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O02 RNA therapy approaches in myotonic dystrophy

โœ Scribed by C. Thornton


Book ID
117670152
Publisher
Elsevier Science
Year
2010
Tongue
English
Weight
81 KB
Volume
20
Category
Article
ISSN
0960-8966

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Myotonic dystrophy type 1 (DM1) is an autosomal dominant neuromuscular disorder caused by a CTG expansion mutation located in the 3 0 untranslated region of the DMPK (DM1 protein kinase) gene. According to current evidence, mutant DMPK mRNAs containing the trinucleotide expansion are retained in the