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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.
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).
For her entire life, college student Olivia Cook had only a small degree of central vision. It was as if she was watching the world through a straw hole, and in dimly lit places, she could not ...
Her laboratory developed the first FDA approved gene therapy for use in humans, which treats a rare form of blindness. She was elected a member of the National Academy of Sciences in 2022. [ 1 ]
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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 ...
Developments in the field of gene therapy are being studied to be included in the scope of this treatment, but of course more research is needed to increase the positive results and minimize the negative effects of gene therapy applications. [27] The CRISPR/Cas9 system is also designed as a gene editing technology for the treatment of HIV-1/AIDS.