Treatment Strategy With Gene Editing for Late-Onset Retinal Degeneration Caused by a Founder Variant in C1QTNF5

• Published in: Investigative ophthalmology & visual science Vol. 64; no. 15; p. 33
• Main Authors: Li, Randa T. H., Roman, Alejandro J., Sumaroka, Alexander, Stanton, Chloe M., Swider, Malgorzata, Garafalo, Alexandra V., Heon, Elise, Vincent, Ajoy, Wright, Alan F., Megaw, Roly, Aleman, Tomas S., Browning, Andrew C., Dhillon, Baljean, Cideciyan, Artur V.
• Format: Journal Article
• Language: English
• Published: United States 01.12.2023
• Subjects: Adult; Aged; Collagen - genetics; Female; Fovea Centralis - pathology; Gene Editing; Genetic Therapy - methods; Humans; Late Onset Disorders - genetics; Late Onset Disorders - pathology; Late Onset Disorders - therapy; Male; Middle Aged; Retinal Degeneration - genetics; Retinal Degeneration - pathology; Retinal Degeneration - therapy; Tomography, Optical Coherence

Genome editing is an emerging group of technologies with the potential to ameliorate dominant, monogenic human diseases such as late-onset retinal degeneration (L-ORD). The goal of this study was to identify disease stages and retinal locations optimal for evaluating the efficacy of a future genome...