Citations 4
Nuclear accumulation of CHMP7 initiates nuclear pore complex injury and subsequent TDP-43 dysfunction in sporadic and familial ALS
Coyne, Alyssa N. , Baskerville, Victoria, Zaepfel, Benjamin L. , Dickson, Dennis W. , Rigo, Frank , Bennett, Frank , Lusk, C. Patrick , Rothstein, Jeffrey D. , Dickson, Dennis W , Coyne, Alyssa N , Rothstein, Jeffrey D
Science Translational Medicine | July 28, 2021 | Volume: 13 | Issue: 604 | Pages: No page available
Nuclear accumulation of CHMP7 leads to multiple cellular pathologies that can be mitigated via CHMP7 ASOs in familial and sporadic ALS iPSNs.
C9orf72 poly(GR) aggregation induces TDP-43 proteinopathy
Cook, Casey , Wu, Yanwei , Odeh, Hana M. , Gendron, Tania F. , Jansen‐West, Karen , Rosso, Giulia del, Yue, Mei, Jiang, Peizhou , Gomes, Edward, Tong, Jimei, Daughrity, Lillian M. , Avendano, Nicole M., Castanedes‐Casey, Monica , Shao, Wei , Oskarsson, Björn , Tomassy, Giulio Srubek, McCampbell, Alexander , Rigo, Frank , Zhang, Yong‐Jie , Petrucelli, Leonard , Cook, Casey N , Del Rosso, Giulia, Dickson, Dennis W , Shorter, James , Zhang, Yong-Jie , Petrucelli, Leonard , Oskarsson, Björn , Gendron, Tania F , Shao, Wei , Dickson, Dennis W. , Shorter, James
Science Translational Medicine | September 2, 2020 | Volume: 12 | Issue: 559 | Pages: No page available
G 4 C 2 repeat–derived poly(GR) plays a key role in mediating TDP-43 mislocalization and aggregation in vitro and in vivo.
Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72 -associated amyotrophic lateral sclerosis
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 D. , Cook, Casey , Pregent, Luc , Belzil, Véronique , Blitterswijk, Marka van , Tabassian, Lilia J., Lee, Chris W. , Yue, Mei, Tong, Jimei, Song, Yuping , Castanedes‐Casey, Monica , Rousseau, Linda , Phillips, Virginia , Rademakers, Rosa , Fryer, John Denis , Rush, Beth , Pedraza, Otto , Caputo, Ana M., Desaro, Pamela , Palmucci, Carla, Robertson, A. , Diehl, Nancy N. , Wiggs, Edythe, Tierney, Michael , Braun, Laura, Farren, Jennifer, Lacomis, David , Fournier, Christina , McCluskey, Leo, Elman, Lauren , Toledo, Jon B. , McBride, Jennifer D. , Tiloca, Cinzia , Morelli, Claudia , Poletti, Barbara , Solca, Federica , Wuu, Joanne , Jockel‐Balsarotti, Jennifer , Rigo, Frank , Ambrose, Christine, Yang, Weixing , Raitcheva, Denitza , Antognetti, Giovanna, McCampbell, Alexander , Boxer, Adam L. , Brown, Robert H. , Bowser, Robert , Miller, Timothy M. , Trojanowski, John Q. , Hu, William T. , Ratti, Antonia , Benatar, Michael , Glass, Jonathan D. , Floeter, Mary Kay, Petrucelli, Leonard , Gendron, Tania F. , Zhang, Yong-Jie , Prudencio, Mercedes , Carlomagno, Yari , Jansen-West, Karen, O'Raw, Aliesha , Pregent, Luc , Belzil, Veronique , van Blitterswijk, Marka , Castanedes-Casey, Monica , Rousseau, Linda , Phillips, Virginia , Rademakers, Rosa , Fryer, John D., Desaro, Pamela , Palmucci, Carla , Robertson, Amelia , Lacomis, David , Ladha, Shafeeq , Tiloca, Cinzia , Poletti, Barbara , Solca, Federica , Prelle, Alessandro , Jockel-Balsarotti, Jennifer , Yang, Weixing , Raitcheva, Denitza , Benatar, Michael , Silani, Vincenzo , Boylan, Kevin B, Petrucelli, Leonard , Dickson, Dennis W. , Heckman, Michael G. , Ladha, Shafeeq , Datta, Abhishek , Swieten, John C. van , Miller, Bruce L. , Grossman, Murray , Berry, James D. , Traynor, Bryan J. , Disney, Matthew D. , Silani, Vincenzo , Rothstein, Jeffrey D. , Boylan, Khrista , Hu, William T.
Science Translational Medicine | March 29, 2017 | Volume: 9 | Issue: 383 | Pages: No page available
Poly(GP) proteins are a promising pharmacodynamic marker for developing and testing therapeutics for treating C9ORF72 -associated amyotrophic lateral sclerosis.
Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion
O’Rourke, Jacqueline G., Meera, Pratap , Muhammad, A.K.M. Ghulam, Grant, Sharday, Simpkinson, Megan, Bell, Shaughn , Carmona, Sharon, Ornelas, Loren, Sahabian, Anais , Petrucelli, Leonard , Baughn, Michael W. , Ravits, John , Harms, Matthew B. , Rigo, Frank , Bennett, C. Frank , Otis, Thomas S. , Svendsen, Clive N. , Baloh, Robert H. , Sareen, Dhruv , Gendron, Tania F.
Science Translational Medicine | October 23, 2013 | Volume: 5 | Issue: 208 | Pages: No page available
Antisense oligonucleotides can correct disease-specific phenotypes in cultured motor neurons differentiated from iPSCs derived from ALS patients with a C9ORF72 repeat expansion.