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Emvododstat, a Potent Dihydroorotate Dehydrogenase Inhibitor, Is Effective in Preclinical Models of Acute Myeloid Leukemia Branstrom, Arthur; Cao, Liangxian; Furia, Bansri; Trotta, Christopher; Santaguida, Marianne; Graci, Jason D.; Colacino, Joseph M.; Ray, Balmiki; Li, Wencheng; Sheedy, Josephine; Mollin, Anna; Yeh, Shirley; Kong, Ronald; Sheridan, Richard; Baird, John D.; O'Keefe, Kylie; Spiegel, Robert; Goodwin, Elizabeth; Keating, Suzanne; Weetall, Marla 分享 收藏
Small molecule splicing modifiers with systemic HTT-lowering activity 具有系统性HTT降低活性的小分子剪接修饰剂 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, Jr.; Southwell, Amber; Hayden, Michael; Colacino, Joseph M.; Peltz, Stuart W. 分享 收藏
ORALLY BIOAVAILABLE SMALL MOLECULE SPLICING MODIFIERS WITH SYSTEMIC AND EVEN HTT-LOWERING ACTIVITY IN VITRO AND IN VIVO Bhattacharyya, Anuradha; Effenberger, Kerstin; Trotta, Christopher; Narasimhan, Jana; Li, Wencheng; Woll, Matthew; Jani, Minakshi; Risher, Nicole; Yeh, Shirley; Cheng, Yaofeng; Sydorenko, Nadiya; Weetall, Marla; Southwell, Amber L.; Hayden, Michael R.; Colacino, Joseph; Peltz, Stuart 分享 收藏
A deep learning approach to identify gene targets of a therapeutic for human splicing disorders Gao, Dadi; Morini, Elisabetta; Salani, Monica; Krauson, Aram J.; Chekuri, Anil; Sharma, Neeraj; Ragavendran, Ashok; Erdin, Serkan; Logan, Emily M.; Li, Wencheng; Dakka, Amal; Narasimhan, Jana; Zhao, Xin; Naryshkin, Nikolai; Trotta, Christopher R.; Effenberger, Kerstin A.; Woll, Matthew G.; Gabbeta, Vijayalakshmi; Karp, Gary; Yu, Yong; Johnson, Graham; Paquette, William D.; Cutting, Garry R.; Talkowski, Michael E.; Slaugenhaupt, Susan A. 分享 收藏
Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical Properties 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. 分享 收藏
The nucleoside analog clitocine is a potent and efficacious readthrough agent Friesen, Westley J.; Trotta, Christopher R.; Tomizawa, Yuki; Zhuo, Jin; Johnson, Briana; Sierra, Jairo; Roy, Bijoyita; Weetall, Marla; Hedrick, Jean; Sheedy, Josephine; Takasugi, James; Moon, Young-Choon; Babu, Suresh; Baiazitov, Ramil; Leszyk, John D.; Davis, Thomas W.; Colacino, Joseph M.; Peltz, Stuart W.; Welch, Ellen M. 分享 收藏
Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression Roy, Bijoyita; Friesen, Westley J.; Tomizawa, Yuki; Leszyk, John D.; Zhuo, Jin; Johnson, Briana; Dakka, Jumana; Trotta, Christopher R.; Xue, Xiaojiao; Mutyam, Venkateshwar; Keeling, Kim M.; Mobley, James A.; Rowe, Steven M.; Bedwell, David M.; Welch, Ellen M.; Jacobson, Allan 分享 收藏
Autosomal-Recessive Mutations in the tRNA Splicing Endonuclease Subunit TSEN15 Cause Pontocerebellar Hypoplasia and Progressive Microcephaly (vol 99, pg 228, 2016) Breuss, Martin W.; Sultan, Tipu; James, Kiely N.; Rosti, Rasim O.; Scott, Eric; Musaev, Damir; Furia, Bansri; Reis, Andre; Sticht, Heinrich; Al-Owain, Mohammed; Alkuraya, Fowzan S.; Reuter, Miriam S.; Abou Jamra, Rami; Trotta, Christopher R.; Gleeson, Joseph G. 分享 收藏
Autosomal- Recessive Mutations in the tRNA Splicing Endonuclease Subunit TSEN15 Cause Pontocerebellar Hypoplasia and Progressive Microcephaly Breuss, Martin W.; Sultan, Tipu; James, Kiely N.; Rosti, Rasim O.; Scott, Eric; Musaev, Damir; Furia, Bansri; Reis, Andre; Sticht, Heinrich; Al-Owain, Mohammed; Alkuraya, Fowzan S.; Reuter, Miriam S.; Abou Jamra, Rami; Trotta, Christopher R.; Gleeson, Joseph G. 分享 收藏
The tRNA Splicing Endonuclease Complex Cleaves the Mitochondria-localized CBP1 mRNA Tsuboi, Tatsuhisa; Yamazaki, Reina; Nobuta, Risa; Ikeuchi, Ken; Makino, Shiho; Ohtaki, Ayumi; Suzuki, Yutaka; Yoshihisa, Tohru; Trotta, Christopher; Inada, Toshifumi 分享 收藏
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