Ontology highlight
ABSTRACT:
SUBMITTER: Sun D
PROVIDER: S-EPMC6952167 | biostudies-literature | 2020 Jan
REPOSITORIES: biostudies-literature
Sun Da D Schur Rebecca M RM Sears Avery E AE Gao Song-Qi SQ Vaidya Amita A Sun Wenyu W Maeda Akiko A Kern Timothy T Palczewski Krzysztof K Lu Zheng-Rong ZR
Molecular therapy : the journal of the American Society of Gene Therapy 20190912 1
Stargardt disease (STGD) is an autosomal recessive retinal disorder caused by a monogenic ABCA4 mutation. Currently, there is no effective therapy to cure Stargardt disease. The replacement of mutated ABCA4 with a functional gene remains an attractive strategy. In this study, we have developed a non-viral gene therapy using nanoparticles self-assembled by a multifunctional pH-sensitive amino lipid ECO and a therapeutic ABCA4 plasmid. The nanoparticles mediated efficient intracellular gene transd ...[more]