Activating internal ribosome entry to treat Duchenne muscular dystrophy.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 25198047.
- Also identified by DOI 10.1038/nm.3677.
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Abstract
Mutations in the DMD gene, encoding dystrophin, cause the most common forms of muscular dystrophy. A new study shows that forcing translation of DMD from an internal ribosome entry site can alleviate Duchenne muscular dystrophy symptoms in a mouse model.
Medical subject headings
- Dystrophin
- Exons
- Muscular Dystrophy, Duchenne
- Protein Biosynthesis
- Protein Isoforms