Oligonucleotide compositions and methods thereof
By designing oligonucleotides with specific structures, the problems of low exon jumping efficiency and high toxicity of oligonucleotides in the treatment of DMD in the prior art have been solved. This has achieved efficient and stable exon 52 jumping and reduced toxicity, and restored the expression of some functional dystrophin proteins.
CN122295443APending Publication Date: 2026-06-26WAVE LIFE SCI LTD
Patent Information
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- WAVE LIFE SCI LTD
- Filing Date
- 2024-09-27
- Publication Date
- 2026-06-26
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Figure CN122295443A_ABST
Abstract
This disclosure provides, in particular, oligonucleotide compositions and methods thereof. In some embodiments, the oligonucleotides comprise various chemical modifications of sugars, nucleobases, and / or internucleotide bonds and their patterns, and are usable for exon skipping. In some embodiments, this disclosure provides techniques for use in exon 52 of jumping dystrophin (DMD) transcripts. In some embodiments, this disclosure provides techniques for modulating DMD transcript splicing. In some embodiments, this disclosure provides methods for preventing or treating conditions, disorders, or diseases including Duchenne muscular dystrophy.
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