Gene therapy to treat inherited and complex retinal degenerative diseases

@article{Rex2015GeneTT,
  title={Gene therapy to treat inherited and complex retinal degenerative diseases},
  author={Tonia S. Rex},
  journal={Molecular Therapy. Methods \& Clinical Development},
  year={2015},
  volume={2}
}
  • T. Rex
  • Published 5 August 2015
  • Biology
  • Molecular Therapy. Methods & Clinical Development
These are exciting times in the field of retinal gene therapy, as impressive successes have been achieved by recombinant adeno-associated viral (rAAV) gene delivery of Rpe65 or Rep1 to the retinal pigment epithelium of patients with Lebers congential amaurosis or choroideremia, respectively.1–5 Instead of being left with incurable blindness, patients are reporting restoration of vision. An advantage of these disease targets is that the affected cell type, the retinal pigment epithelium, is… 
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Retinitis pigmentosa (RP), an inherited retinal degenerative disease causing blindness, is characterized by progressive apoptotic death of photoreceptors. Therapeutic modification of photoreceptor