Activating internal ribosome entry to treat Duchenne muscular dystrophy

作者：Lamande Shireen R^*; North Kathryn N

来源：Nature Medicine, 2014, 20(9): 987-988.

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.

• 出版日期2014-9