लोड हो रहा है...
CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients
Myotonic dystrophy type 1 (DM1) is the most common adult-onset muscular dystrophy, characterized by progressive myopathy, myotonia, and multi-organ involvement. This dystrophy is an inherited autosomal dominant disease caused by a (CTG)n expansion within the 3′ untranslated region of the DMPK gene....
में बचाया:
| में प्रकाशित: | Mol Ther Nucleic Acids |
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| मुख्य लेखकों: | , , , , , , , |
| स्वरूप: | Artigo |
| भाषा: | Inglês |
| प्रकाशित: |
American Society of Gene & Cell Therapy
2017
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| विषय: | |
| ऑनलाइन पहुंच: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5684470/ https://ncbi.nlm.nih.gov/pubmed/29246312 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.10.006 |
| टैग : |
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