Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice
Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice
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London: Nature Publishing Group UK
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English
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London: Nature Publishing Group UK
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Current gene therapy for Duchenne muscular dystrophy (DMD) utilizes adeno-associated virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for striated muscles as it lacks many important functional domains of full-length (FL) dystrophin. Here we develop a triple vector system to deliver FL-dystrophin into skeletal a...
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Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice
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TN_cdi_doaj_primary_oai_doaj_org_article_99a49e53f4014a01a4aca707caf156f6
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_99a49e53f4014a01a4aca707caf156f6
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ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-024-50569-6
