Invention Grant
US07964570B2 Methods and compositions for treatment of myotonia 有权
用于治疗肌强直的方法和组合物

Methods and compositions for treatment of myotonia
Abstract:
The present invention provides methods and compositions for the treatment of diseases associated with aberrant microsatellite expansions. Methods of the present invention comprise the use of recombinant adeno-associated virus vectors containing a transgene encoding at least one muscleblind protein. The present invention also provides an animal model for a disease associated with aberrant microsatellite expansion.
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