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Article

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

1 Nov 2018

Abstract excerpt

Mucopolysaccharidosis type I (MPS I) is a severe disease due to deficiency of the lysosomal hydrolase α-L-iduronidase (IDUA) and the subsequent accumulation of the glycosaminoglycans (GAG), leading to progressive, systemic disease and a shortened lifespan. Current treatment options consist of hematopoietic stem cell transplantation, which carries significant mortality and morbidity risk, and enzyme replacement...

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Identifiers

PMID: 30528089
DOI: 10.1016/j.ymthe.2018.10.018

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