| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10804038 | Biochimie | 2012 | 7 Pages |
Abstract
⺠RNA gain-of-function emerged as a new mechanism for diseases with expanded repeats. ⺠Myotonic dystrophy type 1 is a dominant inherited disease caused by an CTG expansion. ⺠The expanded CTG repeat is located in the 3Ⲡnon-coding region of the DMPK gene. ⺠This review focused on toxic RNA gain-of-function as a pathogenic mechanism for DM1. ⺠DM1 neuromuscular disease belongs to the new family of RNA-dominant disorders.
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Authors
A.F. Klein, E. Gasnier, D. Furling,