Researchers developed and phenotyped a pigmented knockout rat model for lecithin retinol acyltransferase (LRAT) using CRISPR/Cas9.

RPE65-related LCA pivotal trials, . . . have paved the way for a new era of genetic treatments in ophthalmology.

Sawar Zahid MS, MD, Kari Branham MS, CGC, Dana Schlegel MS, MPH, CGC, Mark E. Pennesi MD, PhD, Michel Michaelides MB, MD, John...

Phenotypic similarities to the retinal dysfunction assoc. with RPE65 mutations, suggest that LRAT deficiency may respond to novel therapies.