The new landscape of retinal gene therapy
Author:
Affiliation:
1. Casey Eye Institute Oregon Health & Science University Portland Oregon USA
Publisher
Wiley
Subject
Genetics(clinical),Genetics
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/ajmg.c.31842
Reference140 articles.
1. Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness
2. Rod-Derived Cone Viability Factor Promotes Cone Survival by Stimulating Aerobic Glycolysis
3. Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
4. The human visual cortex responds to gene therapy–mediated recovery of retinal function
5. The intronicABCA4c.5461-10T>C variant, frequently seen in patients with Stargardt disease, causes splice defects and reduced ABCA4 protein level
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