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OLIGOMERS
专利权人:
Linda POPPLEWELL;John George DICKSON;Ian GRAHAM
发明人:
Linda POPPLEWELL,Ian GRAHAM,John George DICKSON
申请号:
US13307825
公开号:
US20120108652A1
申请日:
2011.11.30
申请国别(地区):
US
年份:
2012
代理人:
摘要:
Molecules are provided for inducing or facilitating exon skipping in forming spliced mRNA products from pre-mRNA molecules in cells. The molecules may be provided directly as oligonucleotides or expression products of vectors that are administered to a subject. High rates of skipping can be achieved. High rates of skipping reduce the severity of a disease like Duchene Muscular Dystrophy so that the disease is more like Becker Muscular Dystrophy. This is a severe reduction in symptom severity and mortality.
来源网站:
中国工程科技知识中心
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http://www.ckcest.cn/home/

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