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USH1D Research

Gene: CDH23
Year Identified: 2001

Each research project listed below will include a graphic of the research continuum. The gold box indicates where this project falls on the continuum, illustrating its progress towards reaching people living with Usher syndrome, from "Bench to Bedside."

Click here to learn more about the different stages in the research continuum.

Gene Therapy USH1D

Research Continuum Graphic. Research in Pre-clinical stage. Sections shown in grey: Basic & Translational, Clinical trials: Phase I, Phase II, Phase III, Treatments-Phase IV. Highlighted in yellow: Pre-Clinical.

Triple AAV Vectors for USH1D: 
A research team in Naples, Italy has tested retinal gene transfer in mouse and pig retinas by using triple AAV vectors to maximize the transfer capacity for larger genes involved in Inherited Retinal Diseases such as USH1D. Translation was sufficient; therefore, triple AAV vectors could enable gene therapy for USH1D.
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