Oligonucleotides matching col7a1 exon 73 for epidermolysis bullosa therapy
AssigneeWings Therapeutics, Inc.
InventorsElisabeth Marlene Haisma, Marko Potman, Wouter Beumer, Vera Brinks
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AssigneeWings Therapeutics, Inc.
InventorsElisabeth Marlene Haisma, Marko Potman, Wouter Beumer, Vera Brinks
Estimating the term…
Antisense oligonucleotides capable of preventing or reducing exon 73 inclusion into the human COL7A mRNA are characterized in various ways: (a) the oligonucleotide's sequence includes at most two CpG sequences; (b) the oligonucleotide has a length of no more than 24 nucleotides; (c) the oligonucleotide is capable of annealing to the (SRp40/SC35 binding/ESE) element in exon73. These oligonucleotides can usefully be oligoribonucleotides with modified internucleosidic linkages e.g. phosphorothioate linkages.
1-15. (canceled)
An antisense oligoribonucleotide capable of preventing or reducing exon 73 inclusion into a human collagen type VII alpha 1 chain (COL7A1) mRNA when the mRNA is produced by splicing from a pre-mRNA in a human cell, wherein the oligoribonucleotide comprises a nucleotide sequence that is complementary to a target sequence in a COL7A1 pre-mRNA corresponding to SEQ ID NO:
Dependencies are inferred from the claim text.