Effective gene therapy of Stargardt disease with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles.
Sun D, Sun W, Gao SQ, Lehrer J, Naderi A, Wei C, Lee S, Schilb AL, Scheidt J, Hall RC, Traboulsi EI, Palczewski K, Lu ZR.
Sun D, et al.
Mol Ther Nucleic Acids. 2022 Aug 24;29:823-835. doi: 10.1016/j.omtn.2022.08.026. eCollection 2022 Sep 13.
Mol Ther Nucleic Acids. 2022.
PMID: 36159595
Free PMC article.