Gene therapy for retinitis pigmentosa

Curr Opin Mol Ther. 2000 Aug;2(4):420-5.


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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Apoptosis / genetics
  • Genetic Therapy / methods*
  • Growth Substances / genetics
  • Humans
  • Mutation
  • Retina
  • Retinitis Pigmentosa / genetics
  • Retinitis Pigmentosa / therapy*


  • Growth Substances