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

Baldo, G (通讯作者)，Hosp Clin Porto Alegre, Ctr Terapia Gen, R Ramiro Barcelos 2350, BR-90035903 Porto Alegre, RS, Brazil.

JOURNAL OF GENE MEDICINE, 2022; 24 (4):

Abstract

Background Mucopolysaccharidosis type I (MPS I) is an inherited disease caused by deficiency of the enzyme alpha-l-iduronidase (IDUA). MPS I affects s......

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