Some eye desease can be treated nowadays by  cell therapy, although the results have yet to be confirmed (The Lancet, 15 October 2014). However, there is no effective treatment for inherited retinal degeneration at present.

Proposed eye disease gene therapy

It has now been proposed, for the first time, to treat one of these serious diseases by gene therapy, substituting the affected gene (RPE65) for a healthy gene. The result is a substantial improvement in vision, which is maintained for at least three years (The Lancet 383; 1105-1107, 214).  The treatment of choroideremia, a degenerative retinal disease, has also been specifically proposed (The Lancet 2014; 383, 1129-1137).