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

Retinitis pigmentosa (RP) is the most common cause of inherited blindness in adults. Mutations in the PRPF31 gene produce autosomal dominant RP (adRP). To date there are no effective treatments for this disease. The purpose of this study was to design an efficient non-viral vector for human PRPF31 gene delivery as an approach to treat this form of adRP. Span based nanoparticles were developed to mediate gene transfer in the subretinal space of a mouse model of adRP carrying a point mutation (A216P) in the Prpf31 gene. Funduscopic examination, electroretinogram, optomotor test and optical coherence tomography were conducted to further in vivo evaluate the safety and efficacy of the nanosystems developed. Span-polyarginine (SP-PA) nanoparticles were able to efficiently transfect the GFP and PRPF31 plasmid in mice retinas. Statistically significant improvement in visual acuity and retinal thickness were found in Prpf31 mice treated with the SP-PA-PRPF31 nanomedicine.This work was supported by grants of the Ministry of Economy and Competitiveness of Spain (MAT2013-47501-C2-2-R) and Xunta de Galicia (Competitive Reference Groups, FEDER Funds, Ref. 2014/043). Grants for Biomedical Research Funding and Health Sciences in Andalusia (PI-0084-2013) and The Spanish National Research Council (CSIC) (P09-CT5-4967).Peer Reviewe