Petrucelli, Leonard
Mayo Clinic
Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
Citations 17
Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G 4 C 2 ) repeat expansion in vitro and in vivo ALS models
Bush, Jessica A. , Aikawa, Haruo , Fuerst, Rita, Li, Yue , Ursu, Andrei , Meyer, Samantha M. , Benhamou, Raphael I. , Chen, Jonathan L. , Khan, Tanya , Wagner-Griffin, Sarah , Meter, Montina J. Van , Tong, Yuquan , Olafson, Hailey, McKee, Kendra , Childs‐Disney, Jessica L. , Zhang, Yong‐Jie , Wang, Eric T. , Wang, Kye Won , Petrucelli, Leonard , Van Meter, Montina J , McKee, Kendra K , Gendron, Tania F , Zhang, Yong-Jie , Coyne, Alyssa N , Wang, Eric T , Wang, Kye Won , Petrucelli, Leonard , Rothstein, Jeffrey D , Disney, Matthew D , Benhamou, Raphael I , Aikawa, Haruo , Gendron, Tania F. , Coyne, Alyssa N. , Yildirim, Ilyas , Rothstein, Jeffrey D. , Disney, Matthew D.
Science Translational Medicine | October 27, 2021 | Volume: 13 | Issue: 617 | Pages: No page available
Small-molecule recruitment of an endogenous nuclease rescues pathologies associated with c9ALS/FTD in vivo by targeting an RNA repeat expansion.
Serum neurofilament light protein correlates with unfavorable clinical outcomes in hospitalized patients with COVID-19
Prudencio, Mercedes , Marquez, Christopher P. , Jansen‐West, Karen , Franco-Mesa, Camila , White, Launia J. , Dunmore, Judith A. , Cook, Casey , Lilley, Meredith T. , Song, Yuping , Harlow, Caroline , Oskarsson, Björn , Nicholson, Katharine, Hoyne, Jonathan , Petrucelli, Leonard , Marquez, Christopher P , Jansen-West, Karen R , Franco-Mesa, Camila , White, Launia J , Dunmore, Judith A , Cook, Casey N , Harlow, Caroline F , Oskarsson, Björn , Wszolek, Z.K. , Hickson, LaTonya J , O'Horo, John C , Hoyne, Jonathan B , Gendron, Tania F , Meschia, James F , Petrucelli, Leonard , Prudencio, Mercedes , Lilley, Meredith T , Erben, Young , Heckman, Michael G. , Wszołek, Zbigniew K. , Hickson, LaTonya J. , O’Horo, John C. , Gendron, Tania F. , Meschia, James F.
Science Translational Medicine | June 15, 2021 | Volume: 13 | Issue: 602 | Pages: No page available
Hospitalized patients with COVID-19 show increased serum concentrations of neurofilament light chain that correlate with worse clinical outcomes.
Plasma neurofilament light predicts mortality in patients with stroke
Gendron, Tania F. , Badi, Mohammed K. , Jansen‐West, Karen , Vilanilam, George K. , Burch, Alexander R. , Walton, Ronald L., Ross, Owen A. , Brott, Thomas G. , Miller, Timothy M. , Nicholson, Katharine, Oskarsson, Björn , Falcone, Guido J. , Cucchiara, Brett , Petrucelli, Leonard , Jansen-West, Karen R , Vilanilam, George , Johnson, Patrick W. , Burch, Alexander R , Ross, Owen A , Miller, Timothy M , Wszolek, Z.K. , Oskarsson, Björn , Sheth, Kevin N , Sansing, Lauren H , Falcone, Guido J , Cucchiara, Brett L , Meschia, James F , Petrucelli, Leonard , Badi, Mohammed , Gendron, Tania F , Heckman, Michael G. , Berry, James D. , Wszołek, Zbigniew K. , Sheth, Kevin N. , Meschia, James F.
Science Translational Medicine | November 12, 2020 | Volume: 12 | Issue: 569 | Pages: No page available
Plasma neurofilament light is a reliable, uniform marker to estimate neuroaxonal injury and forecast all-cause mortality across major stroke types.
Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3
Prudencio, Mercedes , García‐Moreno, Héctor , Jansen‐West, Karen , Al‐Shaikh, Rana Hanna , Gendron, Tania F. , Spiegel, Matthew R. , Carlomagno, Yari , Daughrity, Lillian M. , Song, Yuping , Dunmore, Judith A. , Byron, Natalie, Oskarsson, Björn , Nicholson, Katharine, Staff, Nathan P. , Gorcenco, Sorina , Puschmann, Andreas , Lemos, João , Januário, Cristina , LeDoux, Mark S. , Polke, James, Labrum, Robin, McLoughlin, Hayley S. , Paulson, Henry L. , Onodera, Osamu , Tada, Mari , Kakita, Akiyoshi , Fryer, John Denis , Karremo, Christin, Gomes, Inês , Caviness, John N., Pittelkow, Mark R. , Aasly, Jan , Pfeiffer, Ronald F. , Veerappan, Venka , Eggenberger, Eric , Huang, Josephine F. , Uitti, Ryan J. , Wierenga, Klaas J. , Collazo, I. Vanessa Marin , Tipton, Philip W. , Gerpen, Jay A. van, Blitterswijk, Marka van , Bu, Guojun , Petrucelli, Leonard , Jansen-West, Karen R , Carlomagno, Yari , Daughrity, Lillian M , Dunmore, Judith A , Oskarsson, Björn , Staff, Nathan P , Gorcenco, Sorina , Puschmann, Andreas , Lemos, João , Januário, Cristina , Shakkottai, Vikram , McLoughlin, Hayley S , Paulson, Henry L , Konno, Takuya , Onodera, Osamu , Ikeuchi, T , Tada, Mari , Gomes, Inês , Pittelkow, Mark R , Aasly, Jan , Eggenberger, Eric R, Freeman, William D , Huang, Josephine F , Marin Collazo, Iris V , Tipton, Philip W , van Blitterswijk, Marka , Bu, Guojun , Wszolek, Z.K. , Giunti, Paola , Petrucelli, Leonard , Prudencio, Mercedes , Gendron, Tania F , Hanna Al-Shaikh, Rana , Heckman, Michael G. , Friedman, Joseph H. , Konno, Takuya , Ikeuchi, Takeshi , Freeman, William D. , Wszołek, Zbigniew K.
Science Translational Medicine | October 21, 2020 | Volume: 12 | Issue: 566 | Pages: No page available
PolyQ ATXN3 is a pharmacodynamic marker for SCA3, and a SNP associated with the expanded allele may serve to specifically target mutant ATXN3 .
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.
Heterochromatin anomalies and double-stranded RNA accumulation underlie C9orf72 poly(PR) toxicity
Zhang, Yong‐Jie , Guo, Lin , Gonzales, Patrick , Gendron, Tania F. , Wu, Yanwei , Jansen‐West, Karen , O’Raw, Aliesha D. , Pickles, Sarah , Prudencio, Mercedes , Carlomagno, Yari , Gachechiladze, Mariam A. , Tian, Ruilin , Chew, Jeannie , DeTure, Michael , Lin, Wen-Lang, Tong, Jimei, Daughrity, Lillian M. , Yue, Mei, Song, Yuping , Andersen, Jonathan W. , Castanedes‐Casey, Monica , Kurti, Aishe, Antognetti, Giovanna, McCampbell, Alexander , Rademakers, Rosa , Oskarsson, Björn , Kampmann, Martin , Ward, Michael E. , Fryer, John Denis , Link, Christopher D. , Shorter, James , Petrucelli, Leonard , Gonzales, Patrick K , Pickles, Sarah R , Chew, Jeannie , DeTure, Michael , Daughrity, Lillian M , Castanedes-Casey, Monica , McCampbell, Alexander , Rademakers, Rosa , Oskarsson, Björn , Dickson, Dennis W , Kampmann, Martin , Ward, Michael E , Shorter, James , Petrucelli, Leonard , Zhang, Yong-Jie , Prudencio, Mercedes , O'Raw, Aliesha , Gendron, Tania F , Guo, Lin , Ludwig, Connor , Datta, Abhishek, Dickson, Dennis W.
Science | February 15, 2019 | Volume: 363 | Issue: 6428 | Pages: No page available
How dipeptide repeats cause pathology A repeat expansion in the chromosome 9 open reading frame 72 ( C9orf72 ) gene is the most common known cause of two neurodegenerative diseases: frontotemporal dementia and amyotrophic lateral sclerosis. This expansion leads to the abnormal production of proteins of repeating dipeptides, but their contribution to disease pathogenesis remains unclear. Zhang et al. engineered a mouse model to study the consequences of one of these dipeptides—prolinearginine dipeptide repeat protein, poly(PR)—in the brain. They found that poly(PR) caused neuron loss as well as motor and memory impairments. These detrimental effects resulted from poly(PR)-induced perturbation of heterochromatin function, a tightly packed form of DNA that represses gene expression. Science , this issue p. eaav2606
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.