Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a ho...
Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism
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Publisher
New York: Nature Publishing Group US
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Language
English
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Publisher
New York: Nature Publishing Group US
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Contents
An HDR-independent therapeutic genome-editing approach corrected the splice-site mutation in
Lama2
in a mouse model of congenital muscular dystrophy type 1A, and may be applied more broadly to correct splice-site mutations associated with other diseases.
Splice-site defects account for about 10% of pathogenic mutations that cause Mendelian...
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Full title
Correction of a splicing defect in a mouse model of congenital muscular dystrophy type 1A using a homology-directed-repair-independent mechanism
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TN_cdi_proquest_miscellaneous_1920194241
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1920194241
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ISSN
1078-8956
E-ISSN
1546-170X
DOI
10.1038/nm.4367
