Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gen...
Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy
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United States: National Academy of Sciences
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Language
English
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United States: National Academy of Sciences
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Contents
In gene therapy for Duchenne muscular dystrophy there are two potential immunological obstacles. An individual with Duchenne muscular dystrophy has a genetic mutation in dystrophin, and therefore the wild-type protein is “foreign,” and thus potentially immunogenic. The adeno-associated virus serotype-6 (AAV6) vector for delivery of dystrophin is a...
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Full title
Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6166850
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6166850
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ISSN
0027-8424
E-ISSN
1091-6490
DOI
10.1073/pnas.1808648115