title: Retinal dystrophies and gene therapy.
abstract: Retinal dystrophies are inherited disorders of photoreceptor and retinal pigment epithelial function that may result in severe visual impairment. Advances in molecular genetics have helped identify many of the gene defects responsible, and progress in gene transfer technology has enabled therapeutic strategies to be developed and applied. The first human clinical trials of gene therapy for RPE65 associated retinal dystrophy have shown promising initial results and have helped prepare the way for further trials of gene therapy for inherited retinal disorders. The results of these trials will provide further insight into the safety and efficacy of gene therapy for a range of currently untreatable and debilitating eye disorders.
authors.affiliation: Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK.
authors.forename: Venki
authors.initials: V
authors.lastname: Sundaram
authors.forename: Anthony T
authors.initials: AT
authors.lastname: Moore
authors.forename: Robin R
authors.initials: RR
authors.lastname: Ali
authors.forename: James W
authors.initials: JW
authors.lastname: Bainbridge
mesh_terms: D002648:Child; D040181:Genetic Diseases, X-Linked / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D010786:Photoreceptor Cells; D010857:Pigment Epithelium of Eye; D058499:Retinal Dystrophies / Q000235:genetics / Q000628:therapy*
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