METHODS AND COMPOSITIONS FOR TREATMENT OF DISEASES ASSOCIATED WITH ABERRANT MICROSATELLITE EXPANSION

摘要

The present invention provides methods and compositions for the treatment of diseases associated with aberrant microsatellite expansions. Methods of the present invention comprise 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.