Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people with Usher syndrome type 1F, a rare condition that causes deafness and progressive blindness.
- By STEPHANIE DUTCHEN
A new way of delivering a corrected version of the faulty gene that causes Usher syndrome - PCDH15 - restored hearing in mouse models and showed potential in retinal organoids and nonhuman primates for improving vision, the team reports in the Journal of Clinical Investigation.
This is the second experimental gene therapy for Usher syndrome developed by the lab of David Corey, the Bertarelli Professor of Translational Medical Science in the Blavatnik Institute at HMS. Earlier research showed that a different gene-delivery strategy restored hearing in mice. The new method provides a second option if the first approach proves unsafe or ineffective when tested in humans.
