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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]
There is no cure for most causes of color blindness; however there is ongoing research into gene therapy for some severe conditions causing color blindness. [2] Minor forms of color blindness do not significantly affect daily life and the color blind automatically develop adaptations and coping mechanisms to compensate for the deficiency. [ 2 ]
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
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Despite much recent improvement in Gene therapy for color blindness, there is currently no FDA approved treatment for congenital red–green color blindness, and otherwise no cure exists. Management of the condition through the use of color blind glasses to alleviate symptoms or smartphone apps to aid with daily tasks is possible.
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).
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million ...
As achromatopsia is linked to only a few single-gene mutations, it is a good candidate for gene therapy. Gene therapy is a technique for injecting functional genes into the cells that need them, replacing or overruling the original alleles linked to achromatopsia, thereby curing it – at least in part. Achromatopsia has been a focus of gene ...