Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice-Reference-Cited by-同舟云学术

Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

Published:2019-12-11 Issue:1-2 Volume:27 Page:74-84
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Schuh Roselena Silvestri^ORCID,Gonzalez Esteban Alberto^ORCID,Tavares Angela Maria Vicente,Seolin Bruna Gazzi,Elias Lais de Souza,Vera Luisa Natalia Pimentel,Kubaski Francyne,Poletto Edina,Giugliani Roberto,Teixeira Helder Ferreira,Matte Ursula,Baldo Guilherme

Funder

Ministry of Science, Technology and Innovation | Conselho Nacional de Desenvolvimento Científico e Tecnológico

Federal University of Rio Grande do Sul | Pró-Reitoria de Pesquisa, Universidade Federal do Rio Grande do Sul

Coordenação de Aperfeiçoamento de Pessoal de Nível Superior

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/s41434-019-0113-4.pdf

Reference49 articles.

1. Giugliani R, Federhen A, Rojas MVM, Vieira T, Artigalás O, Pinto LL, et al. Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment. Genet Mol Biol. 2010;33:589–604.