Groundbreaking CRISPR Treatment for Blindness Only Works for Subset of Patients

November 22, 2022

(Science) – After some early but cautious optimism, a company is shelving its pioneering gene-editing treatment for a rare inherited blindness disorder. Editas Medicine announced today the trial trying to use the gene editor CRISPR to treat Leber congenital amaurosis 10 (LCA10) led to “clinically meaningful” vision improvements in only three of 14 patients. In the study, patients received a subretinal injection of a modified virus carrying genetic material encoding components of CRISPR, a DNA-cleaving enzyme, and two RNA strands to guide the protein to its target sequences. (Read More)