Article
Span poly-L-arginine nanoparticles are efficient non-viral vectors for PRPF31 gene delivery: An approach of gene therapy to treat retinitis pigmentosa.
Nanomedicine : nanotechnology, biology, and medicine - 1 Nov 2016
Pensado Andrea, Diaz-Corrales Francisco J, De la Cerda Berta, Valdés-Sánchez Lourdes, Del Boz Ana Aramburu, Rodriguez-Martinez Daniel, García-Delgado Ana B, Seijo Begoña, Bhattacharya Shomi S, Sanchez Alejandro
Abstract excerpt
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...
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