Shannon E Boye

Shannon E Boye,

Professor And Associate Division Chief – Cellular And Molecular Therapy, Department Of Pediatrics

Department: MD-PEDS-CELL / MOLEC THERP DIV
Business Phone: (352) 273-9342
Business Email: shaire@ufl.edu

About Shannon E Boye

Dr. Shannon E. Boye received her B.S. in marine biology with a minor in chemistry from Fairleigh Dickinson University in 2001. She graduated with a Ph.D. in Neuroscience from the University of Florida in 2006. Her thesis work involved developing viral vectors for the treatment of retinal disease, specifically GUCY2D Leber Congenital Amaurosis (LCA1). Following a postdoctoral fellowship and research assistant professorship under Dr. William Hauswirth, Dr. Boye was appointed as tenure track Assistant Professor in the University of Florida’s Department of Ophthalmology (2012). In 2016, she received tenure and was promoted to the rank of Associate Professor. In 2020, she transferred to the Department of Pediatrics where she is now Associate Chief of the Division of Cellular and Molecular Therapeutics.

Dr. Boye’s memberships in professional societies include the Association for Research in Vision and Ophthalmology (ARVO) and the American Society of Gene and Cell Therapy (ASGCT). She currently serves as PI, Co-PI or Co-Investigator on several federally as well as privately funded grants and is actively involved in the University of Florida’s teaching mission. She is actively engaged in community outreach and is a highly sought after speaker at patient-oriented conferences. Her real passion is interfacing with those affected by the diseases she studies and educating these individuals about treatment options and ongoing research to develop future treatments. For her efforts, she is the three-time recipient of the Exemplary Teacher Award from UF’s College of Medicine. She has authored over 50 peer-reviewed manuscripts, multiple textbook chapters, is actively involved in grant and manuscript review, and is the recipient of several major awards including the ARVO Foundation/Merck Innovative Ophthalmology Research Award in Gene Therapy and Eye Disease, the Foundation Fighting Blindness Board of Director’s Award, a UF Term Professorship, and the Gund Harrington Scholar Award for excellence in gene therapy research.

When she is not at work, Dr. Boye still spends most of her time in the ‘Wet Lab’ (our nerdy boat name) with her husband and two young children.

Accomplishments

UF Research Foundation Professorship
2019 · University of Florida
Exemplary Teacher Award
2018 · University of Florida
Pfizer Carl Camras Translational Research Award
2018 · ARVO Foundation
Exemplary Teacher Award
2017 · University of Florida
Scholar Award
2017 · Gund Harrington
University Term Professorship
2017 · University of Florida
Visionary Award
2016 · MOMS for Sight 2016
Board of Director's Award
2015 · Foundation Fighting Blindness
Nominee for Life Sciences Category for the Blavatnik Awards for Young Scientists
2015 · University of Florida
Exemplary Teacher Award
2014 · University of Florida
Travel Fellowship
2014 · XVIth International Symposium on Retinal Degenerations
IOVS
2013 · Exceptionally Good Review ranking
Merck Innovative Ophthalmology Research Award in Gene Therapy and Eye Disease Price
2013 · ARVO Foundation
Early Career Reviewer Program
2012 · National Institute of Health
Pediatric Loan Repayment Award Renewal
2012 · National Institute of Health
Pediatric Loan Repayment Award Renewal
2010 · National Institute of Health
Pediatric Loan Repayment Award
2008 · National Institute of Health
Grant Awardee
2006 · T32 Vision Research Training
Office of Naval Research
2002 · Enterprise Fellowship
Alumni Fellowship
2001-2005 · University of Florida's College of Medicine

Research Profile

The focus of my research is developing viral vector-based gene therapies for the treatment of inherited ocular disease. My current focus areas are:

1. to develop AAV-based gene therapy approaches for delivery of genes to the outer retina (notably foveal cones) 2. to develop an Adeno associated virus (AAV)-based gene therapy for treatment of autosomal recessive GUCY2D-Leber congenital amaurosis-1 (LCA1) 3. to develop dual AAV vector platforms that are capable of delivering large transgenes (> 5kb) 4. to develop a AAV-CRISPR/Cas9-based therapies for dominant inherited retinal disease 5. to develop AAV-based gene therapy approaches for delivery of genes to the trabecular meshwork to address treatments for primary open angle glaucoma

I have extensive experience characterizing animal models of inherited retinal disease, developing novel AAV vectors via both rational design and directed evolution, and testing these vectors for their ability to deliver genes to animal models of retinal disease. The work my team conducted over the last 15 years established that gene replacement restores retinal function/visually guided behavior, and preserves retinal structure over the long term in models of autosomal recessive LCA1. As a result, the FDA approved our initiation of PhaseI/II trials to treat patients afflicted with this devastating form of blindness (clinical trials began in fall 2019). I have three awarded patents, and eight pending patents emanating from my research program and am actively funded by the NIH, private foundations, and pharma. I am the recipient of several major awards including the Foundation Fighting Blindness’s Board of Director’s Award, the Gund Harrington Scholar Award, the ARVO Foundation/Merck Innovative Ophthalmology Research Award, and the ARVO/Pfizer Carl Camras Translational Research Award. Since 2010, I have given over 60 invited lectures both within and outside the USA. I have served as a member of study sections both for the NIH and the Department of Defense. I was the recipient of the NIH’s Loan Repayment Award, currently serve as an LRP ambassador and serve on study sections to review new proposals to this grant mechanism. I perform grant review for various other national and international foundations, serve on the editorial advisory board for multiple journals and perform ad hoc peer review for many more. I have participated in workshops including the NIH’s Office of Science Policy/Office of Biotechnology Activities “Gene Therapy: Charting a Future Course”. Outside of my research, I am actively involved in the teaching mission of the Department of Ophthalmology and College of Medicine at the University of Florida and am passionate about outreach/education outside the University. I routinely provide lab tours for visually impaired patients, host foundation meetings and educate patients about ongoing research being conducted to address treatments for their conditions/provide them with information on how to find out more about their disease.

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
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 D-Sequence-Mediated Suppression of Expression of a Human Major Histocompatibility Class II Gene: Implications in the Development of Adeno-Associated Virus Vectors for Modulating Humoral Immune Response.
Human gene therapy. 31(9-10):565-574 [DOI] 10.1089/hum.2020.018. [PMID] 32220217.
2020
Identifying Treatments for Taste and Smell Disorders: Gaps and Opportunities.
Chemical senses. 45(7):493-502 [DOI] 10.1093/chemse/bjaa038. [PMID] 32556127.
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
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.
2017
Defining Outcomes for Clinical Trials of Leber Congenital Amaurosis Caused by GUCY2D Mutations.
American journal of ophthalmology. 177:44-57 [DOI] 10.1016/j.ajo.2017.02.003. [PMID] 28212877.
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.
2017
The GCaMP-R Family of Genetically Encoded Ratiometric Calcium Indicators.
ACS chemical biology. 12(4):1066-1074 [DOI] 10.1021/acschembio.6b00883. [PMID] 28195691.
2016
A Mini-review: Animal Models of GUCY2D Leber Congenital Amaurosis (LCA1).
Advances in experimental medicine and biology. 854:253-8 [DOI] 10.1007/978-3-319-17121-0_34. [PMID] 26427419.
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
Increased vulnerability of photoreceptors to aberrant splicing highlight the utility of AON-based therapy for CEP290-LCA.
Stem cell investigation. 3 [DOI] 10.21037/sci.2016.12.01. [PMID] 28078276.
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
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
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.
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
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
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.
Human molecular genetics. 24(21):6229-39 [DOI] 10.1093/hmg/ddv341. [PMID] 26310623.
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.
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: charting a future course–summary of a National Institutes of Health Workshop, April 12, 2013.
Human gene therapy. 25(6):488-97 [DOI] 10.1089/hum.2014.045. [PMID] 24773122.
2014
Insights gained from gene therapy in animal models of retGC1 deficiency.
Frontiers in molecular neuroscience. 7 [DOI] 10.3389/fnmol.2014.00043. [PMID] 24860425.
2014
Leber congenital amaurosis caused by mutations in GUCY2D.
Cold Spring Harbor perspectives in medicine. 5(1) [DOI] 10.1101/cshperspect.a017350. [PMID] 25256176.
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
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
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
Dual Adeno-Associated Virus Vectors for Delivery of Large Genes To the Retina
Molecular Therapy. 21:S86-S87
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
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
Retinal pigment epithelial detachment in ABCA4-associated Stargardt’s disease.
Ophthalmic surgery, lasers & imaging retina. 44(4):401-4 [DOI] 10.3928/23258160-20130715-11. [PMID] 23883535.
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
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors.
PloS one. 8(4) [DOI] 10.1371/journal.pone.0062097. [PMID] 23637972.
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
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.
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
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.
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
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
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.
2009
Gene Therapy Prevents Cone Degeneration in Two Models of Rpe65 Leber Congenital Amaurosis: Rd12 and Rpe65(-/-):: Rho(-/-) Mice
Molecular Therapy. 17:S288-S289
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

Grants

Nov 2021 ACTIVE
ATSN-CAP-RD-01, P2 Peptide Library Selection
Role: Principal Investigator
Funding: ATSENA THERAPEUTICS
Sep 2021 ACTIVE
Task Order #8 ATSN-201 Vector Bridging Study
Role: Principal Investigator
Funding: ATSENA THERAPEUTICS
Aug 2021 ACTIVE
Task Order #7 – Hybrid Toxicology, Efficacy and Biodistribution (BD) Study in support of an AAV gene therapy for the treatment of XLRS (ATSN-201) following subretinal administration in RS1-/- knockout mic
Role: Principal Investigator
Funding: ATSENA THERAPEUTICS
Oct 2020 ACTIVE
AAV-MEDIATED THERAPY FOR VISUAL IMPAIRMENT ASSOCIATED WITH FRIEDREICH'S ATAXIA
Role: Principal Investigator
Funding: FRIEDRICH'S ATAXIA RESEARCH ALLIANCE
Sep 2020 – Aug 2021
Vascular Gene Delivery and Early Disease Biomarkers in Diabetic Retinopathy Yr. 3
Role: Principal Investigator
Funding: MEDICAL COLLEGE OF WISCONSIN via NATL INST OF HLTH NEI
Jul 2020 ACTIVE
Maintenance of mouse models of LCA1
Role: Principal Investigator
Funding: ATSENA THERAPEUTICS
Jun 2020 – May 2021
Nonhuman Primate Model of Usher Syndrome.
Role: Principal Investigator
Funding: OREGON HLTH AND SCIENCES UNIV via FOU FOR FIGHTING BLINDNESS
Apr 2020 ACTIVE
Task Order #1 – AAV-hRS1 Pharmacology
Role: Principal Investigator
Funding: ATSENA THERAPEUTICS
Apr 2020 ACTIVE
Task Order #2 – Dual AAV-MYO7A Candidate evaluation in Myo7a KO mice
Role: Principal Investigator
Funding: ATSENA THERAPEUTICS
Sep 2019 – Sep 2020
A Ribozyme Rescue Strategy for Autosomal Dominant Retinitis Pigmentosa
Role: Principal Investigator
Funding: *UNIV OF BUFFALO via NATL INST OF HLTH NEI
Sep 2019 ACTIVE
Engineering AAV for safe and efficient gene delivery to the human retina
Role: Principal Investigator
Funding: NATL INST OF HLTH NEI
Sep 2019 – Aug 2020
Vascular gene delivery and early disease biomarkers in diabetic retinopathy
Role: Principal Investigator
Funding: MEDICAL COLLEGE OF WISCONSIN via NATL INST OF HLTH NEI
Jun 2019 ACTIVE
DEVELOPMENT OF AAV-CRISPR/CAS9-BASED THERAPIES FOR CONE ROD DYSTROPHY
Role: Principal Investigator
Funding: NATL INST OF HLTH NEI
Jun 2019 ACTIVE
Enhancing Metabolism in Photoreceptors with a Modified Arrestin to Treat Retinal Degeneration
Role: Project Manager
Funding: FOU FOR FIGHTING BLINDNESS
Apr 2019 – Dec 2020
Dual AAV vector-mediated therapy for MyosinVIIa Usher syndrome (USH1B)
Role: Principal Investigator
Funding: FOU FOR FIGHTING BLINDNESS
Sep 2018 – Aug 2019
Vascular gene delivery and early disease biomarkers in diabetic retinopathy
Role: Principal Investigator
Funding: MEDICAL COLLEGE OF WISCONSIN via NATL INST OF HLTH NEI
Sep 2018 – Dec 2019
Exploring MYO7A function in novel mouse models and improving AAV-Dual Vector Gene Therapy for Usher Syndrome 1B
Role: Other
Funding: NATL INST OF HLTH NEI
Sep 2018 – Mar 2019
Developing a dual AAV vector gene therapy for the treatment of Usher syndrome
Role: Principal Investigator
Funding: HARRINGTON DISCOVERY INSTITUTE via FOU FOR FIGHTING BLINDNESS
Nov 2017 – Dec 2019
Cas9 mediated gene editing therapy for CORD6 cone rod dystrophy/Extension of Sponsored Research Agreement btw Editas Medicine and the University of Florida
Role: Principal Investigator
Funding: EDITAS MEDICINE
Aug 2017 ACTIVE
Optimizing AAV Vectors for Central Nervous System transduction
Role: Co-Investigator
Funding: NATL INST OF HLTH NINDS
May 2017 ACTIVE
Therapy development for open-angle glaucoma
Role: Principal Investigator
Funding: MICHIGAN STATE UNIV via NATL INST OF HLTH NEI
Jan 2016 – Jun 2018
Cas9 mediated in vivo gene editing of photoreceptors in the nonhuman primate and in a mouse model of cone rod dystrophy
Role: Principal Investigator
Funding: EDITAS MEDICINE
Dec 2015 – Dec 2020
miscellaneous donors
Role: Principal Investigator
Funding: MISCELLANEOUS DONORS
Dec 2015 – Nov 2017
AAV-mediated optogenetic gene therapy in bipolar cells
Role: Principal Investigator
Funding: APPLIED GENETICS TECH CORP
Sep 2014 – Aug 2018
AAV Capsid Library Screening
Role: Project Manager
Funding: APPLIED GENETICS TECH CORP
Aug 2014 – Feb 2017
Dual AAV vector-mediated therapy for Myosin7a Usher syndrome (USH1B)
Role: Principal Investigator
Funding: FOU FOR FIGHTING BLINDNESS
Apr 2014 – May 2019
Developing efficient AAV vectors for photoreceptor targeting via the vitreous
Role: Principal Investigator
Funding: NATL INST OF HLTH NEI
Mar 2014 – Dec 2018
Gene Therapy for LCA1
Role: Principal Investigator
Funding: GENZYME CORP

Patents

Issued November 2017
rAAV-Guanylate Cyclase Compositions and Methods for Treating Leber's Congenital Amaurosis-1 (LCA1)
#9,816,108
Published October 2017
rAAV-Guanylate Cyclase Compositions and Methods for Treating Leber's Congenital Amaurosis-1 (LCA1) (CON)
#US-2018-0100165
Published February 2016
rAAV Vector Compositions, Methods for Targeting Vascular Endothelial Cells and Use in Treatment of Type I Diabetes
#us2018/0057840
Published March 2015
Improved rAAV Vectors and Methods for Transduction of Photoreceptors and RPE Cells
#US2016/0369299
Published February 2015
Methods and Compositions for Gene Delivery to On Bipolar Cells
#US2017/0007720
Published May 2014
Dual-AAV Vector-Based Systems and Methods for Delivering Oversized Genes to Mammalian Cells
#US2014/0256802

Education

Postdoctoral fellowship/ Ophthalmology and Gene Therapy
2006-2008 · University of Florida
Ph.D./ Neuroscience
2006 · University of Florida
BS/Marine Biology and Chemistry
2001 · Fairleigh Dickinson University

Teaching Profile

Courses Taught
2020-2021
GMS6252 Molecular Therapy II ? Disease Targets and Applications
2017-2019,2021
GMS6070 Sensory and Motor Systems
2018
MCB4911 Supervised Research in Microbiology and Cell Science
2018
GMS7980 Research for Doctoral Dissertation
2018
GMS7979 Advanced Research
2013-2017
GMS6791 Visual Neuroscience Journal Club
2013-2015
GMS6417 Integrative Aging Physiology
2013,2015
GMS6709 Current Topics in Vision
2015
GMS6029 Brain Journal Club

Contact Details

Phones:
Business:
(352) 273-9342
Emails:
Business:
shaire@ufl.edu