The future of retinal gene therapy: evolving from subretinal to intravitreal vector delivery.

ABSTRACT: Inherited retinal degenerations are a leading and untreatbale cause of blindness, and as such they are targets for gene therapy. Numerous gene therapy treatments have progressed from laboratory research to clinical trails, and a pioneering gene therapy received the first ever FDA approval for treating patients. However, currently retinal gene therapy mostly involves subretinal injection of the therapeutic agent, which treats a limited area, entails retinal detachment and other potential complications, and requires general anesthesia with consequent risks, costs and prolonged recovery. Therefore there is great impetus to develop safer, less invasive and cheapter methods of gene delivery. A promising method is intravitreal injection, that does not cause retinal detachment, can lead to pan-re

SUBMITTER: Ross M 

PROVIDER: S-EPMC8328774 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature