In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice.

ABSTRACT

Dystrophin exon skipping in mdx mice has been the key model for the development of antisense therapy in vivo. Evaluation of exon skipping in this model involves the following two aspects (1) efficiency and accuracy of exon skipping and levels of dystrophin expression determined by RT-PCR, immunochemistry, and western blotting; (2) therapeutic effects on muscle pathology and functions assessed by histology and functional assays including grip strength measurement, treadmill test, echocardiogram, and hemodynamics for cardiac functions. Here we describe some key considerations and the essential methodologies in detail for exon skipping in mdx mice.