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

Gene Therapy, Published online: 11 December 2019; doi:10.1038/s41434-019-0113-4Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

Source: Gene Therapy - December 10, 2019 Category: Genetics & Stem Cells Authors: Roselena Silvestri Schuh Esteban Alberto Gonzalez Angela Maria Vicente Tavares Bruna Gazzi Seolin Lais de Souza Elias Luisa Natalia Pimentel Vera Francyne Kubaski Edina Poletto Roberto Giugliani Helder Ferreira Teixeira Ursula Matte Guilherme Baldo Source Type: research