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The first retinal gene therapy to be approved by the FDA was Voretigene neparvovec in 2017, which treats Leber's congenital amaurosis, a genetic disorder that can lead to blindness. These treatments also use subretinal injections of AAV vector and are therefore foundational to research in gene therapy for color blindness. [2] [3]
This groundbreaking study, published in Nature Medicine on 8 January 2025, demonstrates the therapeutic use of a precision gene editing technology called base editing. Stargardt disease affects 1 in 6,500 individuals and often results in legal blindness. There is currently no effective treatment available.
Gene therapy is a general treatment for genetic disorders; it uses viral vectors to carry typical genes into cells (e.g. cone cells) that are not able to express functional genes (e.g. photopsins). It may be possible to restore color vision by adding missing opsin genes – or a functional copy of the entire gene complex – into the cone cells.
Retinal gene therapy holds a promise in treating different forms of non-inherited and inherited blindness. In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's congenital amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV).
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The episode contains some information about symptoms, clinical diagnosis and mentions gene replacement therapy and clinical trials as hope for help in managing the condition. [ citation needed ] In the Korean drama The King of Dramas (Episode 16, "In Search of Lost Time") Anthony Kim, played by Kim Myung-min , is diagnosed with Leber congenital ...
This is one of the first studies of a CRISPR-based in vivo human gene editing therapy, where the editing takes place inside the human body. [266] The first injection of the CRISPR-Cas System was confirmed in March 2020. [267] Exagamglogene autotemcel, a CRISPR-based human gene editing therapy, was used for sickle cell and thalassemia in ...
Many other possible viral vectors remain options for the treatment of various genetic disorders in the retina that lead to blindness. Retinal gene therapy using lentivirus vectors may be a way to treat a wider range of genetic disorders in the retina because of the various properties of the lentivirus that make it an attractive alternative to ...