Open AccessActivating internal ribosome entry to treat Duchenne muscular dystrophy
Author(s) -
Shireen R. Lamandé,
Klaus North
Publication year - 2014
Publication title -
nature medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 19.536
H-Index - 547
eISSN - 1546-170X
pISSN - 1078-8956
DOI - 10.1038/nm.3677
Subject(s) - duchenne muscular dystrophy , dystrophin , internal ribosome entry site , translation (biology) , muscular dystrophy , medicine , genetics , mutation , gene , biology , bioinformatics , messenger rna
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.
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