Ocular Gene Therapy: A Literature Review With Focus on Current Clinical Trials.

ABSTRACT: Gene therapy has been one of the most researched topics in the last decade. It has now become a revolutionized therapeutic tool of modern medicine. Gene therapy is the alteration of the defective gene involved in the disease process in the host cells. It delivers therapeutic genetic information via modified viral or non-viral vectors. Ocular gene therapy, in particular, has progressed in treating inherited retinal diseases since the eye is a favourable organ for gene therapy development. The advantage of the eye as a target for gene therapy is attributed to its easy accessibility and blood-ocular barrier. Several ongoing clinical trials are investigating various gene therapies for other ocular diseases, including neovascular age-related macular degeneration, retinitis pigmentosa (RP), Ushe

SUBMITTER: Wasnik VB 

PROVIDER: S-EPMC9590687 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature