CRISPR/Cas9-Induced (CTG⋅CAG)(n) Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing

Myotonic dystrophy type 1 (DM1) is caused by (CTG⋅CAG)(n)-repeat expansion within the DMPK gene and thought to be mediated by a toxic RNA gain of function. Current attempts to develop therapy for this disease mainly aim at destroying or blocking abnormal properties of mutant DMPK (CUG)n RNA. Here, w...

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Publicat a:Mol Ther
Autors principals: van Agtmaal, Ellen L., André, Laurène M., Willemse, Marieke, Cumming, Sarah A., van Kessel, Ingeborg D.G., van den Broek, Walther J.A.A., Gourdon, Geneviève, Furling, Denis, Mouly, Vincent, Monckton, Darren G., Wansink, Derick G., Wieringa, Bé
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2017
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363205/
https://ncbi.nlm.nih.gov/pubmed/28129118
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2016.10.014
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