Gene Therapy for Retinal Degeneration.

Rajendra S Apte

Journal: Cell 2019;173(1):5

PMID: 29570997

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Abstract

Biallelic mutations in the RPE65 gene are associated with inherited retinal degenerations/dystrophies (IRD) and disrupt the visual cycle, leading to loss of vision. A new adenoviral vector-based gene therapy surgically delivered to retinal cells provides normal human RPE65 protein that can restore the visual cycle and some vision. To view this Bench to Bedside, open or download the PDF.

Address: Washington University School of Medicine, 660 South Euclid Avenue, Box 8096, St. Louis, MO 63110, USA. Electronic address: [email protected].

DOI: 10.1016/j.cell.2018.03.021

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MeSH Terms: Adenoviridae, Genetic Therapy, Genetic Vectors, Humans, Leber Congenital Amaurosis, Retinal Degeneration, cis-trans-Isomerases