Self-renewal as a therapeutic target in human colorectal cancer von Kreso, Antonija, van Galen, Peter, Pedley, Nicholas M, Lima-Fernandes, Evelyne, Frelin, Catherine, Davis, Thomas, Cao, Liangxian, Baiazitov, Ramil, Du, Wu, Sydorenko, Nadiya, Moon, Young-Choon, Gibson, Lianne, Wang, Yadong, Leung, Cherry, Iscove, Norman N, Arrowsmith, Cheryl H, Szentgyorgyi, Eva, Gallinger, Steven, Dick, John E, O'Brien, Catherine A

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Small molecule splicing modifiers with systemic HTT-lowering activity von Bhattacharyya, Anuradha, Trotta, Christopher R., Narasimhan, Jana, Wiedinger, Kari J., Li, Wencheng, Effenberger, Kerstin A., Woll, Matthew G., Jani, Minakshi B., Risher, Nicole, Yeh, Shirley, Cheng, Yaofeng, Sydorenko, Nadiya, Moon, Young-Choon, Karp, Gary M., Weetall, Marla, Dakka, Amal, Gabbeta, Vijayalakshmi, Naryshkin, Nikolai A., Graci, Jason D., Tripodi, Thomas, Southwell, Amber, Hayden, Michael, Colacino, Joseph M., Peltz, Stuart W.

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Synthesis and Reactions of Trifluoromethyl‐Substituted 1,3,5‐Triazines von Jeon, Woohyung, Baiazitov, Ramil, Chierchia, Matteo, Niederer, Kyle, Ren, Hongyu, Moon, Young‐Choon, Gilbert, Bradley B.

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Discovery and SAR Studies of Potent Modulators of BMI‐1 Expression von Baiazitov, Ramil Y., Sydorenko, Nadiya, Du, Wu, Ren, Hongyu, Cao, Liang, Davis, Thomas, Cintron‐Lue, Katherine, Kim, Min‐Jung, Zhuo, Jin, Mody, Shreshtha, Weetall, Marla, Sheedy, Josephine, Risher, Nicole, Almstead, Neil, Moon, Young‐Choon

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Evaluating the Mechanism and Therapeutic Potential of PTC-028, a Novel Inhibitor of BMI-1 Function in Ovarian Cancer von Dey, Anindya, Xiong, Xunhao, Crim, Aleia, Dwivedi, Shailendra Kumar Dhar, Mustafi, Soumyajit Banerjee, Mukherjee, Priyabrata, Cao, Liangxian, Sydorenko, Nadiya, Baiazitov, Ramil, Moon, Young-Choon, Dumble, Melissa, Davis, Thomas, Bhattacharya, Resham

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The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential von Friesen, Westley J, Johnson, Briana, Sierra, Jairo, Zhuo, Jin, Vazirani, Priya, Xue, Xiaojiao, Tomizawa, Yuki, Baiazitov, Ramil, Morrill, Christie, Ren, Hongyu, Babu, Suresh, Moon, Young-Choon, Branstrom, Art, Mollin, Anna, Hedrick, Jean, Sheedy, Josephine, Elfring, Gary, Weetall, Marla, Colacino, Joseph M, Welch, Ellen M, Peltz, Stuart W

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Preclinical and Early Clinical Development of PTC596, a Novel Small-Molecule Tubulin-Binding Agent von Jernigan, Finith, Branstrom, Arthur, Baird, John D, Cao, Liangxian, Dali, Mandar, Furia, Bansri, Kim, Min Jung, O'Keefe, Kylie, Kong, Ronald, Laskin, Oscar L, Colacino, Joseph M, Pykett, Mark, Mollin, Anna, Sheedy, Josephine, Dumble, Melissa, Moon, Young-Choon, Sheridan, Richard, Mühlethaler, Tobias, Spiegel, Robert J, Prota, Andrea E, Steinmetz, Michel O, Weetall, Marla

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BMI1 is a therapeutic target in recurrent medulloblastoma von Bakhshinyan, David, Venugopal, Chitra, Adile, Ashley A., Garg, Neha, Manoranjan, Branavan, Hallett, Robin, Wang, Xin, Mahendram, Sujeivan, Vora, Parvez, Vijayakumar, Thusyanth, Subapanditha, Minomi, Singh, Mohini, Kameda-Smith, Michelle Masayo, Qazi, Maleeha, McFarlane, Nicole, Mann, Aneet, Ajani, Olufemi A., Yarascavitch, Blake, Ramaswamy, Vijay, Farooq, Hamza, Morrissy, Sorana, Cao, Liangxian, Sydorenko, Nadiya, Baiazitov, Ramil, Du, Wu, Sheedy, Josephine, Weetall, Marla, Moon, Young-Choon, Lee, Chang-Sun, Kwiecien, Jacek M., Delaney, Kathleen H., Doble, Brad, Cho, Yoon-Jae, Mitra, Siddhartha, Kaplan, David, Taylor, Michael D., Davis, Thomas W., Singh, Sheila K.

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Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform von Cao, Liangxian, Weetall, Marla, Bombard, Jenelle, Qi, Hongyan, Arasu, Tamil, Lennox, William, Hedrick, Jean, Sheedy, Josephine, Risher, Nicole, Brooks, Peter C, Trifillis, Panayiota, Trotta, Christopher, Moon, Young-Choon, Babiak, John, Almstead, Neil G, Colacino, Joseph M, Davis, Thomas W, Peltz, Stuart W

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In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren von Kong, Ronald, Ma, Jiyuan, Hwang, Seongwoo, Moon, Young‐Choon, Welch, Ellen M., Weetall, Marla, Colacino, Joseph M., Almstead, Neil, Babiak, John, Goodwin, Elizabeth

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Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation von Weetall, Marla, Davis, Thomas, Elfring, Gary, Northcutt, Valerie, Cao, Liangxian, Moon, Young-Choon, Riebling, Peter, Dali, Mandar, Hirawat, Samit, Babiak, John, Colacino, Joseph, Almstead, Neil, Spiegel, Robert, Peltz, Stuart W.

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SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy von Naryshkin, Nikolai A., Weetall, Marla, Dakka, Amal, Narasimhan, Jana, Zhao, Xin, Feng, Zhihua, Ling, Karen K. Y., Karp, Gary M., Qi, Hongyan, Woll, Matthew G., Chen, Guangming, Zhang, Nanjing, Gabbeta, Vijayalakshmi, Vazirani, Priya, Bhattacharyya, Anuradha, Furia, Bansri, Risher, Nicole, Sheedy, Josephine, Kong, Ronald, Ma, Jiyuan, Turpoff, Anthony, Lee, Chang-Sun, Zhang, Xiaoyan, Moon, Young-Choon, Trifillis, Panayiota, Welch, Ellen M., Colacino, Joseph M., Babiak, John, Almstead, Neil G., Peltz, Stuart W., Eng, Loren A., Chen, Karen S., Mull, Jesse L., Lynes, Maureen S., Rubin, Lee L., Fontoura, Paulo, Santarelli, Luca, Haehnke, Daniel, McCarthy, Kathleen D., Schmucki, Roland, Ebeling, Martin, Sivaramakrishnan, Manaswini, Ko, Chien-Ping, Paushkin, Sergey V., Ratni, Hasane, Gerlach, Irene, Ghosh, Anirvan, Metzger, Friedrich

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Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties von Cao, Liangxian, Weetall, Marla, Trotta, Christopher, Cintron, Katherine, Ma, Jiyuan, Kim, Min Jung, Furia, Bansri, Romfo, Charles, Graci, Jason D, Li, Wencheng, Du, Joshua, Sheedy, Josephine, Hedrick, Jean, Risher, Nicole, Yeh, Shirley, Qi, Hongyan, Arasu, Tamil, Hwang, Seongwoo, Lennox, William, Kong, Ronald, Petruska, Janet, Moon, Young-Choon, Babiak, John, Davis, Thomas W, Jacobson, Allan, Almstead, Neil G, Branstrom, Art, Colacino, Joseph M, Peltz, Stuart W

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Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models von Liu, Grace, Lipari, Philip, Mollin, Anna, Jung, Stephen, Teplova, Irina, Li, Wencheng, Ying, Lanqing, More, Vijay, Lennox, William, Yeh, Shirley, McGann, Eric, Moon, Young-Choon, Rice, Cari, Huarte, Eduardo, Gruszka, Barbara, Ray, Balmiki, Goodwin, Elizabeth, Buckendahl, Patricia, Yurkow, Edward, Braughton, Bruce, Narasimhan, Jana, Welch, Ellen, Voronin, Gregory, Weetall, Marla

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Chemical modifications of G418 (geneticin): Synthesis of novel readthrough aminoglycosides results in an improved in vitro safety window but no improvements in vivo von Baiazitov, Ramil Y., Friesen, Westley, Johnson, Briana, Mollin, Anna, Sheedy, Josephine, Sierra, Jairo, Weetall, Marla, Branstrom, Arthur, Welch, Ellen, Moon, Young-Choon

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SAR studies toward discovery of emvododstat (PTC299), a potent dihydroorotate dehydrogenase (DHODH) inhibitor von Baiazitov, Ramil Y., Qi, Hongyan, Arasu, Tamil, Lennox, William, Cao, Liangxian, Weetall, Marla, Furia, Bansri, Zhuo, Jin, Choi, Soongyu, Kim, Min Jung, Sheedy, Josephine, Davis, Thomas, Moon, Young-Choon

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Unexpected Observation of the Dimroth Rearrangement in the Ribosylation of 4‑Aminopyrimidines von Baiazitov, Ramil Y, Sydorenko, Nadiya, Ren, Hongyu, Moon, Young-Choon

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Guanidino quinazolines and pyrimidines promote readthrough of premature termination codons in cells with native nonsense mutations von Morrill, Christie, Friesen, Westley J., Babu, Suresh, Baiazitov, Ramil Y., Du, Wu, Karloff, Diane B., Lee, Chang-Sun, Moon, Young-Choon, Ren, Hongyu, Sierra, Jairo, Tomizawa, Yuki, Vazirani, Priya, Welch, Ellen M., Xue, Xiaojiao, Zhuo, Jin

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PTC124 targets genetic disorders caused by nonsense mutations von Chen, Guangming, Jacobson, Allan, Spatrick, Phyllis, Peltz, Stuart W, Conn, M. Morgan, Friesen, Westley J, Almstead, Neil G, Hedrick, Jean, Campbell, Jeffrey A, Mollin, Anna, Kawana, Masataka, Wilde, Richard G, Yeh, Shirley, Sweeney, H. Lee, Trotta, Christopher R, Khan, Atiyya, Welch, Ellen M, Barton, Elisabeth R, Hirawat, Samit, Turpoff, Anthony A, Weetall, Marla, Ren, Hongyu, Risher, Nicole, Tomizawa, Yuki, Northcutt, Valerie J, Miller, Langdon L, He, Feng, Jones, Stephen, Colacino, Joseph M, Feng, Huisheng, Branstrom, Arthur A, Corson, Donald, Zhuo, Jin, Ju, William D, Patel, Meenal, Moon, Young-Choon, Hwang, Seongwoo, Paushkin, Sergey, Babiak, John, Trifillis, Panayiota, Karp, Gary, Takasugi, James

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LC–MS/MS quantification of ataluren and ataluren acyl glucuronide in human plasma/urine: application in clinical studies von Kaushik, Diksha, Ma, Jiyuan, Gu, Guodong, Hwang, Seongwoo, Moon, Young-Choon, Kong, Ronald

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