Gene Therapy for Retinal Degeneration

• Published in: Cell Vol. 173; no. 1; p. 5
• Main Author: Apte, Rajendra S.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 22-03-2018
• Subjects: Adenoviridae - genetics; cis-trans-Isomerases - genetics; cis-trans-Isomerases - metabolism; Genetic Therapy; Genetic Vectors - economics; Genetic Vectors - genetics; Genetic Vectors - therapeutic use; Humans; Leber Congenital Amaurosis - epidemiology; Leber Congenital Amaurosis - genetics; Leber Congenital Amaurosis - therapy; Retinal Degeneration - epidemiology; Retinal Degeneration - genetics; Retinal Degeneration - therapy
• ISSN: 0092-8674; 1097-4172
• DOI: 10.1016/j.cell.2018.03.021

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.
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.