Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice

• Main Authors: Dominic Jauvin (Author), Jessina Chrétien (Author), Sanjay K. Pandey (Author), Laurie Martineau (Author), Lucille Revillod (Author), Guillaume Bassez (Author), Aline Lachon (Author), A. Robert McLeod (Author), Geneviève Gourdon (Author), Thurman M. Wheeler (Author), Charles A. Thornton (Author), C. Frank Bennett (Author), Jack Puymirat (Author)
• Format: Book
• Published: Elsevier, 2017-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Myotonic dystrophy type 1 (DM1), a dominant hereditary muscular dystrophy, is caused by an abnormal expansion of a (CTG)n trinucleotide repeat in the 3' UTR of the human dystrophia myotonica protein kinase (DMPK) gene. As a consequence, mutant transcripts containing expanded CUG repeats are ret...