未登录 Broadening activity of checkpoint blockade agents byintratumoral nucleoside cleavage 通过肿瘤内核苷酸切割扩大检查点阻滞剂的作用范围 Rab, Regina; Hong, Jeong S.; Kinney, Brendan L. C.; Schmitt, Nicole C.; Parker, William B.; Westbrook, Adrianna; Bennion, Kelsey B.; Ford, Mandy L.; Weitzel, Douglas H.; de Marval, Paula L. Miliani; Sorscher, Eric J.; Ehrhardt, Annette 分享 收藏
Barriers to the Pharmacologic Rescue of W1282X CFTR W1282X CFTR的药理学拯救障碍 Manfredi, Candela; Rab, Andras; Joshi, Disha; Winters, Ashlyn G.; Jackson, Janise J.; Molina, Sam; Koval, Michael; Khazanov, Netaly; Jacobson, Madison; Oliver, Kathryn; Senderowitz, Hanoch; Sorscher, Eric J.; Hong, Jeong S. 分享 收藏
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Nanoparticle delivery of a prodrug-activating bacterial enzyme leads to anti-tumor responses 纳米粒递送一种前药激活的细菌酶可诱导抗肿瘤反应 Huayamares, Sebastian G.; Lian, Liming; Rab, Regina; Hou, Yuning; Radmand, Afsane; Kim, Hyejin; Zenhausern, Ryan; Achyut, Bhagelu R.; Ross, Melissa Gilbert; Lokugamage, Melissa P.; Loughrey, David; Peck, Hannah E.; Echeverri, Elisa Schrader; Sanchez, Alejandro J. Da Silva; Shajii, Aram; Li, Andrea; Tiegreen, Karen E.; Santangelo, Philip J.; Sorscher, Eric J.; Dahlman, James E. 分享 收藏
Evaluation of elexacaftor-tezacaftor-ivacaftor treatment in individuals with cystic fibrosis and CFTR N1303K in the USA: a prospective, multicentre, open-label, single-arm trial Solomon, George M.; Linnemann, Rachel W.; Rich, Rachel; Streby, Ashleigh; Buehler, Brian; Hunter, Eric; Vijaykumar, Kadambari; Hunt, William R.; Brewington, John J.; Rab, Andras; Bai, Shasha P.; Westbrook, Adrianna L.; McNicholas-Bevensee, Carmel; Hong, Jong; Manfredi, Candela; Barilla, Cristina; Suzuki, Shingo; Davis, Brian R.; Sorscher, Eric J. 分享 收藏
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Translation velocity determines the efficacy of engineered suppressor tRNAs on pathogenic nonsense mutations Bharti, Nikhil; Santos, Leonardo; Davyt, Marcos; Behrmann, Stine; Eichholtz, Marie; Jimenez-Sanchez, Alejandro; Hong, Jeong S.; Rab, Andras; Sorscher, Eric J.; Albers, Suki; Ignatova, Zoya 分享 收藏
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A Novel 7H-[1,2,4]Triazolo[3,4-b]thiadiazine-based Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Directed toward Treatment of Cystic Fibrosis Rab, Andras; Yang, Xun; Tracy, William F.; Hong, Jeong S.; Joshi, Disha; Manfredi, Candela; Ponnaluri, Sadhana S.; Kolykhalov, Alexander A.; Qui, Min; Fu, Haian; Du, Yuhong; Davies, Huw M. L.; Sorscher, Eric J. 分享 收藏
Interference with Ubiquitination in CFTR Modifies Stability of Core Glycosylated and Cell Surface Pools Lee, Seakwoo; Henderson, Mark J.; Schiffhauer, Eric; Despanie, Jordan; Henry, Katherine; Kang, Po Wei; Walker, Douglas; McClure, Michelle L.; Wilson, Landon; Sorscher, Eric J.; Zeitlin, Pamela L. 分享 收藏
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Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation Oren, Yifat S.; Sinai, Michal Irony-Tur; Golec, Anita; Barchad-Avitzur, Ofra; Mutyam, Venkateshwar; Li, Yao; Hong, Jeong; Ozeri-Galai, Efrat; Hatton, Aurelie; Leibson, Chen; Carmel, Liran; Reiter, Joel; Sorscher, Eric J.; Wilton, Steve D.; Kerem, Eitan; Rowe, Steven M.; Sermet-Gaudelus, Isabelle; Kerem, Batsheva 分享 收藏
Association of cystic fibrosis transmembrane conductance regulator with epithelial sodium channel subunits carrying Liddle's syndrome mutations Rooj, Arun K.; Cormet-Boyaka, Estelle; Clark, Edlira B.; Qadri, Yawar J.; Lee, William; Boddu, Ravindra; Agarwal, Anupam; Tambi, Richa; Uddin, Mohammed; Parpura, Vladimir; Sorscher, Eric J.; Fuller, Cathy M.; Berdiev, Bakhrom K. 分享 收藏
A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletion Sharma, Jyoti; Du, Ming; Wong, Eric; Mutyam, Venkateshwar; Li, Yao; Chen, Jianguo; Wangen, Jamie; Thrasher, Kari; Fu, Lianwu; Peng, Ning; Tang, Liping; Liu, Kaimao; Mathew, Bini; Bostwick, Robert J.; Augelli-Szafran, Corinne E.; Bihler, Hermann; Liang, Feng; Mahiou, Jerome; Saltz, Josef; Rab, Andras; Hong, Jeong; Sorscher, Eric J.; Mendenhall, Eric M.; Coppola, Candice J.; Keeling, Kim M.; Green, Rachel; Mense, Martin; Suto, Mark J.; Rowe, Steven M.; Bedwell, David M. 分享 收藏
Stability Prediction for Mutations in the Cytosolic Domains of Cystic Fibrosis Transmembrane Conductance Regulator Bahia, Malkeet Singh; Khazanov, Netaly; Zhou, Qingxian; Yang, Zhengrong; Wang, Chi; Hong, Jeong S.; Rab, Andras; Sorscher, Eric J.; Brouillette, Christie G.; Hunt, John F.; Senderowitz, Hanoch 分享 收藏
Positive epistasis between disease-causing missense mutations and silent polymorphism with effect on mRNA translation velocity 致病错义突变和沉默多态性对mRNA翻译速度的影响 Rauscher, Robert; Bampi, Giovana B.; Guevara-Ferrer, Marta; Santos, Leonardo A.; Joshi, Disha; Mark, David; Strug, Lisa J.; Rommens, Johanna M.; Ballmann, Manfred; Sorscher, Eric J.; Oliver, Kathryn E.; Ignatova, Zoya 分享 收藏
The CFTR P67L variant reveals a key role for N-terminal lasso helices in channel folding, maturation, and pharmacologic rescue Sabusap, Carleen Mae; Joshi, Disha; Simhaev, Luba; Oliver, Kathryn E.; Senderowitz, Hanoch; van Willigen, Marcel; Braakman, Ineke; Rab, Andras; Sorscher, Eric J.; Hong, Jeong S. 分享 收藏
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