Mitochondrial Dysfunction and Oxidative Damage in parkin-deficient Mice von Palacino, James J, Sagi, Dijana, Goldberg, Matthew S, Krauss, Stefan, Motz, Claudia, Wacker, Maik, Klose, Joachim, Shen, Jie

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

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Splicing modulators act at the branch point adenosine binding pocket defined by the PHF5A–SF3b complex von 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

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

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Parkin-deficient Mice Exhibit Nigrostriatal Deficits but Not Loss of Dopaminergic Neurons von 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-Françoise, Maidment, Nigel T., Levine, Michael S., Shen, Jie

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The splice is right von Palacino, James

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

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A novel human embryonic stem cell‐derived Huntington's disease neuronal model exhibits mutant huntingtin (mHTT) aggregates and soluble mHTT‐dependent neurodegeneration von Lu, Boxun, Palacino, James

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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 von 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.

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

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

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

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Identification of NUB1 as a suppressor of mutant Huntingtin toxicity via enhanced protein clearance von 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

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A phase 1, first-in-human, open label, escalation and expansion study of ORM-5029, a highly potent GSPT1 degrader targeting HER2, in patients with HER2-expressing advanced solid tu... von Hurvitz, Sara A., Hamilton, Erika P., Spira, Alexander I., Pohlmann, Paula R, Giordano, Antonio, Clifton, Katherine, Anderson, Barry Douglas, Dutta, Sujoy, Mangipudi, Umesh, Saini, Shikha, Palacino, James, Karunaratne, Tina, Greensmith, Dennise, Christensen, Olaf, Wilks, Sharon

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Drugging unconventional targets: insights from Huntington's disease von Yu, Shenliang, Liang, Yijian, Palacino, James, Difiglia, Marian, Lu, Boxun

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ORM-6151: A First-in-Class, Anti-CD33 Antibody-Enabled GSPT1 Degrader for AML von Palacino, James, Lee, Pedro, Jeong, Hangyeol, Kim, Yeonjoon, Song, Yoojin, Permpoon, Uttapol, Wong, Wesley, Bai, Chen, Fishkin, Nathan, Takrouri, Khuloud, Yu, Eunjin, Yi, Yong, Skaletskaya, Anna, Chang, Ki-Hwan, Kim, Min-Soo, Kim, Da-Yeong, Choi, Dong-Ki, Park, Peter U

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Abstract 383: Survey of spliceosome gene mutations and associated splicing defects across 33 cancer types von Seiler, Michael, Peng, Shouyong, Agrawal, Anant, Tsai, Jennifer, Palacino, James, Buonamici, Silvia, Yu, Lihua

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Abstract 281: Sensitivity to splicing modulation of BCL2 family genes reveals cancer therapeutic strategies for splicing modulators von Aird, Daniel, Teng, Teng, Huang, Chia-Ling, Pazolli, Ermira, Banka, Deepti, Cheung-Ong, Kahlin, Eifert, Cheryl, Furman, Craig, Wu, Jeremy, Seiler, Michael, Buonamici, Silvia, Fekkes, Peter, Karr, Craig, Palacino, James, Park, Eunice, Smith, Peter, Yu, Lihua, Mizui, Yoshiharu, Warmuth, Markus, Chicas, Agustin, Corson, Laura, Zhu, Ping

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Aldose Reductase Inhibition Restores Endothelial Cell Function in Diabetic Rabbit Aorta von Tesfamariam, Belay, Palacino, James J, Weisbrod, Robert M, Cohen, Richard A

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Abstract 4436: A novel antibody-enabled dual precision targeted protein stabilization (TPS2) that augments anti-tumor immune response by targeting CBL-B inhibitor to exhausted T ce... von Lim, Joanne, Skaletskaya, Anna, Permpoon, Uttapol, Kim, Yeonjoon, Saini, Shikha, Takrouri, Khuloud, Ribkovskaia, Zinaida, Bhar, Palash, Fishkin, Nathan, Choi, Dong-Ki, Park, Ji Hyun, Palacino, James, Park, Peter U.

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