721928.
2018
Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma.
Molecular therapy : the journal of the American Society of Gene Therapy. 26(10):2379-2396 [DOI] 10.1016/j.ymthe.2018.08.017. [PMID] 30217731.
2018
Cone Phosphodiesterase-6γ’ Subunit Augments Cone PDE6 Holoenzyme Assembly and Stability in a Mouse Model Lacking Both Rod and Cone PDE6 Catalytic Subunits.
Frontiers in molecular neuroscience. 11 [DOI] 10.3389/fnmol.2018.00233. [PMID] 30038560.
2018
ELOVL4-Mediated Production of Very Long-Chain Ceramides Stabilizes Tight Junctions and Prevents Diabetes-Induced Retinal Vascular Permeability.
Diabetes. 67(4):769-781 [DOI] 10.2337/db17-1034. [PMID] 29362226.
2018
Human L- and M-opsins restore M-cone function in a mouse model for human blue cone monochromacy.
Molecular vision. 24:17-28 [PMID] 29386880.
2018
Longterm Reversal of Severe Visual Loss by Mitochondrial Gene Transfer in a Mouse Model of Leber Hereditary Optic Neuropathy.
Scientific reports. 8(1) [DOI] 10.1038/s41598-018-23836-y. [PMID] 29615737.
2018
Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector.
Proceedings of the National Academy of Sciences of the United States of America. 115(36):E8547-E8556 [DOI] 10.1073/pnas.1805055115. [PMID] 30127005.
2018
Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice.
Advances in experimental medicine and biology. 1074:125-131 [DOI] 10.1007/978-3-319-75402-4_16. [PMID] 29721936.
2018
Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach.
The Journal of clinical investigation. 128(1):190-206 [DOI] 10.1172/JCI95161. [PMID] 29202463.
2018
Publisher Correction: Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy.
Scientific reports. 8(1) [DOI] 10.1038/s41598-018-23131-w. [PMID] 29540812.
2018
Reply.
Ophthalmology. 125(2):e15-e16 [DOI] 10.1016/j.ophtha.2017.08.041. [PMID] 29389414.
2018
Rescue of cone function in cone-only Nphp5 knockout mouse model with Leber congenital amaurosis phenotype.
Molecular vision. 24:834-846 [PMID] 30713422.
2018
Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 Achromatopsia.
Human gene therapy. 29(12):1376-1386 [DOI] 10.1089/hum.2018.076. [PMID] 29926749.
2018
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Human gene therapy. Clinical development. 29(4):188-197 [DOI] 10.1089/humc.2018.168. [PMID] 30280954.
2017
Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors.
Human gene therapy methods. 28(1):49-59 [DOI] 10.1089/hgtb.2016.089. [PMID] 28125909.
2017
Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep.
Investigative ophthalmology & visual science. 58(3):1577-1584 [DOI] 10.1167/iovs.16-20986. [PMID] 28282490.
2017
Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results.
Ophthalmology. 124(11):1621-1634 [DOI] 10.1016/j.ophtha.2017.05.016. [PMID] 28647203.
2017
Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa.
Frontiers in neuroscience. 11 [DOI] 10.3389/fnins.2017.00342. [PMID] 28676737.
2017
Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy.
Scientific reports. 7(1) [DOI] 10.1038/s41598-017-06982-7. [PMID] 28751656.
2017
Long-term photoreceptor rescue in two rodent models of retinitis pigmentosa by adeno-associated virus delivery of Stanniocalcin-1.
Experimental eye research. 165:175-181 [DOI] 10.1016/j.exer.2017.09.011. [PMID] 28974356.
2017
miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity.
Gene therapy. 24(8):462-469 [DOI] 10.1038/gt.2017.50. [PMID] 28617420.
2017
Modeling and Preventing Progressive Hearing Loss in Usher Syndrome III.
Scientific reports. 7(1) [DOI] 10.1038/s41598-017-13620-9. [PMID] 29044151.
2017
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Molecular therapy : the journal of the American Society of Gene Therapy. 25(8):1866-1880 [DOI] 10.1016/j.ymthe.2017.05.004. [PMID] 28566226.
2017
Overexpression of the X-Linked Inhibitor of Apoptosis Protects Against Retinal Degeneration in a Feline Model of Retinal Detachment.
Human gene therapy. 28(6):482-492 [DOI] 10.1089/hum.2016.161. [PMID] 28335619.
2017
rAAV8-733-Mediated Gene Transfer of CHIP/Stub-1 Prevents Hippocampal Neuronal Death in Experimental Brain Ischemia.
Molecular therapy : the journal of the American Society of Gene Therapy. 25(2):392-400 [DOI] 10.1016/j.ymthe.2016.11.017. [PMID] 28153090.
2017
Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS.
Molecular therapy. Nucleic acids. 8:184-197 [DOI] 10.1016/j.omtn.2017.06.011. [PMID] 28918020.
2017
REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA.
Retina (Philadelphia, Pa.). 37(10):1956-1966 [DOI] 10.1097/IAE.0000000000001434. [PMID] 28145975.
2017
Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs.
Human gene therapy. Clinical development. 28(4):197-207 [DOI] 10.1089/humc.2017.125. [PMID] 29020838.
2016
AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy.
PloS one. 11(2) [DOI] 10.1371/journal.pone.0148874. [PMID] 26881841.
2016
Characterization of intravitreally delivered capsid mutant AAV2-Cre vector to induce tissue-specific mutations in murine retinal ganglion cells.
Experimental eye research. 151:61-7 [DOI] 10.1016/j.exer.2016.07.019. [PMID] 27481653.
2016
Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases.
Human gene therapy. 27(1):72-82 [DOI] 10.1089/hum.2015.130. [PMID] 26603570.
2016
Gene Therapy for MERTK-Associated Retinal Degenerations.
Advances in experimental medicine and biology. 854:487-93 [DOI] 10.1007/978-3-319-17121-0_65. [PMID] 26427450.
2016
Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex.
Gene therapy. 23(3):272-82 [DOI] 10.1038/gt.2015.112. [PMID] 26704722.
2016
Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina.
Human gene therapy. 27(8):580-97 [DOI] 10.1089/hum.2016.085. [PMID] 27439313.
2016
Maintaining ocular safety with light exposure, focusing on devices for optogenetic stimulation.
Vision research. 121:57-71 [DOI] 10.1016/j.visres.2016.01.006. [PMID] 26882975.
2016
Occludin S490 Phosphorylation Regulates Vascular Endothelial Growth Factor-Induced Retinal Neovascularization.
The American journal of pathology. 186(9):2486-99 [DOI] 10.1016/j.ajpath.2016.04.018. [PMID] 27423695.
2016
PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina.
Molecular therapy. Methods & clinical development. 3 [DOI] 10.1038/mtm.2016.51. [PMID] 27556059.
2016
Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.
Gene therapy. 23(4) [DOI] 10.1038/gt.2016.10. [PMID] 27052928.
2016
Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.
Gene therapy. 23(2):223-30 [DOI] 10.1038/gt.2015.96. [PMID] 26467396.
2016
Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs.
Gene therapy. 23(6):548-56 [DOI] 10.1038/gt.2016.31. [PMID] 27052802.
2016
Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia.
Investigative ophthalmology & visual science. 57(10):3984-95 [DOI] 10.1167/iovs.16-19313. [PMID] 27479814.
2016
Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.
Ophthalmology. 123(7):1606-20 [DOI] 10.1016/j.ophtha.2016.03.003. [PMID] 27102010.
2016
Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Human gene therapy. Clinical development. 27(1):27-36 [DOI] 10.1089/humc.2015.163. [PMID] 27003752.
2016
Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia.
Human gene therapy. Clinical development. 27(1):37-48 [DOI] 10.1089/humc.2015.164. [PMID] 27003753.
2016
Small GTPases Rab8a and Rab11a Are Dispensable for Rhodopsin Transport in Mouse Photoreceptors.
PloS one. 11(8) [DOI] 10.1371/journal.pone.0161236. [PMID] 27529348.
2016
Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology. 30(12):4313-4325 [PMID] 27623928.
2016
Targeting the Nrf2 Signaling Pathway in the Retina With a Gene-Delivered Secretable and Cell-Penetrating Peptide.
Investigative ophthalmology & visual science. 57(2):372-86 [DOI] 10.1167/iovs.15-17703. [PMID] 26842755.
2016
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.
Human genetics. 135(3):327-43 [DOI] 10.1007/s00439-016-1637-y. [PMID] 26825853.
2015
Aav Delivery of Alpha Synuclein To the Enteric Nervous System Impairs Colonic Motility
Cell Transplantation. 24(4, SI)
2015
AAV-mediated and pharmacological induction of Hsp70 expression stimulates survival of retinal ganglion cells following axonal injury.
Gene therapy. 22(2):138-45 [DOI] 10.1038/gt.2014.105. [PMID] 25427613.
2015
AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa.
Gene therapy. 22(8):619-27 [DOI] 10.1038/gt.2015.42. [PMID] 25965394.
2015
Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?
Human gene therapy. 26(12):779-81 [DOI] 10.1089/hum.2015.29014.kib. [PMID] 26690810.
2015
Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium.
Investigative ophthalmology & visual science. 56(2):918-31 [DOI] 10.1167/iovs.14-16049. [PMID] 25667399.
2015
Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat.
PloS one. 10(6) [DOI] 10.1371/journal.pone.0128759. [PMID] 26052939.
2015
Complex I subunit gene therapy with NDUFA6 ameliorates neurodegeneration in EAE.
Investigative ophthalmology & visual science. 56(2):1129-40 [DOI] 10.1167/iovs.14-15950. [PMID] 25613946.
2015
Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice.
Proceedings of the National Academy of Sciences of the United States of America. 112(42):E5689-98 [DOI] 10.1073/pnas.1506129112. [PMID] 26438859.
2015
Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia.
Molecular therapy : the journal of the American Society of Gene Therapy. 23(9):1423-33 [DOI] 10.1038/mt.2015.114. [PMID] 26087757.
2015
Gene delivery of a viral anti-inflammatory protein to combat ocular inflammation.
Human gene therapy. 26(1):59-68 [DOI] 10.1089/hum.2014.089. [PMID] 25420215.
2015
Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1.
Human gene therapy. 26(9):575-92 [DOI] 10.1089/hum.2015.053. [PMID] 26247368.
2015
Gene therapy with the caspase activation and recruitment domain reduces the ocular inflammatory response.
Molecular therapy : the journal of the American Society of Gene Therapy. 23(5):875-884 [DOI] 10.1038/mt.2015.30. [PMID] 25698151.
2015
Improvement and decline in vision with gene therapy in childhood blindness.
The New England journal of medicine. 372(20):1920-6 [DOI] 10.1056/NEJMoa1412965. [PMID] 25936984.
2015
Improvement in vision: a new goal for treatment of hereditary retinal degenerations.
Expert opinion on orphan drugs. 3(5):563-575 [PMID] 26246977.
2015
Kinesin family 17 (osmotic avoidance abnormal-3) is dispensable for photoreceptor morphology and function.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology. 29(12):4866-80 [DOI] 10.1096/fj.15-275677. [PMID] 26229057.
2015
Pathological Effects of Mutant C1QTNF5 (S163R) Expression in Murine Retinal Pigment Epithelium.
Investigative ophthalmology & visual science. 56(11):6971-80 [DOI] 10.1167/iovs.15-17166. [PMID] 26513502.
2015
Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa.
Human gene therapy. 26(9):593-602 [DOI] 10.1089/hum.2015.035. [PMID] 26076799.
2015
Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.
Proceedings of the National Academy of Sciences of the United States of America. 112(43):E5844-53 [DOI] 10.1073/pnas.1509914112. [PMID] 26460017.
2015
Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection.
Human gene therapy. 26(11):767-76 [DOI] 10.1089/hum.2015.097. [PMID] 26359319.
2015
Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants.
Molecular therapy : the journal of the American Society of Gene Therapy. 23(3):488-500 [DOI] 10.1038/mt.2015.7. [PMID] 25592336.
2015
Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model.
Human molecular genetics. 24(3):670-84 [DOI] 10.1093/hmg/ddu487. [PMID] 25274777.
2015
Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia.
Human molecular genetics. 24(13):3699-707 [DOI] 10.1093/hmg/ddv114. [PMID] 25855802.
2014
AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.
Investigative ophthalmology & visual science. 55(3):1724-34 [DOI] 10.1167/iovs.13-13654. [PMID] 24557352.
2014
CHIP, a carboxy terminus HSP-70 interacting protein, prevents cell death induced by endoplasmic reticulum stress in the central nervous system.
Frontiers in cellular neuroscience. 8 [DOI] 10.3389/fncel.2014.00438. [PMID] 25620910.
2014
Cone specific promoter for use in gene therapy of retinal degenerative diseases.
Advances in experimental medicine and biology. 801:695-701 [DOI] 10.1007/978-1-4614-3209-8_87. [PMID] 24664760.
2014
DICER1/Alu RNA dysmetabolism induces Caspase-8-mediated cell death in age-related macular degeneration.
Proceedings of the National Academy of Sciences of the United States of America. 111(45):16082-7 [DOI] 10.1073/pnas.1403814111. [PMID] 25349431.
2014
Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.
Human gene therapy methods. 25(2):166-77 [DOI] 10.1089/hgtb.2013.212. [PMID] 24568220.
2014
Flicker cone function in normal and day blind sheep: a large animal model for human achromatopsia caused by CNGA3 mutation.
Documenta ophthalmologica. Advances in ophthalmology. 129(3):141-50 [DOI] 10.1007/s10633-014-9458-6. [PMID] 25204753.
2014
Gene augmentation for X-linked retinitis pigmentosa caused by mutations in RPGR.
Cold Spring Harbor perspectives in medicine. 5(2) [DOI] 10.1101/cshperspect.a017392. [PMID] 25301933.
2014
Gene therapy in the rd6 mouse model of retinal degeneration.
Advances in experimental medicine and biology. 801:711-8 [DOI] 10.1007/978-1-4614-3209-8_89. [PMID] 24664762.
2014
Gene therapy with mitochondrial heat shock protein 70 suppresses visual loss and optic atrophy in experimental autoimmune encephalomyelitis.
Investigative ophthalmology & visual science. 55(8):5214-26 [DOI] 10.1167/iovs.14-14688. [PMID] 25015358.
2014
Genetically targeted binary labeling of retinal neurons.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 34(23):7845-61 [DOI] 10.1523/JNEUROSCI.2960-13.2014. [PMID] 24899708.
2014
In vivo knockdown of Piccolino disrupts presynaptic ribbon morphology in mouse photoreceptor synapses.
Frontiers in cellular neuroscience. 8 [DOI] 10.3389/fncel.2014.00259. [PMID] 25232303.
2014
Inhibitor of apoptosis-stimulating protein of p53 (iASPP) is required for neuronal survival after axonal injury.
PloS one. 9(4) [DOI] 10.1371/journal.pone.0094175. [PMID] 24714389.
2014
LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile.
Investigative ophthalmology & visual science. 55(12):7739-53 [DOI] 10.1167/iovs.14-15388. [PMID] 25342621.
2014
Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.
PloS one. 9(3) [DOI] 10.1371/journal.pone.0092928. [PMID] 24671090.
2014
Numb regulates the polarized delivery of cyclic nucleotide-gated ion channels in rod photoreceptor cilia.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 34(42):13976-87 [DOI] 10.1523/JNEUROSCI.1938-14.2014. [PMID] 25319694.
2014
Pseudo-fovea formation after gene therapy for RPE65-LCA.
Investigative ophthalmology & visual science. 56(1):526-37 [DOI] 10.1167/iovs.14-15895. [PMID] 25537204.
2014
Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector.
Human gene therapy methods. 25(1):72-82 [DOI] 10.1089/hgtb.2013.011. [PMID] 24191859.
2014
Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.
Human gene therapy. 25(8):671-8 [DOI] 10.1089/hum.2014.2530. [PMID] 25136913.
2014
Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.
JAMA ophthalmology. 132(4):409-20 [DOI] 10.1001/jamaophthalmol.2013.7630. [PMID] 24457989.
2014
STAT3 promotes survival of mutant photoreceptors in inherited photoreceptor degeneration models.
Proceedings of the National Academy of Sciences of the United States of America. 111(52):E5716-23 [DOI] 10.1073/pnas.1411248112. [PMID] 25512545.
2014
Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors.
Molecular therapy. Methods & clinical development. 1 [PMID] 24761428.
2014
Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach.
Gene therapy. 21(1):96-105 [DOI] 10.1038/gt.2013.64. [PMID] 24225638.
2013
A comprehensive review of retinal gene therapy.
Molecular therapy : the journal of the American Society of Gene Therapy. 21(3):509-19 [DOI] 10.1038/mt.2012.280. [PMID] 23358189.
2013
AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.
Human gene therapy. 24(2):189-202 [DOI] 10.1089/hum.2012.193. [PMID] 23210611.
2013
Cone phosphodiesterase-6α’ restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6β-deficient rd10 mouse.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 33(29):11745-53 [DOI] 10.1523/JNEUROSCI.1536-13.2013. [PMID] 23864662.
2013
Development of an anti-angiogenic therapeutic model combining scAAV2-delivered siRNAs and noninvasive photoacoustic imaging of tumor vasculature development.
Cancer letters. 332(1):120-9 [DOI] 10.1016/j.canlet.2012.11.016. [PMID] 23196055.
2013
Dual Adeno-Associated Virus Vectors for Delivery of Large Genes To the Retina
Molecular Therapy. 21:S86-S87
2013
Endothelin-2-mediated protection of mutant photoreceptors in inherited photoreceptor degeneration.
PloS one. 8(2) [DOI] 10.1371/journal.pone.0058023. [PMID] 23469133.
2013
Enhanced Cns Transduction and Transgene Expression By Raav Capsid Tyrosine Mutants
Molecular Therapy. 21
2013
Evaluation of lateral spread of transgene expression following subretinal AAV-mediated gene delivery in dogs.
PloS one. 8(4) [DOI] 10.1371/journal.pone.0060218. [PMID] 23560080.
2013
Functional genomic screening identifies dual leucine zipper kinase as a key mediator of retinal ganglion cell death.
Proceedings of the National Academy of Sciences of the United States of America. 110(10):4045-50 [DOI] 10.1073/pnas.1211284110. [PMID] 23431148.
2013
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.
Proceedings of the National Academy of Sciences of the United States of America. 110(6):E517-25 [DOI] 10.1073/pnas.1218933110. [PMID] 23341635.
2013
Imaging the response of the retina to electrical stimulation with genetically encoded calcium indicators.
Journal of neurophysiology. 109(7):1979-88 [DOI] 10.1152/jn.00852.2012. [PMID] 23343890.
2013
NADH-dehydrogenase type-2 suppresses irreversible visual loss and neurodegeneration in the EAE animal model of MS.
Molecular therapy : the journal of the American Society of Gene Therapy. 21(10):1876-88 [DOI] 10.1038/mt.2013.104. [PMID] 23752309.
2013
New Minipromoters With Restricted-Retinal Expression When Docked in the Mouse Genome Show the Same Expression When Delivered in Aav
Molecular Therapy. 21:S47-S48
2013
Next-generation sequencing of mitochondrial targeted AAV transfer of human ND4 in mice.
Molecular vision. 19:1482-91 [PMID] 23869167.
2013
Phase I Gene Therapy Trial in Israeli Patients With Leber Congenital Amaurosis Caused By a Founder Rpe65 Mutation: Safety and Efficacy Update With Up To Three Years of Follow-Up
Human Gene Therapy. 24:A6-A7
2013
Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1.
eLife. 2 [DOI] 10.7554/eLife.00324. [PMID] 23795287.
2013
Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa.
Human gene therapy. Clinical development. 24(1):23-8 [DOI] 10.1089/humc.2013.037. [PMID] 23692380.
2013
Preclinical Safety Studies for Aav2-Mertk Gene Therapy Vector for Retinitis Pigmentosa
Molecular Therapy. 21
2013
RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12.
Human molecular genetics. 22(19):3894-905 [DOI] 10.1093/hmg/ddt244. [PMID] 23740938.
2013
Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects.
PloS one. 8(10) [DOI] 10.1371/journal.pone.0075666. [PMID] 24143172.
2013
Reply to Townes-Anderson: RPE65 gene therapy does not alter the natural history of retinal degeneration.
Proceedings of the National Academy of Sciences of the United States of America. 110(19) [PMID] 23789127.
2013
Retinal angiogenesis suppression through small molecule activation of p53.
The Journal of clinical investigation. 123(10):4170-81 [DOI] 10.1172/JCI67315. [PMID] 24018558.
2013
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
Gene therapy. 20(8):824-33 [DOI] 10.1038/gt.2013.3. [PMID] 23344065.
2013
RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model.
PloS one. 8(3) [DOI] 10.1371/journal.pone.0057676. [PMID] 23472098.
2013
scAAV-mediated gene transfer of interleukin-1-receptor antagonist to synovium and articular cartilage in large mammalian joints.
Gene therapy. 20(6):670-7 [DOI] 10.1038/gt.2012.81. [PMID] 23151520.
2013
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors.
PloS one. 8(4) [DOI] 10.1371/journal.pone.0062097. [PMID] 23637972.
2013
Transient photoreceptor deconstruction by CNTF enhances rAAV-mediated cone functional rescue in late stage CNGB3-achromatopsia.
Molecular therapy : the journal of the American Society of Gene Therapy. 21(6):1131-41 [DOI] 10.1038/mt.2013.50. [PMID] 23568263.
2012
AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia.
PloS one. 7(4) [DOI] 10.1371/journal.pone.0035250. [PMID] 22509403.
2012
AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2(-/-)/Cx3cr1(-/-) mice.
Neurobiology of aging. 33(2):433.e1-10 [DOI] 10.1016/j.neurobiolaging.2011.01.009. [PMID] 21397984.
2012
ACE2 and Ang-(1-7) confer protection against development of diabetic retinopathy.
Molecular therapy : the journal of the American Society of Gene Therapy. 20(1):28-36 [DOI] 10.1038/mt.2011.155. [PMID] 21792177.
2012
Caspase inhibition with XIAP as an adjunct to AAV vector gene-replacement therapy: improving efficacy and prolonging the treatment window.
PloS one. 7(5) [DOI] 10.1371/journal.pone.0037197. [PMID] 22615940.
2012
CCN2/CTGF regulates neovessel formation via targeting structurally conserved cystine knot motifs in multiple angiogenic regulators.
FASEB journal : official publication of the Federation of American Societies for Experimental Biology. 26(8):3365-79 [DOI] 10.1096/fj.11-200154. [PMID] 22611085.
2012
DICER1 loss and Alu RNA induce age-related macular degeneration via the NLRP3 inflammasome and MyD88.
Cell. 149(4):847-59 [DOI] 10.1016/j.cell.2012.03.036. [PMID] 22541070.
2012
Efficient cochlear gene transfection in guinea-pigs with adeno-associated viral vectors by partial digestion of round window membrane.
Gene therapy. 19(3):255-63 [DOI] 10.1038/gt.2011.91. [PMID] 21697953.
2012
Efficient Transduction of Tyrosine-To-Phenylalanine Mutated Aav2 Vectors Carrying Human Nd4 Gene and Biodistribution Following Intravitreal Delivery in a Rodent Model – a Gene Therapy for Leber Hereditary Optic Neuropathy
Molecular Therapy. 20
2012
ER stress is involved in T17M rhodopsin-induced retinal degeneration.
Investigative ophthalmology & visual science. 53(7):3792-800 [DOI] 10.1167/iovs.11-9235. [PMID] 22589437.
2012
ERK1/2 activation is a therapeutic target in age-related macular degeneration.
Proceedings of the National Academy of Sciences of the United States of America. 109(34):13781-6 [DOI] 10.1073/pnas.1206494109. [PMID] 22869729.
2012
Functional rescue of P23H rhodopsin photoreceptors by gene delivery.
Advances in experimental medicine and biology. 723:191-7 [DOI] 10.1007/978-1-4614-0631-0_26. [PMID] 22183333.
2012
Gene delivery of wild-type rhodopsin rescues retinal function in an autosomal dominant retinitis pigmentosa mouse model.
Advances in experimental medicine and biology. 723:199-205 [DOI] 10.1007/978-1-4614-0631-0_27. [PMID] 22183334.
2012
Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model.
Proceedings of the National Academy of Sciences of the United States of America. 109(20):E1238-47 [DOI] 10.1073/pnas.1119577109. [PMID] 22523243.
2012
Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
Archives of ophthalmology (Chicago, Ill. : 1960). 130(1):9-24 [DOI] 10.1001/archophthalmol.2011.298. [PMID] 21911650.
2012
Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept.
Human gene therapy. 23(4):367-76 [DOI] 10.1089/hum.2011.169. [PMID] 22142163.
2012
Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa.
Proceedings of the National Academy of Sciences of the United States of America. 109(6):2132-7 [DOI] 10.1073/pnas.1118847109. [PMID] 22308428.
2012
Long-Term Rescue By Aav-Delivered Therapy in Autosomal Dominant Retinitis Pigmentosa (Adrp) Mice
Molecular Therapy. 20
2012
Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice.
Human gene therapy. 23(4):356-66 [DOI] 10.1089/hum.2011.213. [PMID] 22289036.
2012
Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice.
Molecular vision. 18:1668-83 [PMID] 22773905.
2012
Organizational motifs for ground squirrel cone bipolar cells.
The Journal of comparative neurology. 520(13):2864-87 [DOI] 10.1002/cne.23068. [PMID] 22778006.
2012
Progeny of Pronuclear Injections of Mutant Human Mitochondrial Genes
Molecular Therapy. 20
2012
Regenerative medicine for the special senses: restoring the inputs.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 32(41):14053-7 [DOI] 10.1523/JNEUROSCI.3336-12.2012. [PMID] 23055472.
2012
Suppression of rds expression by siRNA and gene replacement strategies for gene therapy using rAAV vector.
Advances in experimental medicine and biology. 723:215-23 [DOI] 10.1007/978-1-4614-0631-0_29. [PMID] 22183336.
2012
Targeted Gene Editing in Rod Photoreceptor Cells in Vivo
Cell Transplantation. 21(4)
2012
The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.
Human gene therapy. 23(10):1101-15 [DOI] 10.1089/hum.2012.125. [PMID] 22845794.
2012
Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats.
Investigative ophthalmology & visual science. 53(4):1895-904 [DOI] 10.1167/iovs.11-8831. [PMID] 22408006.
2012
γ-Secretase inhibition of murine choroidal neovascularization is associated with reduction of superoxide and proinflammatory cytokines.
Investigative ophthalmology & visual science. 53(2):574-85 [DOI] 10.1167/iovs.11-8728. [PMID] 22205609.
2011
AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa.
Human gene therapy. 22(5):567-75 [DOI] 10.1089/hum.2010.140. [PMID] 21126223.
2011
Ab-externo AAV-mediated gene delivery to the suprachoroidal space using a 250 micron flexible microcatheter.
PloS one. 6(2) [DOI] 10.1371/journal.pone.0017140. [PMID] 21347253.
2011
Alternative splice variants of the USH3A gene Clarin 1 (CLRN1).
European journal of human genetics : EJHG. 19(1):30-5 [DOI] 10.1038/ejhg.2010.140. [PMID] 20717163.
2011
DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration.
Nature. 471(7338):325-30 [DOI] 10.1038/nature09830. [PMID] 21297615.
2011
Efficient mutagenesis of the rhodopsin gene in rod photoreceptor neurons in mice.
Nucleic acids research. 39(14):5955-66 [DOI] 10.1093/nar/gkr196. [PMID] 21478169.
2011
Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model.
Proceedings of the National Academy of Sciences of the United States of America. 108(15):6276-81 [DOI] 10.1073/pnas.1019222108. [PMID] 21444805.
2011
Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis.
Investigative ophthalmology & visual science. 52(1):7-15 [DOI] 10.1167/iovs.10-6138. [PMID] 21169527.
2011
Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis.
Human molecular genetics. 20(23):4569-81 [DOI] 10.1093/hmg/ddr391. [PMID] 21880665.
2011
Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule.
Molecular therapy : the journal of the American Society of Gene Therapy. 19(2):260-5 [DOI] 10.1038/mt.2010.230. [PMID] 20978476.
2011
Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.
Investigative ophthalmology & visual science. 52(10):7098-108 [DOI] 10.1167/iovs.11-7867. [PMID] 21778276.
2011
Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.
Molecular therapy : the journal of the American Society of Gene Therapy. 19(2):234-42 [DOI] 10.1038/mt.2010.273. [PMID] 21139570.
2011
Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model.
Proceedings of the National Academy of Sciences of the United States of America. 108(45):18476-81 [DOI] 10.1073/pnas.1112758108. [PMID] 22042849.
2011
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina.
Molecular therapy : the journal of the American Society of Gene Therapy. 19(2):293-301 [DOI] 10.1038/mt.2010.234. [PMID] 21045809.
2011
Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines.
Molecular vision. 17:1090-102 [PMID] 21552473.
2011
Role of connective tissue growth factor in the retinal vasculature during development and ischemia.
Investigative ophthalmology & visual science. 52(12):8701-10 [DOI] 10.1167/iovs.11-7870. [PMID] 21969300.
2011
Towards optogenetic sensory replacement.
Annual International Conference of the IEEE Engineering in Medicine and Biology Society. IEEE Engineering in Medicine and Biology Society. Annual International Conference. 2011:3139-41 [DOI] 10.1109/IEMBS.2011.6090856. [PMID] 22255005.
2011
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness.
Molecular therapy : the journal of the American Society of Gene Therapy. 19(7):1220-9 [DOI] 10.1038/mt.2011.69. [PMID] 21505421.
2011
Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors.
Investigative ophthalmology & visual science. 52(5):2343-51 [DOI] 10.1167/iovs.10-6141. [PMID] 21212183.
2011
XIAP therapy increases survival of transplanted rod precursors in a degenerating host retina.
Investigative ophthalmology & visual science. 52(3):1567-72 [DOI] 10.1167/iovs.10-5998. [PMID] 20926819.
2011
γ-Secretase and presenilin mediate cleavage and phosphorylation of vascular endothelial growth factor receptor-1.
The Journal of biological chemistry. 286(49):42514-42523 [DOI] 10.1074/jbc.M111.296590. [PMID] 22016384.
2010
Achromatopsia as a potential candidate for gene therapy.
Advances in experimental medicine and biology. 664:639-46 [DOI] 10.1007/978-1-4419-1399-9_73. [PMID] 20238068.
2010
Diabetic eNOS-knockout mice develop accelerated retinopathy.
Investigative ophthalmology & visual science. 51(10):5240-6 [DOI] 10.1167/iovs.09-5147. [PMID] 20435587.
2010
Enhanced Cns Transgene Expression By Raav Capsid Tyrosine Mutants
Cell Transplantation. 19
2010
Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.
PloS one. 5(6) [DOI] 10.1371/journal.pone.0011306. [PMID] 20593011.
2010
Gene therapy rescues cone function in congenital achromatopsia.
Human molecular genetics. 19(13):2581-93 [DOI] 10.1093/hmg/ddq136. [PMID] 20378608.
2010
Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus.
Archives of ophthalmology (Chicago, Ill. : 1960). 128(7):876-83 [DOI] 10.1001/archophthalmol.2010.135. [PMID] 20625049.
2010
Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1.
Archives of ophthalmology (Chicago, Ill. : 1960). 128(9):1129-35 [DOI] 10.1001/archophthalmol.2010.201. [PMID] 20837795.
2010
Mechanistic basis for the failure of cone transducin to translocate: why cones are never blinded by light.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 30(20):6815-24 [DOI] 10.1523/JNEUROSCI.0613-10.2010. [PMID] 20484624.
2010
Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel.
Human gene therapy. 21(12):1749-57 [DOI] 10.1089/hum.2010.047. [PMID] 20604683.
2010
rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters.
Gene therapy. 17(9):1162-74 [DOI] 10.1038/gt.2010.56. [PMID] 20428215.
2010
Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78.
Proceedings of the National Academy of Sciences of the United States of America. 107(13):5961-6 [DOI] 10.1073/pnas.0911991107. [PMID] 20231467.
2010
Retinal disease in Rpe65-deficient mice: comparison to human leber congenital amaurosis due to RPE65 mutations.
Investigative ophthalmology & visual science. 51(10):5304-13 [DOI] 10.1167/iovs.10-5559. [PMID] 20484585.
2010
Selective tropism of the recombinant adeno-associated virus 9 serotype for rat cardiac tissue.
The journal of gene medicine. 12(1):22-34 [DOI] 10.1002/jgm.1404. [PMID] 19830780.
2010
Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.
Gene therapy. 17(7):815-26 [DOI] 10.1038/gt.2010.29. [PMID] 20237510.
2010
Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse.
Experimental eye research. 90(5):546-54 [DOI] 10.1016/j.exer.2010.01.011. [PMID] 20138034.
2009
AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector.
Molecular vision. 15:1835-42 [PMID] 19756181.
2009
Angiostatin overexpression is associated with an improvement in chronic kidney injury by an anti-inflammatory mechanism.
American journal of physiology. Renal physiology. 296(1):F145-52 [DOI] 10.1152/ajprenal.90430.2008. [PMID] 18971211.
2009
Angiotensin Type 2 Receptor Improves Cardiac Function and Attenuates Left Ventricular Remodeling Following Myocardial Infarction in the Rat
Hypertension. 54:E71-E72
2009
Clarin-1, encoded by the Usher Syndrome III causative gene, forms a membranous microdomain: possible role of clarin-1 in organizing the actin cytoskeleton.
The Journal of biological chemistry. 284(28):18980-93 [DOI] 10.1074/jbc.M109.003160. [PMID] 19423712.
2009
Dose-Dependent Humoral Immune Responses To Intravitreal Delivery of Aav Vectors and Strategies To Circumvent
Molecular Therapy. 17
2009
Effect of CNTF on retinal ganglion cell survival in experimental glaucoma.
Investigative ophthalmology & visual science. 50(5):2194-200 [DOI] 10.1167/iovs.08-3013. [PMID] 19060281.
2009
Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system.
Investigative ophthalmology & visual science. 50(9):4205-14 [DOI] 10.1167/iovs.08-3214. [PMID] 19387075.
2009
Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina.
Molecular vision. 15:2796-802 [PMID] 20019878.
2009
Functional interchangeability of rod and cone transducin alpha-subunits.
Proceedings of the National Academy of Sciences of the United States of America. 106(42):17681-6 [DOI] 10.1073/pnas.0901382106. [PMID] 19815523.
2009
Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye.
Molecular vision. 15:267-75 [PMID] 19190735.
2009
Gene therapy for red-green colour blindness in adult primates.
Nature. 461(7265):784-7 [DOI] 10.1038/nature08401. [PMID] 19759534.
2009
Gene Therapy Prevents Cone Degeneration in Two Models of Rpe65 Leber Congenital Amaurosis: Rd12 and Rpe65(-/-):: Rho(-/-) Mice
Molecular Therapy. 17:S288-S289
2009
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors.
Molecular therapy : the journal of the American Society of Gene Therapy. 17(3):463-71 [DOI] 10.1038/mt.2008.269. [PMID] 19066593.
2009
Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year.
Human gene therapy. 20(9):999-1004 [DOI] 10.1089/hum.2009.086. [PMID] 19583479.
2009
Lowering blood pressure blocks mesangiolysis and mesangial nodules, but not tubulointerstitial injury, in diabetic eNOS knockout mice.
The American journal of pathology. 174(4):1221-9 [DOI] 10.2353/ajpath.2009.080605. [PMID] 19246639.
2009
Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization.
Gene therapy. 16(1):10-6 [DOI] 10.1038/gt.2008.115. [PMID] 18633446.
2009
Safety and Efficacy of Intravitreal Anti-Vegf Gene Delivery in Non-Human Primates for the Treatment of Ocular Neovascularization
Molecular Therapy. 17:S287-S288
2009
Suppression of Rds Expression By Sirna and Gene Replacement Strategies for Gene Therapy Using Aav Vector
Molecular Therapy. 17
2009
Vision 1 year after gene therapy for Leber’s congenital amaurosis.
The New England journal of medicine. 361(7):725-7 [DOI] 10.1056/NEJMc0903652. [PMID] 19675341.
2009
XIAP effects on retinal detachment-induced photoreceptor apoptosis [corrected].
Investigative ophthalmology & visual science. 50(3):1448-53 [DOI] 10.1167/iovs.08-2855. [PMID] 19060276.
2008
AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation.
Investigative ophthalmology & visual science. 49(10):4278-83 [DOI] 10.1167/iovs.07-1622. [PMID] 18586879.
2008
Aav-Mediated Gene Transfer of Ace2 Protects Retinal Vascular Dysfunction in An Animal Model of Diabetic Retinopathy
Journal of Hypertension. 26
2008
Adeno-Associated Viral Vectors and the Retina
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2008
Adeno-associated viral vectors and the retina.
Advances in experimental medicine and biology. 613:121-8 [DOI] 10.1007/978-0-387-74904-4_13. [PMID] 18188936.
2008
An Anti-Vegf Aav Gene Therapy Approach for Neovascular Complications of Age-Related Macular Degeneration – Efficacy and Safety Studies in Murine and Primate Models
Human Gene Therapy. 19
2008
Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: effects of serotype and site of administration.
Vision research. 48(3):377-85 [PMID] 17950399.
2008
Cone-specific expression using a human red opsin promoter in recombinant AAV.
Vision research. 48(3):332-8 [PMID] 17905404.
2008
Dicer inactivation leads to progressive functional and structural degeneration of the mouse retina.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 28(19):4878-87 [DOI] 10.1523/JNEUROSCI.0828-08.2008. [PMID] 18463241.
2008
Different tropism of adenoviruses and adeno-associated viruses to corneal cells: implications for corneal gene therapy.
Molecular vision. 14:2087-96 [PMID] 19023450.
2008
Downregulation of p22phox in retinal pigment epithelial cells inhibits choroidal neovascularization in mice.
Molecular therapy : the journal of the American Society of Gene Therapy. 16(10):1688-94 [DOI] 10.1038/mt.2008.164. [PMID] 18665154.
2008
Effect of late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouse.
Molecular therapy : the journal of the American Society of Gene Therapy. 16(6):1010-7 [DOI] 10.1038/mt.2008.57. [PMID] 18388913.
2008
Gene therapy for mouse models of ADRP.
Advances in experimental medicine and biology. 613:107-12 [DOI] 10.1007/978-0-387-74904-4_11. [PMID] 18188934.
2008
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.
Proceedings of the National Academy of Sciences of the United States of America. 105(39):15112-7 [DOI] 10.1073/pnas.0807027105. [PMID] 18809924.
2008
Intraocular Route of Aav2 Vector Administration Defines Humoral Immune Response and Therapeutic Potential
Molecular Vision. 14:1760-1769
2008
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential.
Molecular vision. 14:1760-9 [PMID] 18836574.
2008
Longitudinal evaluation of expression of virally delivered transgenes in gerbil cone photoreceptors.
Visual neuroscience. 25(3):273-82 [DOI] 10.1017/S0952523808080577. [PMID] 18598398.
2008
Prospects for retinal cone-targeted gene therapy.
Drug news & perspectives. 21(5):267-71 [DOI] 10.1358/dnp.2008.21.5.1219012. [PMID] 18596991.
2008
Targeting Gene Expression To Cones With Human Cone Opsin Promoters in Recombinant Aav (Vol 15, Pg 1049, 2008)
Gene Therapy. 15(14):1073-1073 [DOI] 10.1038/gt.2008.107.
2008
Targeting gene expression to cones with human cone opsin promoters in recombinant AAV.
Gene therapy. 15(14):1049-55 [DOI] 10.1038/gt.2008.32. [PMID] 18337838.
2008
Toward a higher fidelity model of AMD.
Advances in experimental medicine and biology. 613:185-92 [DOI] 10.1007/978-0-387-74904-4_21. [PMID] 18188944.
2008
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Human gene therapy. 19(10):979-90 [DOI] 10.1089/hum.2008.107. [PMID] 18774912.
2007
Anti-clarin-1 AAV-delivered ribozyme induced apoptosis in the mouse cochlea.
Hearing research. 230(1-2):9-16 [PMID] 17493778.
2007
Brain-derived neurotrophic factor gene transfer with adeno-associated viral and lentiviral vectors prevents rubrospinal neuronal atrophy and stimulates regeneration-associated gene expression after acute cervical spinal cord injury.
Spine. 32(11):1164-73 [PMID] 17495772.
2007
Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation.
PLoS medicine. 4(6) [PMID] 17594175.
2007
Dual gene therapy with extracellular superoxide dismutase and catalase attenuates experimental optic neuritis.
Molecular vision. 13:1-11 [PMID] 17242675.
2007
Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis.
Molecular vision. 13:1701-10 [PMID] 17960108.
2007
Increased sensitivity to light-induced damage in a mouse model of autosomal dominant retinal disease.
Investigative ophthalmology & visual science. 48(5):1942-51 [PMID] 17460245.
2007
Intraocular CNTF reduces vision in normal rats in a dose-dependent manner.
Investigative ophthalmology & visual science. 48(12):5756-66 [PMID] 18055829.
2007
Long-term suppression of neurodegeneration in chronic experimental optic neuritis: antioxidant gene therapy.
Investigative ophthalmology & visual science. 48(12):5360-70 [PMID] 18055782.
2007
Molecular and cellular alterations induced by sustained expression of ciliary neurotrophic factor in a mouse model of retinitis pigmentosa.
Investigative ophthalmology & visual science. 48(3):1389-400 [PMID] 17325188.
2007
Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme.
Experimental eye research. 84(1):44-52 [PMID] 17083931.
2007
Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virus.
Genetic vaccines and therapy. 5 [PMID] 18070352.
2007
Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina.
Journal of the Optical Society of America. A, Optics, image science, and vision. 24(5):1411-6 [PMID] 17429487.
2007
Restoration of cone vision in a mouse model of achromatopsia.
Nature medicine. 13(6):685-7 [PMID] 17515894.
2007
SOD2 knockdown mouse model of early AMD.
Investigative ophthalmology & visual science. 48(10):4407-20 [PMID] 17898259.
2007
Suppression of mitochondrial oxidative stress provides long-term neuroprotection in experimental optic neuritis.
Investigative ophthalmology & visual science. 48(2):681-91 [PMID] 17251466.
2007
Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery.
Vision research. 47(9):1202-8 [PMID] 17292939.
2007
The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse.
Investigative ophthalmology & visual science. 48(1):1-10 [PMID] 17197509.
2007
Use of mitochondrial antioxidant defenses for rescue of cells with a Leber hereditary optic neuropathy-causing mutation.
Archives of ophthalmology (Chicago, Ill. : 1960). 125(2):268-72 [PMID] 17296905.
2007
XIAP protection of photoreceptors in animal models of retinitis pigmentosa.
PloS one. 2(3) [PMID] 17375200.
2006
Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function.
Vision research. 46(22):3926-34 [PMID] 16814838.
2006
Disease mechanisms and gene therapy in a mouse model for X-linked retinoschisis.
Advances in experimental medicine and biology. 572:283-9 [PMID] 17249585.
2006
Does recombinant adeno-associated virus-vectored proximal region of mouse rhodopsin promoter support only rod-type specific expression in vivo?
Molecular vision. 12:298-309 [PMID] 16617297.
2006
Down-regulation of rhodopsin gene expression by AAV-vectored short interfering RNA.
Advances in experimental medicine and biology. 572:233-8 [PMID] 17249579.
2006
Extracellular signal-regulated kinases 1/2 are required for adult retinal ganglion cell axon regeneration induced by fibroblast growth factor-2.
Journal of neuroscience research. 83(6):985-95 [PMID] 16493686.
2006
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis.
Molecular therapy : the journal of the American Society of Gene Therapy. 13(3):565-72 [PMID] 16223604.
2006
Human blue-opsin promoter preferentially targets reporter gene expression to rat s-cone photoreceptors.
Investigative ophthalmology & visual science. 47(8):3505-13 [PMID] 16877422.
2006
Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis.
PLoS medicine. 3(10) [PMID] 17032058.
2006
Lentiviral vectors containing a retinal pigment epithelium specific promoter for leber congenital amaurosis gene therapy. Lentiviral gene therapy for LCA.
Advances in experimental medicine and biology. 572:247-53 [PMID] 17249581.
2006
Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1.
Investigative ophthalmology & visual science. 47(9):3745-53 [PMID] 16936082.
2006
Mitochondrial protein nitration primes neurodegeneration in experimental autoimmune encephalomyelitis.
The Journal of biological chemistry. 281(42):31950-62 [PMID] 16920708.
2006
Proceedings of the Third International Symposium on Retinopathy of Prematurity: an update on ROP from the lab to the nursery (November 2003, Anaheim, California).
Molecular vision. 12:532-80 [PMID] 16735995.
2006
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.
Human gene therapy. 17(8):845-58 [PMID] 16942444.
2006
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection.
Molecular therapy : the journal of the American Society of Gene Therapy. 13(6):1074-84 [PMID] 16644289.
2006
XIAP-mediated neuroprotection in retinal ischemia.
Gene therapy. 13(4):339-47 [PMID] 16307001.
2005
Activation of the extracellular signal-regulated kinase 1/2 pathway by AAV gene transfer protects retinal ganglion cells in glaucoma.
Molecular therapy : the journal of the American Society of Gene Therapy. 12(3):402-12 [PMID] 15975850.
2005
Adeno-associated virus-mediated expression of vascular endothelial growth factor peptides inhibits retinal neovascularization in a mouse model of oxygen-induced retinopathy.
Human gene therapy. 16(11):1247-54 [PMID] 16259558.
2005
Adeno-associated virus-vectored gene therapy for retinal disease.
Human gene therapy. 16(6):649-63 [PMID] 15960597.
2005
Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors.
Human gene therapy. 16(2):235-47 [PMID] 15761263.
2005
Extracellular signal-regulated kinase 1/2 mediates survival, but not axon regeneration, of adult injured central nervous system neurons in vivo.
Journal of neurochemistry. 93(1):72-83 [PMID] 15773907.
2005
IL-10 suppresses chemokines, inflammation, and fibrosis in a model of chronic renal disease.
Journal of the American Society of Nephrology : JASN. 16(12):3651-60 [PMID] 16251240.
2005
Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway.
Proceedings of the National Academy of Sciences of the United States of America. 102(20):7251-6 [PMID] 15878989.
2005
Knockdown of wild-type mouse rhodopsin using an AAV vectored ribozyme as part of an RNA replacement approach.
Molecular vision. 11:648-56 [PMID] 16145542.
2005
Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts.
Molecular therapy : the journal of the American Society of Gene Therapy. 12(3):519-27 [PMID] 15979413.
2005
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
Molecular therapy : the journal of the American Society of Gene Therapy. 12(6):1072-82 [PMID] 16226919.
2005
Modulation of the expression of the transcription factor Max in rat retinal ganglion cells by a recombinant adeno-associated viral vector.
Brazilian journal of medical and biological research = Revista brasileira de pesquisas medicas e biologicas. 38(3):375-9 [PMID] 15761617.
2005
Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis.
PLoS medicine. 2(11) [PMID] 16250670.
2005
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis.
Molecular therapy : the journal of the American Society of Gene Therapy. 12(4):644-51 [PMID] 16027044.
2005
Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA).
Molecular vision. 11:152-62 [PMID] 15765048.
2005
Ribozyme knockdown of the gamma-subunit of rod cGMP phosphodiesterase alters the ERG and retinal morphology in wild-type mice.
Investigative ophthalmology & visual science. 46(10):3836-44 [PMID] 16186371.
2005
The consortium project to treat RPE65 deficiency in humans.
Retina (Philadelphia, Pa.). 25(8 Suppl) [PMID] 16374340.
2004
AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.
Molecular therapy : the journal of the American Society of Gene Therapy. 10(1):106-16 [PMID] 15233947.
2004
Design and validation of therapeutic hammerhead ribozymes for autosomal dominant diseases.
Methods in molecular biology (Clifton, N.J.). 252:221-36 [PMID] 15017052.
2004
Range of retinal diseases potentially treatable by AAV-vectored gene therapy.
Novartis Foundation symposium. 255:179-88; discussion 188 [PMID] 14750604.
2004
SOD2 gene transfer protects against optic neuropathy induced by deficiency of complex I.
Annals of neurology. 56(2):182-91 [PMID] 15293270.
2003
A muscleblind knockout model for myotonic dystrophy.
Science (New York, N.Y.). 302(5652):1978-80 [PMID] 14671308.
2003
Anti-apoptotic effects of CNTF gene transfer on photoreceptor degeneration in experimental antibody-induced retinopathy.
Journal of autoimmunity. 21(2):121-9 [PMID] 12935781.
2003
Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors.
Journal of the American Society of Nephrology : JASN. 14(4):947-58 [PMID] 12660329.
2003
Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model.
Investigative ophthalmology & visual science. 44(10):4357-65 [PMID] 14507880.
2003
Optic neuropathy induced by reductions in mitochondrial superoxide dismutase.
Investigative ophthalmology & visual science. 44(3):1088-96 [PMID] 12601034.
2003
Structural and functional protection of photoreceptors from MNU-induced retinal degeneration by the X-linked inhibitor of apoptosis.
Investigative ophthalmology & visual science. 44(6):2757-63 [PMID] 12766084.
2003
Suppression of complex I gene expression induces optic neuropathy.
Annals of neurology. 53(2):198-205 [PMID] 12557286.
2003
XIAP protects photoreceptors from n-methyl-n-nitrosourea-induced retinal degeneration.
Advances in experimental medicine and biology. 533:385-93 [PMID] 15180289.
2002
AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization.
Investigative ophthalmology & visual science. 43(6):1994-2000 [PMID] 12037010.
2002
Adeno-associated virus type-2 expression of pigmented epithelium-derived factor or Kringles 1-3 of angiostatin reduce retinal neovascularization.
Proceedings of the National Academy of Sciences of the United States of America. 99(13):8909-14 [PMID] 12072560.
2002
Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model.
Molecular therapy : the journal of the American Society of Gene Therapy. 5(6):780-7 [PMID] 12027563.
2002
Designing and characterizing hammerhead ribozymes for use in AAV vector-mediated retinal gene therapies.
Methods in enzymology. 346:358-77 [PMID] 11883079.
2002
Development of hammerhead ribozymes to modulate endogenous gene expression for functional studies.
Methods (San Diego, Calif.). 28(2):276-85 [PMID] 12413427.
2002
Effects of adeno-associated virus-vectored ciliary neurotrophic factor on retinal structure and function in mice with a P216L rds/peripherin mutation.
Experimental eye research. 74(6):719-35 [PMID] 12126945.
2002
In organello footprinting of mtDNA.
Methods in molecular biology (Clifton, N.J.). 197:363-75 [PMID] 12013810.
2002
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors.
Methods (San Diego, Calif.). 28(2):158-67 [PMID] 12413414.
2002
Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy.
Annals of neurology. 52(5):534-42 [PMID] 12402249.
2002
TrkB gene transfer protects retinal ganglion cells from axotomy-induced death in vivo.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 22(10):3977-86 [PMID] 12019317.
2001
An allele-specific hammerhead ribozyme gene therapy for a porcine model of autosomal dominant retinitis pigmentosa.
Molecular vision. 7:6-13 [PMID] 11172137.
2001
Gene therapy restores vision in a canine model of childhood blindness.
Nature genetics. 28(1):92-5 [PMID] 11326284.
2001
Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa.
Molecular therapy : the journal of the American Society of Gene Therapy. 4(6):622-9 [PMID] 11735347.
2001
Inhibition of gene expression by ribozymes.
Methods in molecular medicine. 47:105-24 [DOI] 10.1385/1-59259-085-3:105. [PMID] 21394581.
2001
Noninvasive imaging by optical coherence tomography to monitor retinal degeneration in the mouse.
Investigative ophthalmology & visual science. 42(12):2981-9 [PMID] 11687546.
2001
Ribozyme gene therapy: applications for molecular medicine.
Trends in molecular medicine. 7(5):221-8 [PMID] 11325634.
2001
Use of recombinant angiostatin to prevent retinal neovascularization.
Transactions of the American Clinical and Climatological Association. 112:68-75; discussion 75 [PMID] 11413784.
2000
Ocular gene therapy: quo vadis?
Investigative ophthalmology & visual science. 41(10):2821-6 [PMID] 10967033.
2000
Production and purification of recombinant adeno-associated virus.
Methods in enzymology. 316:743-61 [PMID] 10800712.
2000
Recombinant adeno-associated virus vector-based gene transfer for defects in oxidative metabolism.
Human gene therapy. 11(15):2067-78 [PMID] 11044909.
2000
Ribozyme gene therapy for autosomal dominant retinal disease.
Clinical chemistry and laboratory medicine. 38(2):147-53 [PMID] 10834402.
2000
Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy.
Proceedings of the National Academy of Sciences of the United States of America. 97(21):11488-93 [PMID] 11005848.
2000
Ribozyme uses in retinal gene therapy.
Progress in retinal and eye research. 19(6):689-710 [PMID] 11029552.
2000
Ribozymes in treatment of inherited retinal disease.
Methods in enzymology. 316:761-76 [PMID] 10800713.
2000
The eyes have it.
Molecular medicine today. 6(2) [PMID] 10681166.
1999
Adenoviral gene therapy with catalase suppresses experimental optic neuritis.
Archives of ophthalmology (Chicago, Ill. : 1960). 117(11):1533-9 [PMID] 10565523.
1999
Ischemia induces a selective biphasic response in brain mitochondrial mRNA levels.
Neuroreport. 10(7):1593-8 [PMID] 10380987.
1999
Mitochondrial gene expression is regulated at the level of transcription during early embryogenesis of Xenopus laevis.
The Journal of biological chemistry. 274(10):6265-71 [PMID] 10037714.
1999
Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve.
Archives of ophthalmology (Chicago, Ill. : 1960). 117(7):929-37 [PMID] 10408459.
1998
Adeno-associated viral-mediated catalase expression suppresses optic neuritis in experimental allergic encephalomyelitis.
Proceedings of the National Academy of Sciences of the United States of America. 95(23):13847-52 [PMID] 9811889.
1998
Retinal gene therapy 1998: summary of a workshop.
Molecular vision. 4 [PMID] 9675216.
1998
Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa.
Nature medicine. 4(8):967-71 [PMID] 9701253.
1998
Ribozyme-targeted destruction of RNA associated with autosomal-dominant retinitis pigmentosa.
Investigative ophthalmology & visual science. 39(5):681-9 [PMID] 9538873.
1998
Selective degradation of nonsense beta-phosphodiesterase mRNA in the heterozygous rd mouse.
Investigative ophthalmology & visual science. 39(13):2529-36 [PMID] 9856762.
1997
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus.
Proceedings of the National Academy of Sciences of the United States of America. 94(13):6916-21 [PMID] 9192666.
1996
A “humanized” green fluorescent protein cDNA adapted for high-level expression in mammalian cells.
Journal of virology. 70(7):4646-54 [PMID] 8676491.
1996
Developmentally important DNA elements within the bovine opsin upstream region.
Investigative ophthalmology & visual science. 37(1):154-65 [PMID] 8550318.
1996
Genomic footprinting of mitochondrial DNA: I. In organello analysis of protein-mitochondrial DNA interactions in bovine mitochondria.
Methods in enzymology. 264:12-22 [PMID] 8965685.
1996
Genomic footprinting of mitochondrial DNA: II. In vivo analysis of protein-mitochondrial DNA interactions in Xenopus laevis eggs and embryos.
Methods in enzymology. 264:23-36 [PMID] 8965697.
1995
Bovine opsin gene expression exhibits a late fetal to adult regulatory switch.
Journal of neuroscience research. 40(6):728-36 [PMID] 7543158.
1995
Fetal topography of bovine rhodopsin mRNA suggests retinotopographically determined gene expression.
Investigative ophthalmology & visual science. 36(10):2008-19 [PMID] 7544781.
1995
Selective DNA amplification regulates transcript levels in plant mitochondria.
Current genetics. 28(2):113-21 [PMID] 8590461.
1995
Topographical regulation of cone and rod opsin genes: parallel, position dependent levels of transcription.
Brain research. Developmental brain research. 89(1):146-9 [PMID] 8575088.
1994
Ancient DNA: an introduction.
Experientia. 50(6):521-3 [PMID] 8020611.
1994
In organello footprint analysis of human mitochondrial DNA: human mitochondrial transcription factor A interactions at the origin of replication.
Molecular and cellular biology. 14(12):7717-30 [PMID] 7969115.
1994
Inter- and intrapopulation studies of ancient humans.
Experientia. 50(6):585-91 [PMID] 8020619.
1994
Parallel regulation of fetal gene expression in different photoreceptor cell types.
The Journal of biological chemistry. 269(7):4986-92 [PMID] 8106474.
1993
In organello footprinting. Analysis of protein binding at regulatory regions in bovine mitochondrial DNA.
The Journal of biological chemistry. 268(12):8675-82 [PMID] 8473311.
1993
In vivo and in vitro evidence for slipped mispairing in mammalian mitochondria.
Proceedings of the National Academy of Sciences of the United States of America. 90(16):7671-5 [PMID] 8356068.
1993
Protein binding to a single termination-associated sequence in the mitochondrial DNA D-loop region.
Molecular and cellular biology. 13(4):2162-71 [PMID] 8455604.
1993
Synthesis and stability of retinal photoreceptor mRNAs are coordinately regulated during bovine fetal development.
Experimental eye research. 56(3):257-65 [PMID] 8386100.
1993
Transcribed heteroplasmic repeated sequences in the porcine mitochondrial DNA D-loop region.
Journal of molecular evolution. 37(1):36-7 [PMID] 8360917.
1993
Transcription of photoreceptor genes during fetal retinal development. Evidence for positive and negative regulation.
The Journal of biological chemistry. 268(10):6953-60 [PMID] 8463227.
1992
DNA helicase from mammalian mitochondria.
Proceedings of the National Academy of Sciences of the United States of America. 89(18):8562-6 [PMID] 1326759.
1992
Early expression and localization of rhodopsin and interphotoreceptor retinoid-binding protein (IRBP) in the developing fetal bovine retina.
Experimental eye research. 54(5):661-70 [PMID] 1623951.
1992
Transcription in maize mitochondria: effects of tissue and mitochondrial genotype.
Current genetics. 22(3):235-42 [PMID] 1381994.
1991
Ancient HLA genes from 7,500-year-old archaeological remains.
Nature. 349(6312):785-8 [PMID] 2000147.
1989
Rapid segregation of heteroplasmic bovine mitochondria.
Nucleic acids research. 17(18):7325-31 [PMID] 2798094.
1988
Nucleotide sequence of the Zea mays mitochondrial cytochrome oxidase subunit III gene.
Nucleic acids research. 16(20) [PMID] 2847119.
1988
Sequence and transcriptional analysis of a chloroplast insert in the mitochondrial genome of Zea mays.
Current genetics. 13(6):509-15 [PMID] 2841036.
1988
Unequal partitioning of bovine mitochondrial genotypes among siblings.
Proceedings of the National Academy of Sciences of the United States of America. 85(21):8107-10 [PMID] 2460862.
1987
The molecular basis of inhibitor resistance in a mammalian mitochondrial cytochrome b mutant.
The Journal of biological chemistry. 262(5):2411-4 [PMID] 3818601.
1986
Anatomical, cellular and molecular analysis of 8,000-yr-old human brain tissue from the Windover archaeological site.
Nature. 323(6091):803-6 [PMID] 2430186.
1986
Conserved restriction sites within the ribosomal RNA genes of vertebrates.
Biochimica et biophysica acta. 866(1):19-25 [PMID] 3004584.
1986
Template-directed arrest of mammalian mitochondrial DNA synthesis.
Molecular and cellular biology. 6(4):1261-7 [PMID] 3785163.
1985
A method for assessing damage to mitochondrial DNA caused by radiation and epichlorohydrin.
Molecular pharmacology. 27(1):167-70 [PMID] 3965928.
1985
Length heterogeneity of a conserved displacement-loop sequence in human mitochondrial DNA.
Nucleic acids research. 13(22):8093-104 [PMID] 4070000.
1985
Replication priming and transcription initiate from precisely the same site in mouse mitochondrial DNA.
The EMBO journal. 4(6):1559-67 [PMID] 2411543.
1984
Heterogeneous mitochondrial DNA D-loop sequences in bovine tissue.
Cell. 37(3):1001-7 [PMID] 6744410.
1983
Nucleotide sequence evidence for rapid genotypic shifts in the bovine mitochondrial DNA D-loop.
Nature. 306(5941):400-2 [PMID] 6646218.
1982
Mitochondrial DNA copy number in bovine oocytes and somatic cells.
Developmental biology. 94(1):246-51 [PMID] 6295849.
1982
Mitochondrial DNA polymorphism in a maternal lineage of Holstein cows.
Proceedings of the National Academy of Sciences of the United States of America. 79(15):4686-90 [PMID] 6289312.
1980
Genetic mapping of bovine mitochondrial DNA from a single animal.
Gene. 8(2):193-209 [PMID] 7358273.
1980
Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells.
Journal of virology. 33(2):739-48 [PMID] 6251245.
1979
A physical map of bovine mitochondrial DNA from a single animal.
Biochimica et biophysica acta. 565(1):22-32 [PMID] 508763.
1979
Adeno-associated virus DNA replication.
Cold Spring Harbor symposia on quantitative biology. 43 Pt 2:781-7 [PMID] 226321.
1979
Adeno-associated virus DNA replication: nonunit-length molecules.
Virology. 93(1):57-68 [PMID] 219605.
1979
Adeno-associated viruses.
Advances in virus research. 25:407-49 [PMID] 230727.
1977
Cytidine-C(5)-photoexchange: a kinetic analysis.
Photochemistry and photobiology. 26(3):231-4 [PMID] 918150.
1977
Evidence for two nucleotide sequence orientations within the terminal repetition of adeno-associated virus DNA.
Journal of virology. 24(2):627-34 [PMID] 916029.
1977
Excited state processes and solution conformation of dipyrimidine adducts.
Photochemistry and photobiology. 25(2):161-6 [PMID] 866449.
1977
Origin and termination of adeno-associated virus DNA replication.
Virology. 78(2):488-99 [PMID] 867815.
1973
Pyrimidine adduct fluorescence in UV irradiated nucleic acids.
Biochemical and biophysical research communications. 51(3):819-26 [PMID] 4735776.
1972
Photohydration of pyrimidines in “acid puddles”.
Biochemical and biophysical research communications. 48(6):1622-9 [PMID] 5077841.
1972
Spontaneous and light induced hydration of pyrimidines.
Biochemical and biophysical research communications. 48(6):1614-21 [PMID] 5077840.
1971
Fluorescence of the purine and pyrimidine bases of the nucleic acids in neutral aqueous solution at 300 degrees K.
Science (New York, N.Y.). 171(3972):675-7 [PMID] 5540307.
1971
Fluorescence of thymine in aqueous solution at 300 degrees K.
Photochemistry and photobiology. 13(2):157-63 [PMID] 5552761.

Grants

Feb 2020 – Aug 2021
Preclinical Validation for ADVM-062 in Knock-Out and C198R Knock-In Missense Mouse Models of BCM
Role: Principal Investigator
Funding: ADVERUM BIOTECHNOLOGIES INC
Sep 2019 – Mar 2021
Disease mechanisms of cone opsin mutants and treatment strategies
Role: Co-Investigator
Funding: NATL INST OF HLTH NEI
Mar 2019 ACTIVE
Mito-Targeted AAV to Treat Leber Hereditary Optic Neuropathy Caused by ND4 Mutations
Role: Principal Investigator
Funding: UNIV OF MIAMI via NATL INST OF HLTH NEI
Jan 2019 – Dec 2019
Dual Vectors for Otoferlin
Role: Principal Investigator
Funding: DECIBEL THERAPEUTICS
Jun 2018 – Jun 2019
Gene Therapy for RHO adRP
Role: Faculty
Funding: IVERIC BIO
Apr 2018 – Jan 2022
Translational Gene Therapy for CNGB1 Retinitis Pigmentosa
Role: Principal Investigator
Funding: MICHIGAN STATE UNIV via NATL INST OF HLTH NEI
Oct 2017 – Mar 2019
Toxicology Study of BS01 in Rd1 mice
Role: Project Manager
Funding: APPLIED GENETICS TECH CORP
Sep 2016 – Aug 2021
Translational Research for Retinal Degenerative Therapies
Role: Principal Investigator
Funding: UNIV OF PENNSYLVANIA via NATL INST OF HLTH NEI
Aug 2016 – Jul 2022
Testing Gene Therapy in Models of Geographic Atrophy
Role: Project Manager
Funding: NATL INST OF HLTH NEI
Jul 2016 – Oct 2017
XLRP Mouse Toxicology Study
Role: Faculty
Funding: APPLIED GENETICS TECH CORP
Jan 2016 – Feb 2018
AAV-Mediated Therapy for Best Vitelliform Macular Dystrophy
Role: Principal Investigator
Funding: UNIV OF PENNSYLVANIA via FOU FOR FIGHTING BLINDNESS
Oct 2015 – Oct 2016
DSR Match Support – Gene Therapy for ABCA4 Stargar
Role: Principal Investigator
Funding: UF DIV OF SPONSORED RES MATCHING FUNDS
Sep 2015 – Sep 2018
blue cone monochromancy
Role: Principal Investigator
Funding: APPLIED GENETICS TECH CORP
Sep 2015 – Aug 2016
Translational Research for Retinal Degenerative Therapies
Role: Principal Investigator
Funding: UNIV OF PENNSYLVANIA via NATL INST OF HLTH NEI
May 2015 – Mar 2020
Gene Therapy for ABCA4 Stargardt's Disease
Role: Principal Investigator
Funding: APPLIED GENETICS TECH CORP
Jan 2015 – Jun 2018
Gene Therapy for Blue Cone Monochromacy
Role: Principal Investigator
Funding: BCM Families Foundation
Jun 2013 – Nov 2019
rAAV-CNGB3 Gene Therapy for Achromatopsia: Translational Research Studies
Role: Principal Investigator
Funding: NATL INST OF HLTH NEI
Feb 2013 – Jan 2018
Population Coding in the Retina
Role: Principal Investigator
Funding: CORNELL UNIV via NATL INST OF HLTH
Jan 2013 – Dec 2021
Vitreal delivery of novel AAV vectors for blue cone monochromacy gene therapy
Role: Principal Investigator
Funding: UF FOUNDATION
Nov 2012 – Dec 2016
XIAP gene therapy for the treatment of retinal degeneration: Safety and toxicity studies for translation into the clinic
Role: Principal Investigator
Funding: OTTAWA HOSP RESEARCH INST via FOU FOR FIGHTING BLINDNESS
Oct 2012 – Sep 2016
Gene therapy using novel cone-targeted adeno-associated vital vectors in the CNGA3 sheep model of achromatopsia
Role: Principal Investigator
Funding: US ISRAEL BINATIONAL SCI FOU
Aug 2012 – Jul 2018
Gene Therapy for CEP290 LCA and RPE Dystrophies
Role: Principal Investigator
Funding: FOU FOR FIGHTING BLINDNESS
Mar 2012 – Feb 2018
Translational Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa (Module 1)
Role: Principal Investigator
Funding: UNIV OF PENNSYLVANIA via NATL INST OF HLTH NEI
Oct 2011 – Jun 2016
Gene Therapy for LCA1
Role: Principal Investigator
Funding: FOU FOR FIGHTING BLINDNESS
Aug 2011 – Jul 2016
Vision Research Core
Role: Principal Investigator
Funding: NATL INST OF HLTH NEI
Jul 2011 – Jun 2016
Module 1: Cone Targeted Gene Therapy
Role: Principal Investigator
Funding: FOU FOR FIGHTING BLINDNESS
Jan 2011 – Jan 2021
Purified Recombinant Adeno Associated Virus
Role: Principal Investigator
Funding: MULTIPLE SPONSORS
Jan 2004 – May 2017
03091205
Role: Principal Investigator
Funding: MACULAR VISION RESEARCH FOU

Contact Details

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Business:
(352) 359-4301
Emails:
Addresses:
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PO Box 100284
GAINESVILLE FL 32610
Business Street:
R1254 ARB
GAINESVILLE FL 32611