In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
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Publisher
United States: American Association for the Advancement of Science
Journal title
Language
English
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Publisher
United States: American Association for the Advancement of Science
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Scope and Contents
Contents
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome editing has the potential to restore expression of a modified dystrophin gene from the native locus to modulate disease progression. In this study, adeno-associated virus was used to deliver the...
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Full title
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4883596
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4883596
Other Identifiers
ISSN
0036-8075
E-ISSN
1095-9203
DOI
10.1126/science.aad5143
