Nicholas Muzyczka

Nicholas Muzyczka,

Emeritus Scholar

Department: Molecular Genetics & Microbiology
Business Phone: (352) 273-8114
Business Email: nmuzyczk@ufl.edu

Publications

2023
Structural and Biophysical Analysis of Adeno-Associated Virus Serotype 2 Capsid Assembly Variants.
Journal of virology. 97(7) [DOI] 10.1128/jvi.01772-22. [PMID] 37310260.
2019
Biodistribution of adeno-associated virus type 2 with mutations in the capsid that contribute to heparan sulfate proteoglycan binding.
Virus research. 274 [DOI] 10.1016/j.virusres.2019.197771. [PMID] 31577935.
2018
The Lipase Activity of Phospholipase D2 is Responsible for Nigral Neurodegeneration in a Rat Model of Parkinson’s Disease.
Neuroscience. 377:174-183 [DOI] 10.1016/j.neuroscience.2018.02.047. [PMID] 29526688.
2017
AAV: An Overview of Unanswered Questions.
Human gene therapy. 28(4):308-313 [DOI] 10.1089/hum.2017.048. [PMID] 28335618.
2016
Cryo-electron Microscopy Reconstruction and Stability Studies of the Wild Type and the R432A Variant of Adeno-associated Virus Type 2 Reveal that Capsid Structural Stability Is a Major Factor in Genome Packaging.
Journal of virology. 90(19):8542-51 [DOI] 10.1128/JVI.00575-16. [PMID] 27440903.
2016
Mutants at the 2-Fold Interface of Adeno-associated Virus Type 2 (AAV2) Structural Proteins Suggest a Role in Viral Transcription for AAV Capsids.
Journal of virology. 90(16):7196-7204 [DOI] 10.1128/JVI.00493-16. [PMID] 27252527.
2015
AAV’s Golden Jubilee.
Molecular therapy : the journal of the American Society of Gene Therapy. 23(5):807-808 [DOI] 10.1038/mt.2015.55. [PMID] 25943495.
2015
Adeno-associated virus serotype 1 (AAV1)- and AAV5-antibody complex structures reveal evolutionary commonalities in parvovirus antigenic reactivity.
Journal of virology. 89(3):1794-808 [DOI] 10.1128/JVI.02710-14. [PMID] 25410874.
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
Enhanced Transgene Expression From Recombinant Single-Stranded D-Sequence-Substituted Adeno-Associated Virus Vectors in Human Cell Lines in Vitro and in Murine Hepatocytes in Vivo
Journal Of Virology. 89(2):952-961 [DOI] 10.1128/JVI02581-14.
2015
Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo.
Journal of virology. 89(2):952-61 [DOI] 10.1128/JVI.02581-14. [PMID] 25355884.
2015
The Adeno-Associated Virus Genome Packaging Puzzle.
Journal of molecular and genetic medicine : an international journal of biomedical research. 9(3) [PMID] 26949410.
2015
Transcytosis in the blood-cerebrospinal fluid barrier of the mouse brain with an engineered receptor/ligand system.
Molecular therapy. Methods & clinical development. 2 [DOI] 10.1038/mtm.2015.37. [PMID] 26491705.
2014
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.
Annual review of virology. 1(1):427-51 [DOI] 10.1146/annurev-virology-031413-085355. [PMID] 26958729.
2014
Adeno-associated virus capsid proteins may play a role in transcription and second-strand synthesis of recombinant genomes.
Journal of virology. 88(2):1071-9 [DOI] 10.1128/JVI.02093-13. [PMID] 24198419.
2014
OneBac: platform for scalable and high-titer production of adeno-associated virus serotype 1-12 vectors for gene therapy.
Human gene therapy. 25(3):212-22 [DOI] 10.1089/hum.2013.184. [PMID] 24299301.
2014
Overcoming the cystic fibrosis sputum barrier to leading adeno-associated virus gene therapy vectors.
Molecular therapy : the journal of the American Society of Gene Therapy. 22(8):1484-1493 [DOI] 10.1038/mt.2014.89. [PMID] 24869933.
2013
Capsid antibodies to different adeno-associated virus serotypes bind common regions.
Journal of virology. 87(16):9111-24 [DOI] 10.1128/JVI.00622-13. [PMID] 23760240.
2013
Construction, expression, and purification of recombinant αVβ5 integrin.
Protein expression and purification. 89(2):225-31 [DOI] 10.1016/j.pep.2013.04.002. [PMID] 23583935.
2013
Molecular cloning, overexpression, and an efficient one-step purification of α5β1 integrin.
Protein expression and purification. 92(1):21-8 [DOI] 10.1016/j.pep.2013.08.013. [PMID] 23993979.
2013
Structural insights into adeno-associated virus serotype 5.
Journal of virology. 87(20):11187-99 [DOI] 10.1128/JVI.00867-13. [PMID] 23926356.
2013
Structure and dynamics of adeno-associated virus serotype 1 VP1-unique N-terminal domain and its role in capsid trafficking.
Journal of virology. 87(9):4974-84 [DOI] 10.1128/JVI.02524-12. [PMID] 23427155.
2012
Enhanced expression of Pctk1, Tcf12 and Ccnd1 in hippocampus of rats: Impact on cognitive function, synaptic plasticity and pathology.
Neurobiology of learning and memory. 97(1):69-80 [DOI] 10.1016/j.nlm.2011.09.006. [PMID] 21982980.
2012
Evidence for pH-dependent protease activity in the adeno-associated virus capsid.
Journal of virology. 86(21):11877-85 [DOI] 10.1128/JVI.01717-12. [PMID] 22915820.
2012
Glucose regulated protein 78 diminishes α-synuclein neurotoxicity in a rat model of Parkinson disease.
Molecular therapy : the journal of the American Society of Gene Therapy. 20(7):1327-37 [DOI] 10.1038/mt.2012.28. [PMID] 22434142.
2012
Structural insight into the unique properties of adeno-associated virus serotype 9.
Journal of virology. 86(12):6947-58 [DOI] 10.1128/JVI.07232-11. [PMID] 22496238.
2012
The 3D Structure of Adeno-Associated Virus Serotype 9 and Its Unique Properties
Molecular Therapy. 20
2011
Structural studies of adeno-associated virus serotype 8 capsid transitions associated with endosomal trafficking.
Journal of virology. 85(22):11791-9 [DOI] 10.1128/JVI.05305-11. [PMID] 21900159.
2010
Adeno-Associated Virus-Mediated Knockdown of Pyruvate Dehydrogenase E1 Alpha in Specific Regions of the Rat Brain
Mitochondrion. 10 [DOI] 10.1016/j.mito.2009.12.01.
2010
An animal model of PDH deficiency using AAV8-siRNA vector-mediated knockdown of pyruvate dehydrogenase E1α.
Molecular genetics and metabolism. 101(2-3):183-91 [DOI] 10.1016/j.ymgme.2010.07.008. [PMID] 20685142.
2010
Efficacy of recombinant adeno-associated viral vectors serotypes 1, 2, and 5 for the transduction of pancreatic and colon carcinoma cells.
Anticancer research. 30(6):1931-5 [PMID] 20651336.
2010
In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration.
Molecular therapy : the journal of the American Society of Gene Therapy. 18(8):1450-7 [DOI] 10.1038/mt.2010.115. [PMID] 20551914.
2010
Myosin IIb regulates actin dynamics during synaptic plasticity and memory formation.
Neuron. 67(4):603-17 [DOI] 10.1016/j.neuron.2010.07.016. [PMID] 20797537.
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
The effect of DNA-dependent protein kinase on adeno-associated virus replication.
PloS one. 5(12) [DOI] 10.1371/journal.pone.0015073. [PMID] 21188139.
2010
α-Synuclein expression in rat substantia nigra suppresses phospholipase D2 toxicity and nigral neurodegeneration.
Molecular therapy : the journal of the American Society of Gene Therapy. 18(10):1758-68 [DOI] 10.1038/mt.2010.137. [PMID] 20664530.
2009
Absolute Control of Raav Mediated Gdnf Expression in the Nigrostriatal Tract Using a Single Vector Tetracycline Responsive Expression System
Molecular Therapy. 17
2009
Adeno-Associated Virus-Mediated Knockdown of Pyruvate Dehydrogenase E1F(N)Over-Tilde At in in Specific Regions of the Rat Brain
Molecular Genetics and Metabolism. 98
2009
Bilateral Nigral Raav5-Mediated Gdnf Over-Expression Activates Crh Neurons in the Paraventricular Nucleus of the Hypothalamus and Induces Robust Weight Loss in Obese Leptin-Resistant Rats
Molecular Therapy. 17
2009
Examining Conserved Residues in the Aav2 Capsid, Linking Critical Regions On the Surface and in the Interior
Molecular Therapy. 17
2009
Heparin binding induces conformational changes in Adeno-associated virus serotype 2.
Journal of structural biology. 165(3):146-56 [DOI] 10.1016/j.jsb.2008.12.002. [PMID] 19121398.
2009
Identification of cellular proteins that interact with the adeno-associated virus rep protein.
Journal of virology. 83(1):454-69 [DOI] 10.1128/JVI.01939-08. [PMID] 18971280.
2009
Nigrostriatal rAAV-mediated GDNF overexpression induces robust weight loss in a rat model of age-related obesity.
Molecular therapy : the journal of the American Society of Gene Therapy. 17(6):980-91 [DOI] 10.1038/mt.2009.45. [PMID] 19277011.
2009
Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 9.
Acta crystallographica. Section F, Structural biology and crystallization communications. 65(Pt 7):715-8 [DOI] 10.1107/S1744309109021460. [PMID] 19574648.
2009
Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented.
Molecular therapy : the journal of the American Society of Gene Therapy. 17(3):524-37 [DOI] 10.1038/mt.2008.284. [PMID] 19142181.
2009
Structural Studies of Adeno-Associated Virus Serotypes Towards Improved Gene Therapy Applications
Molecular Therapy. 17
2009
Tight Durable Long-Term Doxycycline-Mediated Gdnf Regulation From a Single Raav Vector in the Nigrostiatal Tract
Cell Transplantation. 18:224-225
2009
Tight Long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector.
Molecular therapy : the journal of the American Society of Gene Therapy. 17(11):1857-67 [DOI] 10.1038/mt.2009.196. [PMID] 19707186.
2009
Towards Characterizing Vector-Host Interactions: Adeno-Associated Virus Capsids in Complex With Antibody Fragments
Molecular Therapy. 17
2009
Weight Loss in Obese Leptin-Resistant Rats Induced By Activation of Crh Neurons in the Paraventricular Nucleus of the Hypothalamus By Overexpression of Substantial Nigral-Injected Raav5-Gdnf
Cell Transplantation. 18
2008
Adeno-associated Viral (AAV) Serotype 5 Vector Mediated Gene Delivery of Endothelin-converting Enzyme Reduces Aβ Deposits in APP + PS1 Transgenic Mice.
Molecular therapy : the journal of the American Society of Gene Therapy. 16(9):1580-1586 [DOI] 10.1038/mt.2008.148. [PMID] 28189012.
2008
Complete in vitro reconstitution of adeno-associated virus DNA replication requires the minichromosome maintenance complex proteins.
Journal of virology. 82(3):1458-64 [PMID] 18057257.
2008
Genome-wide analysis of aging and learning-related genes in the hippocampal dentate gyrus
Neurobiology of Learning and Memory. 89(4):379-396
2008
Monocyte Gene Therapy in Alzheimer’S Disease App+Ps1 Transgenic Mice
Cell Transplantation. 17:470-471
2008
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses.
Proceedings of the National Academy of Sciences of the United States of America. 105(22):7827-32 [DOI] 10.1073/pnas.0802866105. [PMID] 18511559.
2008
Pres 30-Locating the Vp1 Structural Protein in Adeno-Associated Viruses
. 235
2008
Pres 33-Ph Induced Conformational Changes On the Surface of the Aav1 Capsid: Implications for Capsid Trafficking and Uncoating
. 235
2008
The phosphorylation state of Ser-129 in human alpha-synuclein determines neurodegeneration in a rat model of Parkinson disease.
Proceedings of the National Academy of Sciences of the United States of America. 105(2):763-8 [DOI] 10.1073/pnas.0711053105. [PMID] 18178617.
2008
Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression.
Virology. 381(2):194-202 [DOI] 10.1016/j.virol.2008.08.027. [PMID] 18834608.
2007
Changes in transcription within the CA1 field of the hippocampus are associated with age-related spatial learning impairments.
Neurobiology of learning and memory. 87(1):21-41 [PMID] 16829144.
2007
Long-term consequences of human alpha-synuclein overexpression in the primate ventral midbrain.
Brain : a journal of neurology. 130(Pt 3):799-815 [PMID] 17303591.
2007
Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 7.
Acta crystallographica. Section F, Structural biology and crystallization communications. 63(Pt 12):1073-6 [PMID] 18084098.
2007
Purification of host cell enzymes involved in adeno-associated virus DNA replication.
Journal of virology. 81(11):5777-87 [PMID] 17360744.
2007
rAAV-mediated nigral human parkin over-expression partially ameliorates motor deficits via enhanced dopamine neurotransmission in a rat model of Parkinson’s disease.
Experimental neurology. 207(2):289-301 [PMID] 17678648.
2007
Structure of adeno-associated virus serotype 8, a gene therapy vector.
Journal of virology. 81(22):12260-71 [PMID] 17728238.
2007
Time course of transgene expression after intrastriatal pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 transduction in the rat.
Molecular therapy : the journal of the American Society of Gene Therapy. 15(8):1504-11 [PMID] 17565350.
2006
Hypothalamic rAAV-mediated GDNF gene delivery ameliorates age-related obesity.
Neurobiology of aging. 27(3):459-70 [PMID] 15964099.
2006
Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.
Human gene therapy. 17(12):1177-86 [PMID] 17115945.
2006
Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 1.
Acta crystallographica. Section F, Structural biology and crystallization communications. 62(Pt 12):1271-4 [PMID] 17142915.
2006
Recombinant adeno-associated and adenoviral vectors for the transduction of pancreatic and colon carcinoma.
Anticancer research. 26(1A):311-7 [PMID] 16475712.
2006
Structurally mapping the diverse phenotype of adeno-associated virus serotype 4.
Journal of virology. 80(23):11556-70 [PMID] 16971437.
2005
Altering AAV tropism with mosaic viral capsids.
Molecular therapy : the journal of the American Society of Gene Therapy. 11(6):856-65 [PMID] 15922956.
2005
Continuous low-level glial cell line-derived neurotrophic factor delivery using recombinant adeno-associated viral vectors provides neuroprotection and induces behavioral recovery in a primate model of Parkinson’s disease.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 25(4):769-77 [PMID] 15673656.
2005
Custom adeno-associated virus capsids: the next generation of recombinant vectors with novel tropism.
Human gene therapy. 16(4):408-16 [PMID] 15871672.
2005
Production, purification, crystallization and preliminary X-ray analysis of adeno-associated virus serotype 8.
Acta crystallographica. Section F, Structural biology and crystallization communications. 61(Pt 6):558-61 [PMID] 16511095.
2005
Production, purification, crystallization and preliminary X-ray structural studies of adeno-associated virus serotype 5.
Acta crystallographica. Section F, Structural biology and crystallization communications. 61(Pt 10):917-21 [PMID] 16511195.
2005
Reversal of dyskinesias in an animal model of Parkinson’s disease by continuous L-DOPA delivery using rAAV vectors.
Brain : a journal of neurology. 128(Pt 3):559-69 [PMID] 15659429.
2005
Structure of adeno-associated virus type 4.
Journal of virology. 79(8):5047-58 [PMID] 15795290.
2005
Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system.
Molecular therapy : the journal of the American Society of Gene Therapy. 12(6):1217-25 [PMID] 16213797.
2004
Adeno-associated virus Rep78/Rep68 promotes localized melting of the rep binding element in the absence of adenosine triphosphate.
Chembiochem : a European journal of chemical biology. 5(3):324-32 [PMID] 14997524.
2004
Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus.
Journal of virology. 78(12):6595-609 [PMID] 15163751.
2004
Circulating anti-wild-type adeno-associated virus type 2 (AAV2) antibodies inhibit recombinant AAV2 (rAAV2)-mediated, but not rAAV5-mediated, gene transfer in the brain.
Journal of virology. 78(12):6344-59 [PMID] 15163728.
2004
Differential modulation of energy balance by leptin, ciliary neurotrophic factor, and leukemia inhibitory factor gene delivery: microarray deoxyribonucleic acid-chip analysis of gene expression.
Endocrinology. 145(4):2035-45 [PMID] 14715713.
2004
Patterns of gene expression reveal a temporally orchestrated wound healing response in the injured spinal cord.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 24(39):8562-76 [PMID] 15456830.
2004
Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system.
Molecular therapy : the journal of the American Society of Gene Therapy. 10(2):302-17 [PMID] 15294177.
2003
Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease.
Molecular therapy : the journal of the American Society of Gene Therapy. 7(5 Pt 1):580-7 [PMID] 12718900.
2003
Characterization of the adenoassociated virus Rep protein complex formed on the viral origin of DNA replication.
Virology. 313(2):364-76 [PMID] 12954205.
2003
Gene therapy delivery of endostatin enhances the treatment efficacy of radiation.
Radiotherapy and oncology : journal of the European Society for Therapeutic Radiology and Oncology. 66(1):1-9 [PMID] 12559515.
2003
Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding.
Journal of virology. 77(12):6995-7006 [PMID] 12768018.
2003
Nigrostriatal alpha-synucleinopathy induced by viral vector-mediated overexpression of human alpha-synuclein: a new primate model of Parkinson’s disease.
Proceedings of the National Academy of Sciences of the United States of America. 100(5):2884-9 [PMID] 12601150.
2003
Recombinant adeno-associated viral vector (rAAV) delivery of GDNF provides protection against 6-OHDA lesion in the common marmoset monkey (Callithrix jacchus).
Experimental neurology. 184(1):536-48 [PMID] 14637123.
2003
Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver.
Gene therapy. 10(18):1551-8 [PMID] 12907946.
2002
Adeno-associated virus-mediated gene transfer of endostatin inhibits angiogenesis and tumor growth in vivo.
Cancer gene therapy. 9(6):513-21 [PMID] 12032662.
2002
Comparison of the EF-1 alpha and the CMV promoter for engineering stable tumor cell lines using recombinant adeno-associated virus.
Anticancer research. 22(6A):3325-30 [PMID] 12530082.
2002
Neuropathological and behavioral consequences of adeno-associated viral vector-mediated continuous intrastriatal neurotrophin delivery in a focal ischemia model in rats.
Neurobiology of disease. 9(2):187-204 [PMID] 11895371.
2002
Parkinson-like neurodegeneration induced by targeted overexpression of alpha-synuclein in the nigrostriatal system.
The Journal of neuroscience : the official journal of the Society for Neuroscience. 22(7):2780-91 [PMID] 11923443.
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
Reversal of motor impairments in parkinsonian rats by continuous intrastriatal delivery of L-dopa using rAAV-mediated gene transfer.
Proceedings of the National Academy of Sciences of the United States of America. 99(7):4708-13 [PMID] 11917105.
2002
Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors.
Methods in enzymology. 346:413-30 [PMID] 11883083.
2002
Studies of the mechanism of transactivation of the adeno-associated virus p19 promoter by Rep protein.
Journal of virology. 76(16):8225-35 [PMID] 12134028.
2000
Adeno-associated virus vector-mediated gene transfer to somatic cells in the central nervous system.
Advances in virus research. 55:507-28 [PMID] 11050954.
2000
Cre-mediated recombination in the pituitary gland.
Genesis (New York, N.Y. : 2000). 28(3-4):167-74 [PMID] 11105060.
2000
Long-term differential modulation of genes encoding orexigenic and anorexigenic peptides by leptin delivered by rAAV vector in ob/ob mice. Relationship with body weight change.
Regulatory peptides. 92(1-3):97-105 [PMID] 11024571.
2000
Mechanism of Rep-mediated adeno-associated virus origin nicking.
Journal of virology. 74(17):7762-71 [PMID] 10933682.
2000
Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism.
Journal of virology. 74(18):8635-47 [PMID] 10954565.
2000
NGF gene transfer to intrinsic basal forebrain neurons increases cholinergic cell size and protects from age-related, spatial memory deficits in middle-aged rats.
Brain research. 875(1-2):144-51 [PMID] 10967308.
2000
Production and purification of recombinant adeno-associated virus.
Methods in enzymology. 316:743-61 [PMID] 10800712.
1999
Biochemical characterization of adeno-associated virus rep68 DNA helicase and ATPase activities.
Journal of virology. 73(2):1580-90 [PMID] 9882364.
1999
Charge-to-alanine mutagenesis of the adeno-associated virus type 2 Rep78/68 proteins yields temperature-sensitive and magnesium-dependent variants.
Journal of virology. 73(11):9433-45 [PMID] 10516052.
1999
Generation of aberrant sprouting in the adult rat brain by GAP-43 somatic gene transfer.
Brain research. 832(1-2):136-44 [PMID] 10375659.
1999
High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap.
Gene therapy. 6(6):986-93 [PMID] 10455400.
1999
Long-term actions of vector-derived nerve growth factor or brain-derived neurotrophic factor on choline acetyltransferase and Trk receptor levels in the adult rat basal forebrain.
Neuroscience. 90(3):815-21 [PMID] 10218782.
1999
Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.
Human gene therapy. 10(1):85-94 [PMID] 10022533.
1999
Prevention of 6-hydroxydopamine-induced rotational behavior by BDNF somatic gene transfer.
Brain research. 847(2):314-20 [PMID] 10575102.
1999
Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield.
Gene therapy. 6(6):973-85 [PMID] 10455399.
1999
Rep-mediated nicking of the adeno-associated virus origin requires two biochemical activities, DNA helicase activity and transesterification.
Journal of virology. 73(11):9325-36 [PMID] 10516041.
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
Cellular proteins required for adeno-associated virus DNA replication in the absence of adenovirus coinfection.
Journal of virology. 72(4):2777-87 [PMID] 9525597.
1998
Developing VDEPT for DT-diaphorase (NQO1) using an AAV vector plasmid.
International journal of radiation oncology, biology, physics. 42(4):909-12 [PMID] 9845120.
1998
Expression of adeno-associated virus integrated transgene within the mammalian vestibular organs.
The American journal of otology. 19(3):390-5 [PMID] 9596192.
1998
In vitro packaging of adeno-associated virus DNA.
Journal of virology. 72(4):3241-7 [PMID] 9525651.
1998
Long-term in vivo cochlear transgene expression mediated by recombinant adeno-associated virus.
Gene therapy. 5(2):277-81 [PMID] 9578849.
1998
Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors.
Experimental neurology. 150(2):183-94 [PMID] 9527887.
1998
Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors.
Proceedings of the National Academy of Sciences of the United States of America. 95(24):14384-8 [PMID] 9826709.
1997
Characterization of recombinant adeno-associated virus-2 as a vehicle for gene delivery and expression into vascular cells.
Journal of investigative medicine : the official publication of the American Federation for Clinical Research. 45(2):87-98 [PMID] 9084579.
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.
1997
Efficient transduction of green fluorescent protein in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters.
Gene therapy. 4(1):16-24 [PMID] 9068791.
1997
Gene therapy for murine mucopolysaccharidosis type VII.
Neuromuscular disorders : NMD. 7(5):352-60 [PMID] 9267850.
1997
Green fluorescent protein as a reporter for gene transfer studies in the cochlea.
Hearing research. 114(1-2):139-47 [PMID] 9447928.
1997
Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex virus type 1 amplicon expressing Rep and Cap.
Journal of virology. 71(11):8780-9 [PMID] 9343238.
1997
The adeno-associated virus (AAV) Rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection.
Journal of virology. 71(2):1079-88 [PMID] 8995628.
1997
The adeno-associated virus type 2 p40 promoter requires a proximal Sp1 interaction and a p19 CArG-like element to facilitate Rep transactivation.
Journal of virology. 71(6):4300-9 [PMID] 9151818.
1997
The cellular transcription factor SP1 and an unknown cellular protein are required to mediate Rep protein activation of the adeno-associated virus p19 promoter.
Journal of virology. 71(3):1747-56 [PMID] 9032303.
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
Development of in vivo gene therapy for hearing disorders: introduction of adeno-associated virus into the cochlea of the guinea pig.
Gene therapy. 3(7):588-92 [PMID] 8818645.
1996
Sequence requirements for binding of Rep68 to the adeno-associated virus terminal repeats.
Journal of virology. 70(3):1542-53 [PMID] 8627673.
1995
Regulated high-level human beta-globin gene expression in erythroid cells following recombinant adeno-associated virus-mediated gene transfer.
Gene therapy. 2(5):336-43 [PMID] 7671109.
1994
Adeno-associated virus (AAV) vectors: will they work?
The Journal of clinical investigation. 94(4) [PMID] 7929808.
1994
Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein.
Journal of virology. 68(8):4988-97 [PMID] 8035498.
1994
In vitro replication of adeno-associated virus DNA.
Journal of virology. 68(2):1128-38 [PMID] 8289342.
1994
Interaction of the adeno-associated virus Rep protein with a sequence within the A palindrome of the viral terminal repeat.
Journal of virology. 68(8):4998-5006 [PMID] 8035499.
1993
Features of the adeno-associated virus origin involved in substrate recognition by the viral Rep protein.
Journal of virology. 67(10):6096-104 [PMID] 8396670.
1992
Analysis of mutations in adeno-associated virus Rep protein in vivo and in vitro.
Journal of virology. 66(7):4050-7 [PMID] 1318396.
1992
Partial purification of adeno-associated virus Rep78, Rep52, and Rep40 and their biochemical characterization.
Journal of virology. 66(2):1119-28 [PMID] 1309894.
1992
Use of adeno-associated virus as a general transduction vector for mammalian cells.
Current topics in microbiology and immunology. 158:97-129 [PMID] 1316261.
1991
Sequences required for coordinate induction of adeno-associated virus p19 and p40 promoters by Rep protein.
Journal of virology. 65(6):2936-45 [PMID] 2033660.
1990
Evidence for covalent attachment of the adeno-associated virus (AAV) rep protein to the ends of the AAV genome.
Journal of virology. 64(12):6204-13 [PMID] 2173787.
1990
In vitro resolution of covalently joined AAV chromosome ends.
Cell. 60(1):105-13 [PMID] 2153052.
1990
Purification and characterization of HeLa endonuclease R. A G-specific mammalian endonuclease.
The Journal of biological chemistry. 265(19):10836-41 [PMID] 2358441.
1990
Site-specific integration by adeno-associated virus.
Proceedings of the National Academy of Sciences of the United States of America. 87(6):2211-5 [PMID] 2156265.
1990
Substrate specificity of HeLa endonuclease R. A G-specific mammalian endonuclease.
The Journal of biological chemistry. 265(19):10842-50 [PMID] 2358442.
1990
The AAV origin binding protein Rep68 is an ATP-dependent site-specific endonuclease with DNA helicase activity.
Cell. 61(3):447-57 [PMID] 2159383.
1989
Factors that bind to adeno-associated virus terminal repeats.
Journal of virology. 63(7):3095-104 [PMID] 2542617.
1988
Adeno-associated virus general transduction vectors: analysis of proviral structures.
Journal of virology. 62(6):1963-73 [PMID] 2835501.
1988
In vitro excision of adeno-associated virus DNA from recombinant plasmids: isolation of an enzyme fraction from HeLa cells that cleaves DNA at poly(G) sequences.
Molecular and cellular biology. 8(6):2513-22 [PMID] 2841582.
1985
Comparison of the DNA sequence and secondary structure of the herpes simplex virus L/S junction and the adeno-associated virus terminal repeat.
Journal of theoretical biology. 115(4):477-94 [PMID] 2995732.
1984
Genetics of adeno-associated virus: isolation and preliminary characterization of adeno-associated virus type 2 mutants.
Journal of virology. 51(2):329-39 [PMID] 6086948.
1984
The genetics of adeno-associated virus.
Advances in experimental medicine and biology. 179:151-61 [PMID] 6098150.
1984
Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.
Proceedings of the National Academy of Sciences of the United States of America. 81(20):6466-70 [PMID] 6093102.
1983
Characterization of am404, an amber mutation in the simian virus 40 T antigen gene.
Journal of virology. 47(1):202-16 [PMID] 6191041.
1983
Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV.
Cell. 33(1):135-43 [PMID] 6088052.
1982
Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells.
Proceedings of the National Academy of Sciences of the United States of America. 79(6):2077-81 [PMID] 6281795.
1980
Construction of a specific amber codon in the simian virus 40 T-antigen gene by site-directed mutagenesis.
Journal of virology. 36(2):611-6 [PMID] 6253682.
1980
Construction of an SV40-derived cloning vector.
Gene. 11(1-2):63-77 [PMID] 6254855.
1979
Persistence of phage lambda DNA in genomes of mouse cells transformed by lambda-carrying SV40 vectors.
Gene. 6(2):107-22 [PMID] 225241.
1974
Studies on the biochemical basis of spontaneous mutation. II. The incorporation of a base and its analogue into DNA by wild-type, mutator and antimutator DNA polymerases.
Journal of molecular biology. 88(2):409-21 [PMID] 4616089.
1972
Studies on the biochemical basis of spontaneous mutation. I. A comparison of the deoxyribonucleic acid polymerases of mutator, antimutator, and wild type strains of bacteriophage T4.
The Journal of biological chemistry. 247(22):7116-22 [PMID] 4565077.

Grants

Nov 2015 – Feb 2017
A NEW METHOD FOR DELIVERING TEMOZOLOMIDE ACROSS THE BRAINBARRIERS FOR GLIOBLASTOMA
Role: Principal Investigator
Funding: PARDEE FOU, ELSA U
Sep 2014 – Aug 2019
The role of pH and protease activity in AAV viral transduction
Role: Co-Investigator
Funding: NATL INST OF HLTH NIGMS

Contact Details

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