Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis

There is no effective treatment for amyotrophic lateral sclerosis (ALS), a devastating motor neuron disease. However, discovery of a G(4)C(2) repeat expansion in the C9ORF72 gene as the most common genetic cause of ALS has opened up new avenues for therapeutic intervention for this form of ALS. G(4)...

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Publicado no:Sci Transl Med
Main Authors: Gendron, Tania F., Chew, Jeannie, Stankowski, Jeannette N., Hayes, Lindsey R., Zhang, Yong-Jie, Prudencio, Mercedes, Carlomagno, Yari, Daughrity, Lillian M., Jansen-West, Karen, Perkerson, Emilie A., O’Raw, Aliesha, Cook, Casey, Pregent, Luc, Belzil, Veronique, van Blitterswijk, Marka, Tabassian, Lilia J., Lee, Chris W., Yue, Mei, Tong, Jimei, Song, Yuping, Castanedes-Casey, Monica, Rousseau, Linda, Phillips, Virginia, Dickson, Dennis W., Rademakers, Rosa, Fryer, John D., Rush, Beth K., Pedraza, Otto, Caputo, Ana M., Desaro, Pamela, Palmucci, Carla, Robertson, Amelia, Heckman, Michael G., Diehl, Nancy N., Wiggs, Edythe, Tierney, Michael, Braun, Laura, Farren, Jennifer, Lacomis, David, Ladha, Shafeeq, Fournier, Christina N., McCluskey, Leo F., Elman, Lauren B., Toledo, Jon B., McBride, Jennifer D., Tiloca, Cinzia, Morelli, Claudia, Poletti, Barbara, Solca, Federica, Prelle, Alessandro, Wuu, Joanne, Jockel-Balsarotti1, Jennifer, Rigo, Frank, Ambrose, Christine, Datta, Abhishek, Yang, Weixing, Raitcheva, Denitza, Antognetti, Giovanna, McCampbell, Alexander, Van Swieten, John C., Miller, Bruce L., Boxer, Adam L., Brown, Robert H., Bowser, Robert, Miller, Timothy M., Trojanowski, John Q., Grossman, Murray, Berry, James D., Hu, William T., Ratti, Antonia, Traynor, Bryan J., Disney, Matthew D., Benatar, Michael, Silani, Vincenzo, Glass, Jonathan D., Floeter, Mary Kay, Rothstein, Jeffrey D., Boylan, Kevin B., Petrucelli, Leonard
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5576451/
https://ncbi.nlm.nih.gov/pubmed/28356511
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aai7866
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