Regenerative medicine association from Romania

Gene therapy effective in genetic eye disease

First results from a phase I/II clinical trial are giving hope for improving condition of patients with a genetic eye disease called choroidemia which leads to blindness due to mutations in the CHM gene, which encodes the Rab escort protein 1 (REP1). Gene therapy with AAV.REP1 partially restored vison in six patients giving hope to being able to stop retinal degeneration and improve quality of life of these patients. The article has been published in Lancet on 16 Th of january http://www.thelancet.com/journals/lancet/article/PIIS0140-6736%2813%2962117-0/abstract

 

 

 

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