Top Institutions in Ophthalmic Gene Therapy for Usher Syndrome Type 1B
Leading institutions conduct early-phase clinical trials and translational research involving AAV-mediated gene delivery targeting the MYO7A gene, utilizing advanced ophthalmic imaging and functional vision assessments to evaluate safety and efficacy.
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#1
University Hospital of Campania
Naples, Italy
Leading the LUCE-1 phase 1/2 trial of AAVB-081 gene therapy for Usher 1B, with principal investigator Dr. Francesca Simonelli presenting promising safety and efficacy data at EURETINA 2025.
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#2
Massachusetts Eye and Ear Infirmary, Harvard Medical School
Boston, MA
Pioneers in retinal gene therapy research with extensive experience in clinical trials for inherited retinal dystrophies including Usher syndrome, leveraging advanced molecular and imaging techniques.
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#3
Johns Hopkins Wilmer Eye Institute
Baltimore, MD
Renowned for clinical and basic research in genetic retinal disorders and gene therapy, with ongoing studies targeting Usher syndrome and related retinal degenerations.
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#4
University of Pennsylvania Perelman School of Medicine
Philadelphia, PA
Leads innovative gene therapy research programs with a focus on AAV vector optimization and clinical translation for retinal dystrophies including Usher syndrome.