Oligomers
AssigneeRoyal Holloway, University of London
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.
Claims20
An oligomer for ameliorating DMD, the oligomer comprising at least 25 contiguous bases of a base sequence selected from the group consisting of:
- a) CAG XXX GCC GCX GCC CAA XGC CAX CCX GGA (SEQ ID NO:
- 4) and b) XXG CCG CXG CCC AAX GCC AXC CXG GAG XXC (SEQ ID NO:
- 5);
- wherein X=U or T, wherein the sequence of the at least 25 contiguous bases can vary from SEQ ID NO:
- 4 or 5 at up to two base positions, and wherein the oligomer comprises one or more synthetically modified nucleotides.
Dependencies are inferred from the claim text.