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rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy
X-linked myotubular myopathy (XLMTM) is a life-threatening skeletal muscle disease caused by mutations in the MTM1 gene. XLMTM fibres display a population of nuclei mispositioned in the centre. In the present study, we aimed to explore whether positioning and overall distribution of nuclei affects c...
保存先:
| 出版年: | Acta Neuropathol Commun |
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| 主要な著者: | , , , , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
BioMed Central
2020
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7574461/ https://ncbi.nlm.nih.gov/pubmed/33076971 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s40478-020-01048-8 |
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