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Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
Duchenne muscular dystrophy (DMD) is a severe muscle-degenerative disease caused by a mutation in the dystrophin gene. Genetic correction of patient-derived induced pluripotent stem cells (iPSCs) by TALENs or CRISPR-Cas9 holds promise for DMD gene therapy; however, the safety of such nuclease treatm...
Αποθηκεύτηκε σε:
Τόπος έκδοσης: | Stem Cell Reports |
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Κύριοι συγγραφείς: | , , , , , , , , , , , , |
Μορφή: | Artigo |
Γλώσσα: | Inglês |
Έκδοση: |
Elsevier
2014
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Θέματα: | |
Διαθέσιμο Online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4297888/ https://ncbi.nlm.nih.gov/pubmed/25434822 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2014.10.013 |
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