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Exon Skipping in a Dysf-Missense Mutant Mouse Model
Limb girdle muscular dystrophy 2B (LGMD2B) is without treatment and caused by mutations in the dysferlin gene (DYSF). One-third is missense mutations leading to dysferlin aggregation and amyloid formation, in addition to defects in sarcolemmal repair and progressive muscle wasting. Dysferlin-null mo...
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| Publicado no: | Mol Ther Nucleic Acids |
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| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6172476/ https://ncbi.nlm.nih.gov/pubmed/30292141 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.08.013 |
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