Rescue of sight by gene therapy - closer than it may appear.
This review will cover the state of the field in retinal degeneration and gene therapy with a focus on the great strides that have been made in retina gene therapy. Topics ranging from the development of animal models to clinical trials (for the treatment of Leber congenital amaurosis, age-related macular degeneration, and retinoblastoma) will be discussed. In addition, the results of gene therapy studies targeting the photoreceptors will be presented. Finally, strategies and progress in overcoming the challenges of photoreceptor-directed gene therapy will be presented.
Rex
Tonia S
TS
F.M. Kirby Center for Molecular Ophthalmology, University of Pennsylvania, Philadelphia, USA. trex@utmem.edu
D000818:Animals; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000639:trends; D006801:Humans; D008268:Macular Degeneration / Q000628:therapy; D029242:Optic Atrophy, Hereditary, Leber / Q000628:therapy; D020419:Photoreceptor Cells, Vertebrate; D012162:Retinal Degeneration / Q000503:physiopathology / Q000628:therapy*; D019572:Retinal Neoplasms / Q000628:therapy; D012175:Retinoblastoma / Q000628:therapy