Gene therapy of inherited retinal degenerations: prospects and challenges

Human Gene Therapy
Ivana TrapaniAlberto Auricchio

Abstract

Because of its favorable anatomical and immunological characteristics, the eye has been at the forefront of translational gene therapy. Dozens of promising proofs of concept have been obtained in animal models of inherited retinal degenerations (IRDs), and some of them have been relayed to the clinic. The results from the first clinical trials for a congenital form of blindness have generated great interest and have demonstrated the safety and efficacy of intraocular administrations of viral vectors in humans. However, this progress has also generated new questions and posed challenges that need to be addressed to further expand the applicability of gene therapy in the eye, including safe delivery of viral vectors to the outer retina, treatment of dominant IRDs as well as of IRDs caused by mutations in large genes, and, finally, selection of the appropriate IRDs and patients to maximize the efficacy of gene transfer. This review summarizes the strategies that are currently being exploited to overcome these challenges and drive the clinical development of retinal gene therapy.

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Jul 30, 2015·Human Gene Therapy·Thomas J Corydon
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Methods Mentioned

BETA
genotyping

Clinical Trials Mentioned

NCT00516477
NCT00643747
NCT00481546
NCT00999609
NCT01496040
NCT01482195
NCT01367444
NCT01505062
NCT01267422
NCT02064569

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