期刊
GENE THERAPY
卷 19, 期 7, 页码 781-785出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/gt.2011.193
关键词
allele-specific RNAi; siRNA; FOP; ALK2; BMP signaling; adverse effects
类别
资金
- Ministry of Health, Labour and Welfare of Japan
- National Hospital Organization
- Japan Society for the Promotion of Science
- Grants-in-Aid for Scientific Research [24592278, 21390423, 23659732] Funding Source: KAKEN
Fibrodysplasia ossificans progressiva (FOP) is an autosomal dominant congenital disorder characterized by progressive heterotopic bone formation. Currently, no definitive treatment exists for FOP. The activin receptor type IA / activin-like kinase 2 (ACVR1/ALK2) gene has been identified as the responsible gene for FOP, and disease-associated ALK2 mutations have been found. Chemical inhibitors to the pathogenic ALK2 receptors are considered possible medical agents for FOP, but their adverse effects on normal ALK2 and other receptors cannot be excluded. Here we describe another treatment strategy for FOP using allele-specific RNA interference (ASP-RNAi), and show modified small interfering RNAs (siRNAs) conferring allele-specific silencing against disease-causing ALK2 mutants found in FOP, without affecting normal ALK2 allele. Thus, the siRNAs presented here may become novel therapeutic agents for FOP, and their induced ASP-RNAi may pave the way for the achievement of radical treatment of FOP and/or for the relief of its severe symptoms. Gene Therapy (2012) 19, 781-785; doi:10.1038/gt.2011.193; published online 1 December 2011
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