Targeting RNA foci in iPSC-derived motor neurons from ALS patients with C9ORF72 repeat expansion

Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative condition characterized by loss of motor neurons in the brain and spinal cord. Expansions of a hexanucleotide repeat (GGGGCC) in the noncoding region of the C9ORF72 gene are the most common cause of the familial form of ALS (C9-ALS),...

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Huvudupphovsmän: Sareen, D., O’Rourke, J. G., Meera, P., Muhammad, A.K.M.G., Grant, S., Simpkinson, M., Bell, S., Carmona, S., Ornelas, L., Sahabian, A., Gendron, T., Petrucelli, L., Baughn, M., Ravits, J., Harms, M. B., Rigo, F., Bennett, C. F., Otis, T. S., Svendsen, C. N., Baloh, R. H.
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2013
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Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4090945/
https://ncbi.nlm.nih.gov/pubmed/24154603
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.3007529
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