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Oligomers
专利权人:
Royal Holloway; University of London
发明人:
Linda Popplewell,Ian Graham,John George Dickson
申请号:
US15466631
公开号:
US09970010B2
申请日:
2017.03.22
申请国别(地区):
US
年份:
2018
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.
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