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Sanford L. Boye · SciRah

Researcher profile · Semantic Scholar

Sanford L. Boye

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Sanford L. Boye — researcher profile on SciRah

h-index: 53
Works: 252
Citations: 11K

Most-cited papers

Current Clinical Applications of In Vivo Gene Therapy with AAVs
J. Mendell, S. Al-Zaidy, L. Rodino-Klapac, Kimberly Goodspeed +9 more
2020Molecular Therapy615 citations Open access
Semantic Scholar
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial
N. Ghazi, E. Abboud, S. Nowilaty, H. Alkuraya +19 more
2016Human Genetics233 citations
Semantic Scholar
Current Clinical Applications of AAV-mediated Gene Therapy
B. Byrne, K. Flanigan, S. Matesanz, R. Finkel +24 more
2025Molecular Therapy111 citations Open access
Semantic Scholar
Complement C3-targeted gene therapy restricts onset and progression of neurodegeneration in chronic mouse glaucoma
Alejandra Bosco, Sarah R. Anderson, Kevin T. Breen, Cesar O. Romero +6 more
2018bioRxiv102 citations Open access
Semantic Scholar
Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease
William A. Beltran, A. Cideciyan, S. Iwabe, M. Świder +11 more
2015Proceedings of the National Academy of Sciences of the United States of America93 citations Open access
Semantic Scholar
Impaired ABCA1/ABCG1-mediated lipid efflux in the mouse retinal pigment epithelium (RPE) leads to retinal degeneration
F. Storti, Katrin Klee, V. Todorova, Regula Steiner +18 more
2019eLife90 citations Open access
Semantic Scholar
Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases
G. Ye, E. Budzynski, P. Sonnentag, T. Nork +7 more
2016Human Gene Therapy81 citations Open access
Semantic Scholar
Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina
Shannon E. Boye, J. Alexander, C. Witherspoon, Sanford L. Boye +6 more
2016Human Gene Therapy79 citations Open access
Semantic Scholar
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
William A. Beltran, A. Cideciyan, Shannon E. Boye, G. Ye +17 more
2017Molecular Therapy78 citations Open access
Semantic Scholar
Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque
K. T. Mccullough, Sanford L. Boye, Diego S. Fajardo, Kaitlyn R. Calabro +11 more
2019Human Gene Therapy73 citations Open access
Semantic Scholar
Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors
Sanford L. Boye, A. Bennett, Miranda L. Scalabrino, K. T. Mccullough +6 more
2016Journal of Virology69 citations Open access
Semantic Scholar
Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.
Miranda L. Scalabrino, Sanford L. Boye, K. M. Fransen, Jennifer M. Noel +9 more
2015Human Molecular Genetics68 citations Open access
Semantic Scholar
Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS
N. Kanaan, Rhyomi C. Sellnow, Sanford L. Boye, Ben Coberly +6 more
2017Molecular Therapy: Nucleic Acids67 citations Open access
Semantic Scholar
SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration
Y. Sasaki, Hiroki Kakita, S. Kubota, A. Séne +9 more
2020bioRxiv65 citations Open access
Semantic Scholar
Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking
Bridget Lins-Austin, Saajan D. Patel, M. Mietzsch, Dewey Brooke +12 more
2020Viruses53 citations Open access
Semantic Scholar
Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach
S. Petersen-Jones, L. Occelli, Paige A. Winkler, Winston Lee +12 more
2017Journal of Clinical Investigation52 citations Open access
Semantic Scholar
Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa
F. Mowat, L. Occelli, Joshua T. Bartoe, K. Gervais +6 more
2017Frontiers in Neuroscience48 citations Open access
Semantic Scholar
ELOVL4-Mediated Production of Very Long-Chain Ceramides Stabilizes Tight Junctions and Prevents Diabetes-Induced Retinal Vascular Permeability
N. Kady, Xu-Wen Liu, T. Lydic, Meesum Syed +12 more
2018Diabetes45 citations Open access
Semantic Scholar
Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy
Yu-Xin Zhang, Wen-Tao Deng, W. Du, Ping Zhu +9 more
2017Scientific Reports41 citations Open access
Semantic Scholar
Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice
Hong Yu, Rajeshwari D. Koilkonda, Tsung-Han Chou, V. Porciatti +7 more
2015Proceedings of the National Academy of Sciences of the United States of America37 citations Open access
Semantic Scholar
SubILM Injection of AAV for Gene Delivery to the Retina
Paul D. Gamlin, J. Alexander, Sanford L. Boye, C. Witherspoon +1 more
2019Methods in molecular biology36 citations
Semantic Scholar
NF1 is a direct G protein effector essential for opioid signaling to Ras in the striatum
Ke-Qiang Xie, L. Colgan, Maria T. Dao, Brian S Muntean +10 more
2016Current Biology36 citations Open access
Semantic Scholar
Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations
S. Jacobson, A. Cideciyan, A. Ho, I. Peshenko +12 more
2021iScience35 citations Open access
Semantic Scholar
Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study.
Paul Yang, Laura P. Pardon, Allen C Ho, A. Lauer +14 more
2024The Lancet34 citations
Semantic Scholar
A drug-tunable gene therapy for broad-spectrum protection against retinal degeneration
Clayton P. Santiago, C. Keuthan, Sanford L. Boye, Shannon E. Boye +2 more
2018bioRxiv31 citations Open access
Semantic Scholar
Night vision restored in days after decades of congenital blindness
S. Jacobson, A. Cideciyan, A. Ho, Alejandro J. Roman +13 more
2022iScience30 citations Open access
Semantic Scholar
A Novel Mouse Model of MYO7A USH1B Reveals Auditory and Visual System Haploinsufficiencies
Kaitlyn R. Calabro, Sanford L. Boye, S. Choudhury, Diego S. Fajardo +8 more
2019Frontiers in Neuroscience29 citations Open access
Semantic Scholar
Small GTPases Rab8a and Rab11a Are Dispensable for Rhodopsin Transport in Mouse Photoreceptors
Guoxin Ying, Cecilia D. Gerstner, J. Frederick, Sanford L. Boye +2 more
2016PLoS ONE28 citations Open access
Semantic Scholar
Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis
G. Aguirre, A. Cideciyan, V. Dufour, Ana Ripolles García +8 more
2020bioRxiv27 citations Open access
Semantic Scholar
Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors
Min Chen, Kyungah Maeng, Akbar Nawab, Rony A. Francois +8 more
2017Human Gene Therapy Methods27 citations Open access
Semantic Scholar
Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B
Samantha C. Lau, M. Grati, Kevin T. Isgrig, Moaz Sinan +11 more
2023Molecular Therapy: Methods & Clinical Development24 citations Open access
Semantic Scholar
Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy.
Sanford L. Boye, S. Choudhury, S. Crosson, G. Di Pasquale +12 more
2020Molecular Therapy24 citations Open access
Semantic Scholar
Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration
Fan Yang, Hong-Wei Ma, Josh Belcher, Michael R. Butler +4 more
2016The FASEB Journal24 citations Open access
Semantic Scholar
Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion
Patrick C. Kellish, Damien Marsic, Sean M. Crosson, S. Choudhury +16 more
2023Molecular Therapy23 citations Open access
Semantic Scholar
PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina
J. Hickmott, Chih-Yu Chen, David J. Arenillas, Andrea J. Korecki +11 more
2016Molecular Therapy: Methods & Clinical Development22 citations Open access
Semantic Scholar
Utilizing minimally purified secreted rAAV for rapid and cost-effective manipulation of gene expression in the CNS
Marshall S. Goodwin, C. L. Croft, Hunter S. Futch, Daniel H. Ryu +17 more
2020Molecular Neurodegeneration18 citations Open access
Semantic Scholar
Intravitreal Administration of AAV2-SIRT1 Reverses Diabetic Retinopathy in a Mouse Model of Type 2 Diabetes
Yvonne Adu-Agyeiwaah, Cristiano P. Vieira, Bright Asare-Bediako, Sergio Li Calzi +6 more
2023Translational Vision Science & Technology17 citations Open access
Semantic Scholar
Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection
D. Lipinski, Christopher A. Reid, Sanford L. Boye, James J. Peterson +4 more
2015Human Gene Therapy17 citations Open access
Semantic Scholar
AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy
A. Dinculescu, Rachel M. Stupay, Wen-Tao Deng, Frank M. Dyka +12 more
2016PLoS ONE16 citations Open access
Semantic Scholar
Long-term photoreceptor rescue in two rodent models of retinitis pigmentosa by adeno-associated virus delivery of Stanniocalcin-1
G. Roddy, D. Yasumura, M. Matthes, M. Alavi +5 more
2016Experimental Eye Research14 citations Open access
Semantic Scholar
Site-Specific Modifications to AAV8 Capsid Yields Enhanced Brain Transduction in the Neonatal MPS IIIB Mouse
Janine A. Gilkes, Benjamin L. Judkins, Brontie N. Herrera, R. Mandel +4 more
2020Gene Therapy11 citations Open access
Semantic Scholar
A Comprehensive Review of Clinically Applied Adeno-Associated Virus-Based Gene Therapies for Ocular Disease
Valerie G. Hinsch, Sanford L. Boye, Shannon E. Boye
2025Human Gene Therapy10 citations Open access
Semantic Scholar
Engineered AAV capsid transport mutants overcome transduction deficiencies in the aged CNS
I. Sandoval, Christy M Kelley, L. Bernal-Conde, K. Steece-Collier +6 more
2024Molecular Therapy: Nucleic Acids8 citations Open access
Semantic Scholar
Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis
Sanford L. Boye, C. O'Riordan, James E. Morris, M. Lukason +9 more
2022Molecular Therapy: Methods & Clinical Development8 citations Open access
Semantic Scholar
Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6
Russell W. Mellen, Kaitlyn R. Calabro, K. T. Mccullough, S. Crosson +5 more
2023Molecular Therapy: Methods & Clinical Development7 citations Open access
Semantic Scholar
β-secretase 1 overexpression by AAV-mediated gene delivery prevents retina degeneration in a mouse model of age-related macular degeneration
Xiao-Ping Qi, Carolina Francelin, S. Mitter, Sanford L. Boye +4 more
2023Molecular Therapy7 citations Open access
Semantic Scholar
Effects of Altering Heparan Sulfate Proteoglycan Binding and Capsid Hydrophilicity on Retinal Transduction by Adeno-associated Virus
S. Crosson, A. Bennett, Diego S. Fajardo, James J. Peterson +8 more
2021Journal of Virology7 citations Open access
Semantic Scholar
Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice.
Fan Yang, Hong-Wei Ma, Sanford L. Boye, W. Hauswirth +1 more
2018Advances in Experimental Medicine and Biology7 citations
Semantic Scholar
Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells
S. Choudhury, Christianne E. Strang, J. Alexander, Miranda L. Scalabrino +6 more
2016Frontiers in Neuroscience7 citations Open access
Semantic Scholar
Promotion of endoplasmic reticulum retrotranslocation by overexpression of E3 ubiquitin-protein ligase synoviolin 1 reduces endoplasmic reticulum stress and preserves cone photoreceptors in cyclic nucleotide-gated channel deficiency
Fan Yang, Hong-Wei Ma, Sanford L. Boye, Shannon E. Boye +1 more
2024The FASEB Journal6 citations
Semantic Scholar

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