Span poly-L-arginine nanoparticles are efficient non-viral vectors for PRPF31 gene delivery: An approach of gene therapy to treat retinitis pigmentosa

Subretinal injection of SP-PA-PRPF31 nanoparticles in heterozygous knock-in (KI) Prpf31A216P/+ mice. The retinal thickness measured by optical coherence tomography (OCT) in KI Prpf31A216P/+ mice treated with SP-PA-PRPF31nanoparticles was a similar value to wild type mice. These nanomedicines not only improved visual function but also retarded or reversed retinal degeneration in KI Prpf31A216P/+ mice, evidenced by the absence of retinal atrophy.161