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.
申请公布号
US8461325(B2)
申请公布日期
2013.06.11
申请号
US201113307825
申请日期
2011.11.30
申请人
POPPLEWELL LINDA;GRAHAM IAN;DICKSON JOHN GEORGE;ROYAL HOLLOWAY, UNIVERSITY OF LONDON