Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
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Title
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
Authors
Keywords
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Journal
GENE THERAPY
Volume 27, Issue 1-2, Pages 74-84
Publisher
Springer Science and Business Media LLC
Online
2019-12-11
DOI
10.1038/s41434-019-0113-4
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