Three-Year Longitudinal Analysis of Splicing Changes in Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is caused by a CTG expansion in the 3′ untranslated region of the DMPK gene that results in the expression of expanded CUG RNA which trap splicing factors resulting in widespread alternative splicing dysregulation. A panel of these splicing events, or splicing index (SI), is currently us...