未登录 分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
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
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 relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy Thangarajh, Mathula; Elfring, Gary L.; Trifillis, Panayiota; McIntosh, Joseph; Peitz, Stuart W.; Ryan, Monique M.; Kornberg, Andrew J.; RodriguezCasero, Victoria; Wray, Alison; Jones, Kristi J.; North, Kathryn; Goemans, Nathalie; Buyse, Gunnar Marceo; Campbell, Craig; Mah, Jean; Sarnat, Harvey; Selby, Kathryn; Voit, Thomas; Doppler, Valerie; De Castro, Denis; Chabrol, Brigitte; Levy, Nicolas; Halbert, Cecile; Pereon, Yann; Magot, Armelle; Perrier, Julie; Mahe, Jean-Yves; Schara, Ulrike; Lutz, Soren; Busse, Melanie; Della Marina, Adela; Kirschner, Janbernd; Stanescu, Angela; Pohl, Annette; RensingZimmerman, Cornelia; Bertini, Enrico; D'Amico, Adele; Kofler, Annamaria; Carlesi, Adelina; Bonetti, Anna Maria; Santecchia, Luigino; Emma, Francesco; Bergami, Gianluca; Mercuri, Eugenio Maria; Vasco, Gessica; Bianco, Flaviana; Mazzone, Elena Stacy; De Sanctis, Roberto; Alfieri, Paolo; Pane, Marika; Messina, Sonia; Comi, Giacomo Pietro; Magri, Francesca; Lucchini, Valeria; Corti, Stefania Paola; Moggio, Maurizio Gualtiero; Sciacco, Monica; Bresolin, Nereo; Prelle, Alessandro Cesare; Magri, Roberta; Virgilio, Roberta; Lamperti, Costanza; Nevo, Yoram; DorWollman, Taia; Vilchez, Juan; Muelas, Nuria; Sevilla, Teresa; Smeyers, Patricia; de la Osa, Alberto; Colomer, Jaume; Ortez, Carlos Ignacio; Nascimento, Andres; Febrer, Ana; Medina, Julita; Tulinus, Mar; Thorarinsdottir, Brynja; Darin, Niklas; Sejersen, Thomas; Hovmoller, Mia; Bushby, Katherine; Straub, Volker; Guglieri, Michela; Sarkozy, Anna; Willis, Tracey; Eagle, Michelle; Mayhew, Anna; Muntoni, Francesco; Cirak, Sebahattin; Manzur, Adnan Yousaf; Robb, Stephanie Ann; Kinali, Maria; Quinlivan, Rosaline Christina Mary; Smith, Martin Richard; Pandey, Rajesh; Wong, Brenda; Collins, James; Finkel, Richard; Bonnemann, Cartsen; Yang, Michele; Foley, Aileen Reghan; Yum, Sabrina; Sampson, Jacinda; Bromberg, Mark; Swoboda, Kathryn; Day, John; Karachunski, Peter; Mathews, Katherine; Bonthius, Daniel; Laubenthal, Karla Sue; Darras, Basil; Kang, Peter; Parson, Julie; Barohn, Richard; Dasouki, Majed; Anderson, Heather; Burns, Jeffrey; Dimachkie, Mazen; Pasnoor, Mamatha; Wang, Yunxia; Ciafaloni, Emma; Heatwole, Chad; Connolly, Anne; Pestronk, Alan; Al-Lozi, Muhammad; Lopate, Glenn; Golumbek, Paul; Sommerville, Brian; Wang, Leo; Wojcicka-Mitchell, Anna; Godbey, Andrew; Harms, Matthew; Varadachary, Arun; Iyadurai, Stanley; Rojas, Luisa; Iannacone, Susan; Khonghatithum, Chaiyos; Sproule, Douglas; De Vivo, Darryl; Constantinescu, Andre; McDonald, Craig; Han, Jay; Ben Renfroe; Russman, Barry; Sussman, Michael; BurnsWechsler, Stephanie; Juel, Vern; Hobson-Webb, Lisa; Smith, Edward 分享 收藏
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial McDonald, Craig M.; Campbell, Craig; Erazo Torricelli, Ricardo; Finkel, Richard S.; Flanigan, Kevin M.; Goemans, Nathalie; Heydemann, Peter; Kaminska, Anna; Kirschner, Janbernd; Muntoni, Francesco; Nascimento Osorio, Andres; Schara, Ulrike; Sejersen, Thomas; Shieh, Perry B.; Sweeney, H. Lee; Topaloglu, Haluk; Tulinius, Mar; Vilchez, Juan J.; Voit, Thomas; Wong, Brenda; Elfring, Gary; Kroger, Hans; Luo, Xiaohui; McIntosh, Joseph; Ong, Tuyen; Riebling, Peter; Souza, Marcio; Spiegel, Robert J.; Peltz, Stuart W.; Mercuri, Eugenio 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
分享 收藏
Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial Kerem, Elton; Konstan, Michael W.; De Boeck, Kris; Accurso, Frank J.; Sermet-Gaudelus, Isabelle; Wilschanski, Michael; Elborn, J. Stuart; Melotti, Paola; Bronsveld, Inez; Fajac, Isabelle; Malfroot, Anne; Rosenbluth, Daniel B.; Walker, Patricia A.; McColley, Susanna A.; Knoop, Christiane; Quattrucci, Serena; Rietschel, Ernst; Zeitlin, Pamela L.; Barth, Jay; Elfring, Guy L.; Welch, Ellen M.; Branstrom, Arthur; Spiegel, Robert J.; Peltz, Stuart W.; Ajayi, Temitayo; Rowe, Steven M. 分享 收藏
Identification of PTC725, an Orally Bioavailable Small Molecule That Selectively Targets the Hepatitis C Virus NS4B Protein Gu, Zhengxian; Graci, Jason D.; Lahser, Frederick C.; Breslin, Jamie J.; Jung, Stephen P.; Crona, James H.; McMonagle, Patricia; Xia, Ellen; Liu, Shaotang; Karp, Gary; Zhu, Jin; Huang, Song; Nomeir, Amin; Weetall, Marla; Almstead, Neil G.; Peltz, Stuart W.; Tong, Xiao; Ralston, Robert; Colacino, Joseph M. 分享 收藏
分享 收藏
High-throughput Screening Uncovers a Compound That Activates Latent HIV-1 and Acts Cooperatively with a Histone Deacetylase (HDAC) Inhibitor Micheva-Viteva, Sofiya; Kobayashi, Yoshifumi; Edelstein, Leonard C.; Pacchia, Annmarie L.; Lee, Hui-Ling Rose; Graci, Jason D.; Breslin, Jamie; Phelan, Bradley D.; Miller, Leia K.; Colacino, Joseph M.; Gu, Zhengxian; Ron, Yacov; Peltz, Stuart W.; Dougherty, Joseph P. 分享 收藏
Ambulatory quantitative waking and sleeping cough assessment in patients with cystic fibrosis Kerem, Eitan; Wilschanski, Michael; Miller, Nilsen L.; Pugatsch, Thea; Cohen, Tali; Blau, Hannah; Rivlin, Joseph; Shoseyov, David; Reha, Allen; Constantine, Scott; Ajayi, Temitayo; Hirawat, Samit; Elfring, Gary L.; Peltz, Stuart W.; Miller, Langdon L. 分享 收藏
Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression Wang, Bingjing; Yang, Zhaohui; Brisson, Becky K.; Feng, Huisheng; Zhang, Zhiqian; Welch, Ellen M.; Peltz, Stuart W.; Barton, Elisabeth R.; Brown, Robert H., Jr.; Sweeney, H. Lee 分享 收藏
.png){kind=icon}
未登录.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
.png){kind=icon}
