100-fold improvement in sight seen after gene therapy trial
Vision improved 100 times on average in 15 people with Leber congenital amaurosis after gene therapy targeted mutations in the GUCY2D gene. The Phase I/II trial, co-led by researchers from the Perelman School of Medicine at the University of Pennsylvania and published in The Lancet, included three pediatric patients. Some patients receiving the highest dose experienced a 10,000-fold improvement. One patient reported navigating outdoors at midnight with only the light of a bonfire.
The therapy improved severe vision loss caused by LCA1, a rare inherited condition affecting less than 100,000 people worldwide.
Source: 100-fold improvement in sight seen after gene therapy trial (medicalxpress.com). Spotted via Phys.org.
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