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US 2025/0179495 A1Published application
Antisense polynucleotides to induce exon skipping and method of treating dystrophies
AssigneeThe University of Chicago
InventorElizabeth McNally
Life of this patent
Estimating the term…
Abstract
Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy.
Claims12
An isolated antisense polynucleotide wherein the polynucleotide specifically hybridizes to an exon target region of a gamma sarcoglycan RNA, wherein the exon is selected from the group consisting of exon 4 (SEQ ID NO:
- 1), exon 5 (SEQ ID NO:
- 2), exon 6 (SEQ ID NO:
- 3), exon 7 (SEQ ID NO:
- 4) and a combination thereof.
1 more element
The antisense polynucleotide of claim 5, wherein the modified moiety is a Morpholino.
Dependencies are inferred from the claim text.