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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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發表在:PLoS One
Main Authors: González-Barriga, Anchel, Kranzen, Julia, Croes, Huib J. E., Bijl, Suzanne, van den Broek, Walther J. A. A., van Kessel, Ingeborg D. G., van Engelen, Baziel G. M., van Deutekom, Judith C. T., Wieringa, Bé, Mulders, Susan A. M., Wansink, Derick G.
格式: Artigo
語言:Inglês
出版: Public Library of Science 2015
主題:
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC4370802/
https://ncbi.nlm.nih.gov/pubmed/25799359
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0121556
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