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Journal Article
Molecular Therapy - Nucleic Acids, ISSN 2162-2531, 06/2017, Volume 7, Issue C, pp. 11 - 19
Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR... 
dystrophin | CRISPR/Cas9 | gene editing | duplication | lentivirus | MEDICINE, RESEARCH & EXPERIMENTAL | CELLS | RESTORES DYSTROPHIN EXPRESSION | GENE | MOUSE MODEL | MUSCULAR-DYSTROPHY | MUSCLE | DUCHENNE | MUTATIONS | REARRANGEMENTS | NUCLEASES | CRISPR | Cas9 | Original
Journal Article
The EMBO Journal, ISSN 0261-4189, 04/2017, Volume 36, Issue 8, pp. 981 - 994
Journal Article
PLoS ONE, ISSN 1932-6203, 01/2013, Volume 8, Issue 1, p. e55721
Journal Article
Genes, ISSN 2073-4425, 2014, Volume 5, Issue 2, pp. 415 - 429
Journal Article
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