HSA LR Mice (Mouse Model of Myotonic Dystrophy)

Applications
The invention offers a transgenic mouse model for myotonic dystrophy.  This mouse model expresses expanded CUG repeats and develops myotonia and myopathy.  This model can be utilized to examine interactions between the loss- and gain-of-function effects of the mutation and to investigate the mechanism for nuclear retention and toxicity of expanded CUG repeats.

Advantages
In vivo  model for muscular dystrophy.

Patent Information: