<i>In vivo</i> adenine base editing corrects newborn murine model of Hurler syndrome

Abstract excerpt

Mucopolysaccharidosis type I (MPS I) is a severe disease caused by loss-of-function mutations variants in the α-L-iduronidase ( IDUA ) gene. In vivo genome editing represents a promising strategy to correct IDUA mutations, and has the potential to permanently restore IDUA function over the lifespan of the patients. Here, we used adenine base editing to directly convert A>G (TAG>TGG) in newborn murine model harb...

Topics

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• Autism Spectrum Disorder Research
• CRISPR and Genetic Engineering
• Disease Models, Animal
• Hepatocytes
• Lysosomal Storage Diseases
• Lysosomal Storage Disorders Research
• Mutation
• Myocytes, Cardiac

Identifiers and source

Literature Corpus work

6c35614e-83d9-5542-aed7-04de31e6cbd3

DOI

10.1101/2021.10.16.464213