July 8, 2020
Shannon Boye, PhD
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Presentation Summary:
This presentation will focus on the following main points:
- Description of Usher syndrome 1B
- MYO7A gene/protein function
- Clinical characterization (retina)
- MYO7A deficient mice don’t mimic patient condition (have very subtle phenotype rather than retinal degeneration and dysfunction)
- That is likely due to differential expression of MYO7A between mice and primates (MYO7A not very important in mouse photoreceptors, but very important in primate photoreceptors)
- MYO7A is too large to fit inside a conventional AAV vector. For this reason, initial attempts focused on lentivirus. Clinical trials using lentivirus to delivery MYO7A have shown no signs of efficacy.
- Dual AAV vector technology
- Improvements we’ve made to dual vectors
- Dual AAV-MYO7A vectors well-tolerated in subretinally injected macaque
Speaker Bio:
Dr. Shannon E. Boye is Associate Chief of the Division of Cellular and Molecular Therapy in the University of Florida’s Department of Pediatrics. She 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, 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 Therapy.
Dr. Boye 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, the ARVO Foundation/Pfizer Ophthalmics Carl Camras Translational Research Award, a University of Florida Research Foundation Professorship, and the Gund Harrington Scholar Award for excellence in gene therapy research.
