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Autor:
Anchel González-Barriga, Susan AM Mulders, Jeroen van de Giessen, Jeroen D Hooijer, Suzanne Bijl, Ingeborg DG van Kessel, Josee van Beers, Judith CT van Deutekom, Jack AM Fransen, Bé Wieringa, Derick G Wansink
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 2, Iss C (2013)
Myotonic dystrophy type 1 (DM1) is caused by DM protein kinase (DMPK) transcripts containing an expanded (CUG)n repeat. Antisense oligonucleotide (AON)-mediated suppression of these mutant RNAs is considered a promising therapeutic strategy for this
Externí odkaz:
https://doaj.org/article/e1ac78deefa247bd9ae098816a38c96d
