Oligomers
OwnerLinda Popplewell, Ian Graham +1inventors
InventorsLinda Popplewell, Ian Graham, John George Dickson
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Abstract
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.
Claims14
An oligomer for ameliorating DMD, the oligomer comprising a base sequence selected from the group consisting of:
- (SEQ ID NO:
- 6)
- a) XGC XGC XCX XXX CCA GGX XCA AGX GGG AXA;
- (SEQ ID NO:
- 7)
- b) CXX XXA GXX GCX GCX CXX XXC CAG GXX CAA;
- (SEQ ID NO:
- 8)
- c) CXX XXC XXX XAG XXG CXG CXC XXX XCC AGG;
- and
- (SEQ ID NO:
- 9)
- d) XXA GXX GCX GCX CXX XXC CAG GXX CAA GXG;
- wherein X=U or T, wherein the oligomer's base sequence can vary from the above sequence at up to two base positions, and wherein the molecule can bind to a target site to cause exon skipping in an exon of the dystrophin gene.
Dependencies are inferred from the claim text.