Ascidian Therapeutics is developing therapies for inherited disorders that work by editing RNA with an approach that could offer advantages over currently available genetic medicines. The biotech’s lead program is in preclinical development for Stargardt disease, a rare eye disorder caused by multiple genetic mutations.
Kathy Bleitz suffers from a condition called Stargardt disease and has been legally blind since the age of 11. It’s an inherited degenerative disease that significantly reduces vision in the central part of the retina, but peripheral vision is sometimes retained. IFLScience explained how some new technology actually allowed Bleitz to see her new baby: […]
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