On Retinal Gene Therapy | Zendy

M. Dominik Fischer | Zendy

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On Retinal Gene Therapy

Author(s) -

M. Dominik Fischer

Publication year - 2016

Publication title -

ophthalmologica

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.639

H-Index - 60

eISSN - 1423-0267

pISSN - 0030-3755

DOI - 10.1159/000445782

Subject(s) - genetic enhancement , retinal , blindness , retinal degeneration , retinitis pigmentosa , neuroscience , medicine , bioinformatics , gene , biology , ophthalmology , optometry , genetics

Mutations in a large number of genes cause retinal degeneration and blindness with no cure currently available. Retinal gene therapy has evolved over the last decades to become a promising new treatment paradigm for these rare disorders. This article reflects on the ideas and concepts arising from basic science towards the translation of retinal gene therapy into the clinical realm. It describes the advances and present thinking on the efficacy of current clinical trials and discusses potential roadblocks and solutions for the future of retinal gene therapy.

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