Antibody therapy targeting RAN proteins rescues C9 ALS/FTD phenotypes in C9orf72 mouse model

The intronic C9orf72 G4C2 expansion, the most common genetic cause of ALS and FTD, produces sense- and antisense-expansion RNAs and six dipeptide repeat-associated, non-ATG (RAN) proteins, but their roles in disease are unclear. We generated high-affinity human antibodies targeting GA or GP RAN prot...

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Publicado no:Neuron
Main Authors: Nguyen, Lien, Montrasio, Fabio, Pattamatta, Amrutha, Tusi, Solaleh Khoramian, Bardhi, Olgert, Meyer, Kevin D., Hayes, Lindsey, Nakamura, Katsuya, Banez-Coronel, Monica, Coyne, Alyssa, Guo, Shu, Laboissonniere, Lauren A., Gu, Yuanzheng, Narayanan, Saravanakumar, Smith, Benjamin, Nitsch, Roger M., Kankel, Mark W., Rushe, Mia, Rothstein, Jeffrey, Zu, Tao, Grimm, Jan, Ranum, Laura P.W.
Formato: Artigo
Idioma:Inglês
Publicado em: 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7391607/
https://ncbi.nlm.nih.gov/pubmed/31831332
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2019.11.007
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