Oligomers
First Claim
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1. A method of ameliorating Duchenne muscular dystrophy, the method comprising administering at least one oligomer to a patient, wherein the at least one oligomer comprises a sequence of:
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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.
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14 Claims
- 1. A method of ameliorating Duchenne muscular dystrophy, the method comprising administering at least one oligomer to a patient, wherein the at least one oligomer comprises a sequence of:
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