Oligonucleotide comprising an inosine for treating DMD
OwnerJudith Christina Theodora van Deutekom, Josephus Johannes de Kimpe +1inventors
InventorsJudith Christina Theodora van Deutekom, Josephus Johannes de Kimpe, Gerard Johannes Platenburg
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Abstract
The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the oligonucleotide, or a functional equivalent thereof, comprises a sequence which is complementary to at least part of a dystrophin pre-m RNA exon or at least part of a non-exon region of a dystrophin pre-m RNA said part being a contiguous stretch comprising at least 8 nucleotides. The invention further provides the use of said oligonucleotide for preventing or treating DMD or BMD.
Claims9
An oligonucleotide consisting of the base sequence of any one of SEQ ID NO:
- 141, 142 or 144-188, wherein at least one guanosine base in the sequence is substituted with an inosine base, and wherein said oligonucleotide comprises a modification and induces skipping of exon 53 of human dystrophin pre-mRNA.
Dependencies are inferred from the claim text.