CRISPR-Mediated Correction of ABCA4 Mutations in Stem Cells May Provide Treatment for Incurable Inherited Disease Causing Vision Loss
Stargardt’s disease is an inherited macular neurodegenerative disorder that causes retinal dystrophy and vision loss. Stargardt disease is caused by mutations in the ABCA4 gene, which encodes a protein involved in the visual cycle and the transport of toxic photoproducts out of the retina. Currently, there is no treatment for Stargardt’s disease, and although restoring […]