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US 9,499,818 B2Granted
Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene
AssigneesBioMarin Technologies B.V., Academisch Ziekenhuis Leiden
InventorJudith Christina Theodora van Deutekom
Life of this patent
Estimating the term…
Abstract
The invention relates to a method where a molecule is used for inducing and/or promoting the skipping of at least one of the exons 51-53, 55, 57 and 59 of the DMD pre-mRNA in a patient, preferably in an isolated cell of a patient. The method comprising providing the cell and/or the patient with a molecule. The invention also relates to the molecule and its composition that is being used for inducing exon skipping.
Claims17
An isolated antisense oligonucleotide whose base sequence consists of the base sequence 5′-GGUAAUGAGUUCUUCCAACUGG-3′ (SEQ ID NO:
- 287), said oligonucleotide comprising a modification.
Dependencies are inferred from the claim text.