Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients

The most common pathogenic mutation in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) is an intronic GGGGCC (G(4)C(2)) repeat in the chromosome 9 open reading frame 72 (C9orf72) gene. Cellular toxicity due to RNA foci and dipeptide repeat (DPR) proteins produced by the sense a...

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Xehetasun bibliografikoak
Argitaratua izan da:Mol Ther Nucleic Acids
Egile Nagusiak: Martier, Raygene, Liefhebber, Jolanda M., Miniarikova, Jana, van der Zon, Tom, Snapper, Jolanda, Kolder, Iris, Petry, Harald, van Deventer, Sander J., Evers, Melvin M., Konstantinova, Pavlina
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Gene & Cell Therapy 2019
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC6378669/
https://ncbi.nlm.nih.gov/pubmed/30776581
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2019.01.010
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