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Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
Duchenne muscular dystrophy (DMD) is an X-linked progressive disease characterized by loss of dystrophin protein that typically results from truncating mutations in the DMD gene. Current exon-skipping therapies have sought to treat deletion mutations that abolish an open reading frame (ORF) by skipp...
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| 發表在: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
American Society of Gene & Cell Therapy
2021
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8047432/ https://ncbi.nlm.nih.gov/pubmed/33898631 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.03.014 |
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