
pmid: 29570997
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.
cis-trans-Isomerases, Genetic Vectors, Leber Congenital Amaurosis, Retinal Degeneration, Humans, Genetic Therapy, Adenoviridae, Retinoid Isomerohydrolase
cis-trans-Isomerases, Genetic Vectors, Leber Congenital Amaurosis, Retinal Degeneration, Humans, Genetic Therapy, Adenoviridae, Retinoid Isomerohydrolase
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| influence This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically). | Top 10% | |
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