Functional recovery of a novel knockin mouse model of dysferlinopathy by readthrough of nonsense mutation
Biallelic mutations in the dysferlin gene cause limb-girdle muscular dystrophy 2B or Miyoshi distal myopathy. We found that nonsense mutations are the most common mutation type among Korean patients with dysferlinopathy; more than half of the patients have at least one nonsense allele, which may be...
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| Hlavní autoři: | , , , , |
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| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
Elsevier
2021-06-01
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| Edice: | Molecular Therapy: Methods & Clinical Development |
| Témata: | |
| On-line přístup: | http://www.sciencedirect.com/science/article/pii/S2329050121000814 |
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