Cell membrane integrity in myotonic dystrophy type 1: implications for therapy.

Cell membrane integrity in myotonic dystrophy type 1: implications for therapy.

Myotonic Dystrophy type 1 (DM1) is a multisystemic disease caused by toxic RNA from a DMPK gene carrying an expanded (CTG•CAG)n repeat. Promising strategies for treatment of DM1 patients are currently being tested. These include antisense oligonucleotides and drugs for elimination of expanded RNA or...

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Bibliographic Details
Main Authors: Anchel González-Barriga, Julia Kranzen, Huib J E Croes, Suzanne Bijl, Walther J A A van den Broek, Ingeborg D G van Kessel, Baziel G M van Engelen, Judith C T van Deutekom, Bé Wieringa, Susan A M Mulders, Derick G Wansink
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2015-01-01
Series:PLoS ONE
Online Access:https://doi.org/10.1371/journal.pone.0121556

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https://doi.org/10.1371/journal.pone.0121556

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