The new landscape of retinal gene therapy. Issue 3 (5th September 2020)

• Record Type: Journal Article
• Authors: Ku, Cristy A.; Pennesi, Mark E.
• Other Names: Hufnagel Robert guestEditor.; Walter Michael guestEditor.; Arno Gavin guestEditor.
• Is Part Of: American journal of medical genetics. Volume 184:Issue 3(2020)
• Page Start: 846
• Page End: 859
• Publication Date: 2020-09-05
• Subjects: gene editing -- gene therapy -- inherited retinal dystrophies -- RNA modulation; Medical genetics -- Periodicals; 616.04205
• DOI: 10.1002/ajmg.c.31842
• Languages: English
• ISSNs: 1552-4868

Abstract

Abstract: Novel therapeutics for inherited retinal dystrophies (IRDs) have rapidly evolved since groundbreaking clinical trials for LCA due to RPE65 mutations led to the first FDA‐approved in vivo gene therapy. Since then, advancements in viral vectors have led to more efficient AAV transduction and developed other viral vectors for gene augmentation therapy of large gene targets. Furthermore, significant developments in gene editing and RNA modulation technologies have introduced novel capabilities for treatment of autosomal dominant diseases, intronic mutations, and/or large genes otherwise unable to be treated with current viral vectors. We highlight strategies currently being evaluated in gene therapy clinical trials and promising preclinical developments for IRDs.