Targeted insertion of an optimized donor DNA is effective in a humanized mouse model of dominant retinitis pigmentosa.
Retinitis pigmentosa (RP) affects 1 in 3,000 individuals worldwide, with 30%-40% of cases inherited as autosomal dominant (AD). Mutations in RHO (RP4) are the most common cause of ADRP. Because most RHO mutations exert gain-of-function or dominant-negative effects, conventional gene supplementation is insufficient, req...
F. Esposito, Arjun Padmanabhan, M. Rhiel et al.
· Cell Reports Medicine · 0 citations