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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Bibliografiska uppgifter
I publikationen:Mol Ther
Huvudupphovsmän: 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
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2017
Ämnen:
Original Article
Länkar: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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