A recently published study, led by the Corey Lab at Harvard Medical School and with our collaborative efforts, explores a novel gene therapy for Usher syndrome type 1F (USH1F)—a genetic condition causing hearing, balance, and vision impairments. The study details a dual-adeno-associated virus (AAV) strategy designed to overcome the limited packaging capacity of AAV vectors, allowing delivery of the large PCDH15 gene, which is crucial for treating USH1F.
The Corey Lab demonstrated the efficacy of this dual-AAV approach in mouse models, restoring hearing and balance. In our contribution, we tested the therapy in human retinal organoids, showing it successfully targets photoreceptor cells and supports correct PCDH15 protein expression—an important step in assessing the therapy’s potential for vision restoration.
This dual-AAV approach marks a promising advance toward a comprehensive treatment for USH1F, addressing its complex sensory challenges.
See paper here: https://www.jci.org/articles/view/177700
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