Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system.
Vera LNP, Schuh RS, Fachel FNS, Poletto E, Piovesan E, Kubaski F, Couto E, Brum B, Rodrigues G, Souza H, Giugliani R, Matte U, Baldo G, Teixeira HF.
Vera LNP, et al. Among authors: souza h.
J Gene Med. 2022 Apr;24(4):e3410. doi: 10.1002/jgm.3410. Epub 2022 Jan 31.
J Gene Med. 2022.
PMID: 35032067