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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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Опубликовано в: :Hum Gene Ther Methods
Главные авторы: Nalbandian, Angèle, Llewellyn, Katrina J., Nguyen, Christopher, Monuki, Edward S., Kimonis, Virginia E.
Формат: Artigo
Язык:Inglês
Опубликовано: Mary Ann Liebert, Inc. 2015
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Online-ссылка: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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