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

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

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...

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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.
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