טוען...
Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder caused by reading frame disrupting mutations in the DMD gene. Exon skipping is a therapeutic approach for DMD. It employs antisense oligonucleotides (AONs) to restore the disrupted open reading frame, allowing the pro...
שמור ב:
| הוצא לאור ב: | Mol Ther |
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| Main Authors: | , , , , , , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
American Society of Gene & Cell Therapy
2018
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| נושאים: | |
| גישה מקוונת: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5763161/ https://ncbi.nlm.nih.gov/pubmed/29103911 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.10.004 |
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