POLY(Beta-AMINO ESTER) NANOPARTICLES FOR THE NON-VIRAL DELIVERY OF PLASMID DNA FOR GENE EDITING AND RETINAL GENE THERAPY

摘要

Biodegradable particles for delivering a nucleic acid encoding gene-editing factors or a nucleic acid associated with a therapeutic protein to a cell, and compositions, methods, systems, and kits for gene editing in vivo or ex vivo or gene therapy for treating retinal eye diseases are disclosed.