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Bardet Biedl Syndrome Family Association

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

Update on Gene Therapy for Retinal Degeneration Trial

The first trial for a gene therapy treatment for a retinal degeneration was focused on a disease called Leber Congenital Amaurosis or LCA. The initial results were very promising. 

Three year follow-ups have been disappointing as this article reports. But as Dr. Eric Pierce, who spoke at our 2012 conference, says in the piece, it would have been shocking to see the very first attempt at gene therapy to restore degraded retinas work perfectly. The most important news is still the same: the technique shows promise.

Note that this article is from a business magazine so they take an overly pessimistic view, mainly concerned about the impact on the current stock price of a particular company, not about the scientific advances in general.

There are also a number of other gene therapy trials at different stages. The bottom line: there is still a long way to go, but progress is being made.