CA2957661A1 — Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
Assigned to University of Western Australia · Expires 2016-02-18 · 10y expired
What this patent protects
The present invention relates to the delivery of oligomers for treating patients with a 5' mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulti…
USPTO Abstract
The present invention relates to the delivery of oligomers for treating patients with a 5' mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulting in translation of a functional truncated isoform of dystrophin. The methods and materials can be used for the treatment of muscular dystrophies arising from 5' mutations in the DMD gene such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.
Drugs covered by this patent
- Amondys 45 (CASIMERSEN) · Sarepta
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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