Preclinical evaluation of ADVM-062, a novel intravitreal gene therapy vector for the treatment of blue cone monochromacy
K Hanna, J Nieves, C Dowd, KO Bender, P Sharma… - Molecular Therapy, 2023 - cell.com
Blue cone monochromacy (BCM) is a rare X-linked retinal disease characterized by the
absence of L-and M-opsin in cone photoreceptors, considered a potential gene therapy …
absence of L-and M-opsin in cone photoreceptors, considered a potential gene therapy …