September 1, 2020

Disease mechanisms and gene therapy for Usher syndrome

This 2020 review summarizes how research efforts have advanced greatly allowing medical professionals to make diagnoses quicker. Having a greater understanding of how this rare disease operates supports both development of therapeutic products and clinical trials.

What this means for Usher syndrome: Understanding the role that USH genes play in the inner ear as well as the retina is the first step to develop treatments.

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