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Targeted Excision of VCP R155H Mutation by Cre-LoxP Technology as a Promising Therapeutic Strategy for Valosin-Containing Protein Disease
Inclusion body myopathy associated with Paget's disease of the bone and frontotemporal dementia is attributed to mutations in the valosin-containing protein (VCP) gene, mapped to chromosomal region 9p13.3–12. Affected individuals exhibit scapular winging and die from progressive muscle weakness...
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| Publicado en: | Hum Gene Ther Methods |
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| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Mary Ann Liebert, Inc.
2015
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4337464/ https://ncbi.nlm.nih.gov/pubmed/25545721 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hgtb.2014.096 |
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