Gene therapy for retinitis pigmentosa
Bennett, J.
Current Opinion in Molecular Therapeutics 2(4): 420-425
2000
ISSN/ISBN: 1464-8431 PMID: 11249772 Document Number: 523803
Retinitis pigmentosa (RP) is a group of retinal degenerative diseases in which there is a slow and progressive loss of photoreceptors. There is no cure for RP and photoreceptor loss leads ultimately to blindness. There has been tremendous progress in the last decade in delineating the molecular basis of RP. Simultaneously, gene transfer experiments have demonstrated that it is possible to deliver transgenes to the retina in vivo in a stable and efficient fashion with minimal toxicity. Proof-of-principle for gene therapy for RP has been established in a number of different animal models. While much more progress needs to be made before moving from the laboratory to the clinic, gene therapy now holds much promise for slowing or even preventing blindness due to RP.