Sanford L Boye

Sanford L Boye,

Associate Scientist, Director Of Vector Core

Department: MD-POWELL GENE THERAPY CENTER
Business Phone: (352) 273-6549
Business Email: sboye@ufl.edu

About Sanford L Boye

Sanford Boye is an Associate Scientist of Pediatrics at the University of Florida and Associate Director of the Powell Gene Therapy Center. He is the director of PGTC the Vector Core Laboratory, which specializes in the manufacture of AAV based gene therapy vectors. Sanford’s research focuses on 1) the design, construction and utilization of recombinant adeno-associated virus (AAV) vectors for gene transfer and 2) the evaluation of clinically relevant AAV vectors in animal models of human disease, with emphasis on retinal degenerative disorders. He was a member of the National Eye Institute- funded consortium who developed an AAV gene therapy for the RPE65 version of Leber Congenital Amaurosis (LCA2), designing the clinically relevant AAV vector. This led to several successful, first in human clinical trials of gene therapy for blindness and in part to the FDA’s first approval of a gene therapy for an inherited disease. He has designed AAV vectors that have been used to successfully address a variety of retinal disease models, including autosomal recessive retinitis pigmentosa, achromatopsia, X-linked juvenile retinoschisis, Bardet-Biedl syndrome, Usher syndrome and multiple forms of Leber congenital amaurosis (LCA). Recently his work has focused on modifications of the AAV capsid to improve vector performance and safety. Currently he splits his time between working with Dr. Shannon Boye on both basic science research and translational programs- gaining FDA approval for AAV based treatments for retinal disease and directing Vector Core within the Powell Gene Therapy Center.

Research Profile

Sanford Boye’s research focuses on 1) the design, construction and utilization of recombinant adeno-associated virus (AAV) vectors for gene transfer and 2) the evaluation of clinically relevant AAV vectors in animal models of human disease, with emphasis on retinal degenerative disorders. He was a member of the National Eye Institute- funded consortium who developed an AAV gene therapy for the RPE65 version of Leber Congenital Amaurosis (LCA2), designing the clinically relevant AAV vector. This led to several successful, first in human clinical trials of gene therapy for blindness and in part to the FDA’s first approval of a gene therapy for an inherited disease. He has designed AAV vectors that have been used to successfully address a variety of retinal disease models, including autosomal recessive retinitis pigmentosa, achromatopsia, X-linked juvenile retinoschisis, Bardet-Biedl syndrome, Usher syndrome and multiple forms of Leber congenital amaurosis (LCA). Recently his work has focused on modifications of the AAV capsid to vector performance and safety. Currently he splits his time between working with Dr. Shannon Boye on both basic science research and translational programs- gaining FDA approval for AAV based treatments for retinal disease and directing Vector Core within the Powell Gene Therapy Center.

Publications

2021
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Molecular therapy : the journal of the American Society of Gene Therapy. 29(2):464-488 [DOI] 10.1016/j.ymthe.2020.12.007. [PMID] 33309881.
2021
Effects of Altering HSPG Binding and Capsid Hydrophilicity on Retinal Transduction by AAV.
Journal of virology. [DOI] 10.1128/JVI.02440-20. [PMID] 33658343.
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. [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
Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations.
iScience. 24(5) [DOI] 10.1016/j.isci.2021.102409. [PMID] 33997691.
2021
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse.
Gene therapy. 28(7-8):447-455 [DOI] 10.1038/s41434-020-00206-w. [PMID] 33244179.
2020
Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking.
Viruses. 12(6) [DOI] 10.3390/v12060668. [PMID] 32575696.
2020
Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy.
Molecular therapy : the journal of the American Society of Gene Therapy. 28(6):1464-1478 [DOI] 10.1016/j.ymthe.2020.04.002. [PMID] 32304666.
2020
SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration.
eLife. 9 [DOI] 10.7554/eLife.62027. [PMID] 33107823.
2020
Utilizing minimally purified secreted rAAV for rapid and cost-effective manipulation of gene expression in the CNS.
Molecular neurodegeneration. 15(1) [DOI] 10.1186/s13024-020-00361-z. [PMID] 32122372.
2019
A Novel Mouse Model of MYO7A USH1B Reveals Auditory and Visual System Haploinsufficiencies.
Frontiers in neuroscience. 13 [DOI] 10.3389/fnins.2019.01255. [PMID] 31824252.
2019
Impaired ABCA1/ABCG1-mediated lipid efflux in the mouse retinal pigment epithelium (RPE) leads to retinal degeneration.
eLife. 8 [DOI] 10.7554/eLife.45100. [PMID] 30864945.
2019
Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque.
Human gene therapy. 30(5):571-589 [DOI] 10.1089/hum.2018.193. [PMID] 30358434.
2019
SubILM Injection of AAV for Gene Delivery to the Retina.
Methods in molecular biology (Clifton, N.J.). 1950:249-262 [DOI] 10.1007/978-1-4939-9139-6_14. [PMID] 30783978.
2018
A Drug-Tunable Gene Therapy for Broad-Spectrum Protection against Retinal Degeneration.
Molecular therapy : the journal of the American Society of Gene Therapy. 26(10):2407-2417 [DOI] 10.1016/j.ymthe.2018.07.016. [PMID] 30078764.
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
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
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.
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 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
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
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.
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
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
Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas.
Molecular vision. 22:1342-1351 [PMID] 27881908.
View on: PubMed
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
Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors.
Journal of virology. 90(8):4215-4231 [DOI] 10.1128/JVI.00200-16. [PMID] 26865709.
2016
NF1 Is a Direct G Protein Effector Essential for Opioid Signaling to Ras in the Striatum.
Current biology : CB. 26(22):2992-3003 [DOI] 10.1016/j.cub.2016.09.010. [PMID] 27773571.
2016
Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells.
Frontiers in neuroscience. 10 [PMID] 27990105.
View on: PubMed
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
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.
View on: PubMed
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
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
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 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
Safety and Biodistribution Evaluation of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin, in RS1-Deficient Mice.
Human gene therapy. Clinical development. 26(3):177-84 [DOI] 10.1089/humc.2015.077. [PMID] 26390091.
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.
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
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
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
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
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
Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors.
Molecular therapy. Methods & clinical development. 1 [PMID] 24761428.
View on: PubMed
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
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
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.
View on: PubMed
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
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
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
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
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
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.
View on: PubMed
2012
Progeny of Pronuclear Injections of Mutant Human Mitochondrial Genes
Molecular Therapy. 20
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
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
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 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
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
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.
View on: PubMed
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
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
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
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
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
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.
View on: PubMed
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 Prevents Cone Degeneration in Two Models of Rpe65 Leber Congenital Amaurosis: Rd12 and Rpe65(-/-):: Rho(-/-) Mice
Molecular Therapy. 17:S288-S289
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
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.
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
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
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
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.
View on: PubMed
2007
Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virus.
Genetic vaccines and therapy. 5 [PMID] 18070352.
View on: PubMed
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.
View on: PubMed
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.
View on: PubMed
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.
View on: PubMed
2004
Differential contribution of active site residues in substrate recognition sites 1 and 5 to cytochrome P450 2C8 substrate selectivity and regioselectivity.
Biochemistry. 43(24):7834-42 [PMID] 15196026.
View on: PubMed
2004
Optimizing bacterial expression of catalytically active human cytochromes P450: comparison of CYP2C8 and CYP2C9.
Xenobiotica; the fate of foreign compounds in biological systems. 34(1):49-60 [PMID] 14742136.
View on: PubMed
2000
Nicotinic receptors modulating somatodendritic and terminal dopamine release differ pharmacologically.
European journal of pharmacology. 393(1-3):39-49 [PMID] 10770996.
View on: PubMed

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