Article
CRISPR-Cas9-mediated gene editing in human MPS I fibroblasts.
Gene - 15 Dec 2018
de Carvalho Talita Giacomet, Schuh Roselena, Pasqualim Gabriela, Pellenz Felipe Matheus, Filippi-Chiela Eduardo Cremonese, Giugliani Roberto, Baldo Guilherme, Matte Ursula
Abstract excerpt
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder (LSD). It is caused by mutations in the IDUA gene, which lead to the accumulation of the glycosaminoglycans dermatan and heparan sulfate. The CRISPR-Cas9 system is a new and powerful tool that allows gene editing at precise points of the genome, resulting in gene correction through the introduction and genomic integration of a wildtype sequence....
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