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Bardet Biedl Syndrome Family Association

Connecting families and sharing information on research, treatment, and therapies for Bardet Biedl Syndrome.

A new approach to treating retinal degeneration

Scientists at the University of Oxford have tested a new approach to restoring retinal cells on mice with great success. Current work on gene therapies have focused on introducing a replacement gene for a defective one in people with a degenerative retinal condition. This approach injects not a replacement gene, but full replacement cells.

The gene therapy approach is in clinical trials for people with Leber’s Congenital Amaurosis (LCA) and in animal trials for one BBS-variant. This new almost-stem-cell approach has only been tested in mice. Typically it takes almost ten years between animal testing and the beginning of a clinical trial in people.

Blind Mice See After Cell Transplant