title: Gene therapy for eye as regenerative medicine? Lessons from RPE65 gene therapy for Leber's Congenital Amaurosis.
abstract: Recombinant virus mediated gene therapy of Leber's Congenital Amaurosis has provided a wide range of data on the utility of gene replacement therapy for recessive diseases. Studies to date demonstrate that gene therapy in the eye is safe and can result in long-term recovery of visual function, but they also highlight that further research is required to identify optimum intervention time-points, target populations and the compatibility of associate therapies. This article is part of a directed issue entitled: Regenerative Medicine: the challenge of translation.
authors.affiliation: Centre for Ophthalmology and Visual Sciences, The University of Western Australia, Crawley, 6009, Western Australia, Australia. Electronic address: elizabeth.rakoczy@uwa.edu.au.
authors.forename: Elizabeth P
authors.initials: EP
authors.lastname: Rakoczy
authors.affiliation: College of Veterinary Medicine, University of Missouri, Columbia, MO, USA.
authors.forename: Kristina
authors.initials: K
authors.lastname: Narfström
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D000229:Dependovirus / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D057130:Leber Congenital Amaurosis / Q000235:genetics* / Q000628:therapy*; D009154:Mutation; D011995:Recombination, Genetic; D044968:Regenerative Medicine / Q000379:methods* / Q000639:trends; D013997:Time Factors; D016896:Treatment Outcome; D014785:Vision, Ocular; D019745:cis-trans-Isomerases / Q000235:genetics* / Q000378:metabolism; D000099178:Retinoid Isomerohydrolase
chemical_list: D019745:cis-trans-Isomerases; D000099178:Retinoid Isomerohydrolase
keywords: Animal models; Gene therapy; Human trials; Leber's Congenital Amaurosis; Retinal degeneration