Gene Therapy for Retinal Degeneration.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 29570997.
- Also identified by DOI 10.1016/j.cell.2018.03.021.
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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.
Medical subject headings
- Retinal Degeneration