Precision gene editing could prevent vision loss from Stargardt disease
Researchers have developed a therapy to treat Stargardt disease, the most common form of inherited macular degeneration, ...
AZoLifeSciences on MSN5d
Breakthrough Gene Editing Technique Offers Hope for Stargardt Disease Patients
Rewriting genes to preserve eyesight IOB and Beam Therapeutics develop efficient gene editing technique for Stargardt disease ...
Insmed’s gene therapy poised to challenge DMD landscape after IND clearance
Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved ...
GEN5d
Base Editing Addresses Inherited Macular Degeneration in Primate, Human Tissue Models
The adenine base editor corrected the most common mutation associated with Stargardt disease in the ABCA4 gene with no off-target effects.
Medical Xpress5d
Precision gene editing could prevent vision loss from Stargardt disease
Stargardt disease affects around one in 6,500 people and is therefore a rare disease. Those affected increasingly lose the ability to see in the center of their field of vision, which makes it ...