Pesquisa | BVS Doenças Infecciosas e Parasitárias

1.

SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.

Palacino, James; Swalley, Susanne E; Song, Cheng; Cheung, Atwood K; Shu, Lei; Zhang, Xiaolu; Van Hoosear, Mailin; Shin, Youngah; Chin, Donovan N; Keller, Caroline Gubser; Beibel, Martin; Renaud, Nicole A; Smith, Thomas M; Salcius, Michael; Shi, Xiaoying; Hild, Marc; Servais, Rebecca; Jain, Monish; Deng, Lin; Bullock, Caroline; McLellan, Michael; Schuierer, Sven; Murphy, Leo; Blommers, Marcel J J; Blaustein, Cecile; Berenshteyn, Frada; Lacoste, Arnaud; Thomas, Jason R; Roma, Guglielmo; Michaud, Gregory A; Tseng, Brian S; Porter, Jeffery A; Myer, Vic E; Tallarico, John A; Hamann, Lawrence G; Curtis, Daniel; Fishman, Mark C; Dietrich, William F; Dales, Natalie A; Sivasankaran, Rajeev.

Nat Chem Biol ; 11(7): 511-7, 2015 Jul.

Artigo em Inglês | MEDLINE | ID: mdl-26030728

2.

The splice is right.

Palacino, James.

Nat Chem Biol ; 14(12): 1068-1069, 2018 12.

Artigo em Inglês | MEDLINE | ID: mdl-30323218

3.

A novel human embryonic stem cell-derived Huntington's disease neuronal model exhibits mutant huntingtin (mHTT) aggregates and soluble mHTT-dependent neurodegeneration.

Lu, Boxun; Palacino, James.

FASEB J ; 27(5): 1820-9, 2013 May.

Artigo em Inglês | MEDLINE | ID: mdl-23325320

4.

Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.

Palacino, James; Swalley, Susanne E; Song, Cheng; Cheung, Atwood K; Shu, Lei; Zhang, Xiaolu; Van Hoosear, Mailin; Shin, Youngah; Chin, Donovan N; Keller, Caroline Gubser; Beibel, Martin; Renaud, Nicole A; Smith, Thomas M; Salcius, Michael; Shi, Xiaoying; Hild, Marc; Servais, Rebecca; Jain, Monish; Deng, Lin; Bullock, Caroline; McLellan, Michael; Schuierer, Sven; Murphy, Leo; Blommers, Marcel J J; Blaustein, Cecile; Berenshteyn, Frada; Lacoste, Arnaud; Thomas, Jason R; Roma, Guglielmo; Michaud, Gregory A; Tseng, Brian S; Porter, Jeffery A; Myer, Vic E; Tallarico, John A; Hamann, Lawrence G; Curtis, Daniel; Fishman, Mark C; Dietrich, William F; Dales, Natalie A; Sivasankaran, Rajeev.

Nat Chem Biol ; 12(4): 304, 2016 Apr.

Artigo em Inglês | MEDLINE | ID: mdl-26991088

5.

Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.

Palacino, James; Swalley, Susanne E; Song, Cheng; Cheung, Atwood K; Shu, Lei; Zhang, Xiaolu; Van Hoosear, Mailin; Shin, Youngah; Chin, Donovan N; Keller, Caroline Gubser; Beibel, Martin; Renaud, Nicole A; Smith, Thomas M; Salcius, Michael; Shi, Xiaoying; Hild, Marc; Servais, Rebecca; Jain, Monish; Deng, Lin; Bullock, Caroline; McLellan, Michael; Schuierer, Sven; Murphy, Leo; Blommers, Marcel J J; Blaustein, Cecile; Berenshteyn, Frada; Lacoste, Arnaud; Thomas, Jason R; Roma, Guglielmo; Michaud, Gregory A; Tseng, Brian S; Porter, Jeffery A; Myer, Vic E; Tallarico, John A; Hamann, Lawrence G; Curtis, Daniel; Fishman, Mark C; Dietrich, William F; Dales, Natalie A; Sivasankaran, Rajeev.

Nat Chem Biol ; 11(9): 741, 2015 Sep.

Artigo em Inglês | MEDLINE | ID: mdl-26284678

6.

Sensitivity to splicing modulation of BCL2 family genes defines cancer therapeutic strategies for splicing modulators.

Aird, Daniel; Teng, Teng; Huang, Chia-Ling; Pazolli, Ermira; Banka, Deepti; Cheung-Ong, Kahlin; Eifert, Cheryl; Furman, Craig; Wu, Zhenhua Jeremy; Seiler, Michael; Buonamici, Silvia; Fekkes, Peter; Karr, Craig; Palacino, James; Park, Eunice; Smith, Peter G; Yu, Lihua; Mizui, Yoshiharu; Warmuth, Markus; Chicas, Agustin; Corson, Laura; Zhu, Ping.

Nat Commun ; 10(1): 137, 2019 01 11.

Artigo em Inglês | MEDLINE | ID: mdl-30635584

7.

Somatic Mutational Landscape of Splicing Factor Genes and Their Functional Consequences across 33 Cancer Types.

Seiler, Michael; Peng, Shouyong; Agrawal, Anant A; Palacino, James; Teng, Teng; Zhu, Ping; Smith, Peter G; Buonamici, Silvia; Yu, Lihua.

Cell Rep ; 23(1): 282-296.e4, 2018 04 03.

Artigo em Inglês | MEDLINE | ID: mdl-29617667

8.

High-Throughput Functional Analysis Distinguishes Pathogenic, Nonpathogenic, and Compensatory Transcriptional Changes in Neurodegeneration.

Al-Ramahi, Ismael; Lu, Boxun; Di Paola, Simone; Pang, Kaifang; de Haro, Maria; Peluso, Ivana; Gallego-Flores, Tatiana; Malik, Nazish T; Erikson, Kelly; Bleiberg, Benjamin A; Avalos, Matthew; Fan, George; Rivers, Laura Elizabeth; Laitman, Andrew M; Diaz-García, Javier R; Hild, Marc; Palacino, James; Liu, Zhandong; Medina, Diego L; Botas, Juan.

Cell Syst ; 7(1): 28-40.e4, 2018 07 25.

Artigo em Inglês | MEDLINE | ID: mdl-29936182

9.

Synthetic Lethal and Convergent Biological Effects of Cancer-Associated Spliceosomal Gene Mutations.

Lee, Stanley Chun-Wei; North, Khrystyna; Kim, Eunhee; Jang, Eunjung; Obeng, Esther; Lu, Sydney X; Liu, Bo; Inoue, Daichi; Yoshimi, Akihide; Ki, Michelle; Yeo, Mirae; Zhang, Xiao Jing; Kim, Min Kyung; Cho, Hana; Chung, Young Rock; Taylor, Justin; Durham, Benjamin H; Kim, Young Joon; Pastore, Alessandro; Monette, Sebastien; Palacino, James; Seiler, Michael; Buonamici, Silvia; Smith, Peter G; Ebert, Benjamin L; Bradley, Robert K; Abdel-Wahab, Omar.

Cancer Cell ; 34(2): 225-241.e8, 2018 08 13.

Artigo em Inglês | MEDLINE | ID: mdl-30107174

10.

Splicing modulators act at the branch point adenosine binding pocket defined by the PHF5A-SF3b complex.

Teng, Teng; Tsai, Jennifer Hc; Puyang, Xiaoling; Seiler, Michael; Peng, Shouyong; Prajapati, Sudeep; Aird, Daniel; Buonamici, Silvia; Caleb, Benjamin; Chan, Betty; Corson, Laura; Feala, Jacob; Fekkes, Peter; Gerard, Baudouin; Karr, Craig; Korpal, Manav; Liu, Xiang; T Lowe, Jason; Mizui, Yoshiharu; Palacino, James; Park, Eunice; Smith, Peter G; Subramanian, Vanitha; Wu, Zhenhua Jeremy; Zou, Jian; Yu, Lihua; Chicas, Agustin; Warmuth, Markus; Larsen, Nicholas; Zhu, Ping.

Nat Commun ; 8: 15522, 2017 05 25.

Artigo em Inglês | MEDLINE | ID: mdl-28541300

11.

Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteins.

Lee, Stanley Chun-Wei; Dvinge, Heidi; Kim, Eunhee; Cho, Hana; Micol, Jean-Baptiste; Chung, Young Rock; Durham, Benjamin H; Yoshimi, Akihide; Kim, Young Joon; Thomas, Michael; Lobry, Camille; Chen, Chun-Wei; Pastore, Alessandro; Taylor, Justin; Wang, Xujun; Krivtsov, Andrei; Armstrong, Scott A; Palacino, James; Buonamici, Silvia; Smith, Peter G; Bradley, Robert K; Abdel-Wahab, Omar.

Nat Med ; 22(6): 672-8, 2016 06.

Artigo em Inglês | MEDLINE | ID: mdl-27135740

12.

A striatal-enriched intronic GPCR modulates huntingtin levels and toxicity.

Yao, Yuwei; Cui, Xiaotian; Al-Ramahi, Ismael; Sun, Xiaoli; Li, Bo; Hou, Jiapeng; Difiglia, Marian; Palacino, James; Wu, Zhi-Ying; Ma, Lixiang; Botas, Juan; Lu, Boxun.

Elife ; 42015 Mar 04.

Artigo em Inglês | MEDLINE | ID: mdl-25738228

13.

Drugging unconventional targets: insights from Huntington's disease.

Yu, Shenliang; Liang, Yijian; Palacino, James; Difiglia, Marian; Lu, Boxun.

Trends Pharmacol Sci ; 35(2): 53-62, 2014 Feb.

Artigo em Inglês | MEDLINE | ID: mdl-24388390

14.

Identification of NUB1 as a suppressor of mutant Huntington toxicity via enhanced protein clearance.

Lu, Boxun; Al-Ramahi, Ismael; Valencia, Antonio; Wang, Qiong; Berenshteyn, Frada; Yang, Haidi; Gallego-Flores, Tatiana; Ichcho, Salah; Lacoste, Arnaud; Hild, Marc; Difiglia, Marian; Botas, Juan; Palacino, James.

Nat Neurosci ; 16(5): 562-70, 2013 May.

Artigo em Inglês | MEDLINE | ID: mdl-23525043

15.

Erratum: Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteins.

Lee, Stanley Chun-Wei; Dvinge, Heidi; Kim, Eunhee; Cho, Hana; Micol, Jean-Baptiste; Chung, Young Rock; Durham, Benjamin H; Yoshimi, Akihide; Kim, Young Joon; Thomas, Michael; Lobry, Camille; Chen, Chun-Wei; Pastore, Alessandro; Taylor, Justin; Wang, Xujun; Krivtsov, Andrei; Armstrong, Scott A; Palacino, James; Buonamici, Silvia; Smith, Peter G; Bradley, Robert K; Abdel-Wahab, Omar.

Nat Med ; 22(6): 692, 2016 06 07.

Artigo em Inglês | MEDLINE | ID: mdl-27270779

16.

Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors.

Kegel, Kimberly B; Sapp, Ellen; Alexander, Jonathan; Reeves, Patrick; Bleckmann, Dorothee; Sobin, Linsday; Masso, Nicholas; Valencia, Antonio; Jeong, Hyunkyung; Krainc, Dimitri; Palacino, James; Curtis, Daniel; Kuhn, Rainer; Betschart, Claudia; Sena-Esteves, Miguel; Aronin, Neil; Paganetti, Paolo; Difiglia, Marian.

Mol Neurodegener ; 5: 58, 2010 Dec 14.

Artigo em Inglês | MEDLINE | ID: mdl-21156064

17.

Intrinsic resistance of neural stem cells to toxic metabolites may make them well suited for cell non-autonomous disorders: evidence from a mouse model of Krabbe leukodystrophy.

Taylor, Roseanne M; Lee, Jean Pyo; Palacino, James J; Bower, Kate A; Li, Jianxue; Vanier, Marie T; Wenger, David A; Sidman, Richard L; Snyder, Evan Y.

J Neurochem ; 97(6): 1585-99, 2006 Jun.

Artigo em Inglês | MEDLINE | ID: mdl-16805770

18.

Mitochondrial dysfunction and oxidative damage in parkin-deficient mice.

Palacino, James J; Sagi, Dijana; Goldberg, Matthew S; Krauss, Stefan; Motz, Claudia; Wacker, Maik; Klose, Joachim; Shen, Jie.

J Biol Chem ; 279(18): 18614-22, 2004 Apr 30.

Artigo em Inglês | MEDLINE | ID: mdl-14985362

19.

Parkin-deficient mice exhibit nigrostriatal deficits but not loss of dopaminergic neurons.

Goldberg, Matthew S; Fleming, Sheila M; Palacino, James J; Cepeda, Carlos; Lam, Hoa A; Bhatnagar, Anushree; Meloni, Edward G; Wu, Nanping; Ackerson, Larry C; Klapstein, Gloria J; Gajendiran, Mahadevan; Roth, Bryan L; Chesselet, Marie-Francoise; Maidment, Nigel T; Levine, Michael S; Shen, Jie.

J Biol Chem ; 278(44): 43628-35, 2003 Oct 31.

Artigo em Inglês | MEDLINE | ID: mdl-12930822

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SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.

The splice is right.

A novel human embryonic stem cell-derived Huntington's disease neuronal model exhibits mutant huntingtin (mHTT) aggregates and soluble mHTT-dependent neurodegeneration.

Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.

Corrigendum: SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.

Sensitivity to splicing modulation of BCL2 family genes defines cancer therapeutic strategies for splicing modulators.

Somatic Mutational Landscape of Splicing Factor Genes and Their Functional Consequences across 33 Cancer Types.

High-Throughput Functional Analysis Distinguishes Pathogenic, Nonpathogenic, and Compensatory Transcriptional Changes in Neurodegeneration.

Synthetic Lethal and Convergent Biological Effects of Cancer-Associated Spliceosomal Gene Mutations.

Splicing modulators act at the branch point adenosine binding pocket defined by the PHF5A-SF3b complex.

Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteins.

A striatal-enriched intronic GPCR modulates huntingtin levels and toxicity.

Drugging unconventional targets: insights from Huntington's disease.

Identification of NUB1 as a suppressor of mutant Huntington toxicity via enhanced protein clearance.

Erratum: Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteins.

Huntingtin cleavage product A forms in neurons and is reduced by gamma-secretase inhibitors.

Intrinsic resistance of neural stem cells to toxic metabolites may make them well suited for cell non-autonomous disorders: evidence from a mouse model of Krabbe leukodystrophy.

Mitochondrial dysfunction and oxidative damage in parkin-deficient mice.

Parkin-deficient mice exhibit nigrostriatal deficits but not loss of dopaminergic neurons.