Ataluren as an agent for therapeutic nonsense suppression

nork Peltz, Stuart W., Morsy, Manal, Welch, Ellen M., Jacobson, Allan
Argitaratua 2012

PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model

nork Du, Ming, Liu, Xiaoli, Welch, Ellen M., Hirawat, Samit, Peltz, Stuart W., Bedwell, David M.
Argitaratua 2008

Nonsense suppression activity of PTC124 (ataluren)

nork Peltz, Stuart W., Welch, Ellen M., Jacobson, Allan, Trotta, Christopher R., Naryshkin, Nikolai, Sweeney, H. Lee, Bedwell, David M.
Argitaratua 2009

In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren

nork Kong, Ronald, Ma, Jiyuan, Hwang, Seongwoo, Moon, Young‐Choon, Welch, Ellen M., Weetall, Marla, Colacino, Joseph M., Almstead, Neil, Babiak, John, Goodwin, Elizabeth
Argitaratua izan da Pharmacol Res Perspect (2020)

Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression

nork Wang, Bingjing, Yang, Zhaohui, Brisson, Becky K., Feng, Huisheng, Zhang, Zhiqian, Welch, Ellen M., Peltz, Stuart W., Barton, Elisabeth R., Brown, Robert H., Sweeney, H. Lee
Argitaratua 2010

Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression

nork 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
Argitaratua izan da Proc Natl Acad Sci U S A (2016)

Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy

nork Zhao, Xin, Feng, Zhihua, Ling, Karen K. Y., Mollin, Anna, Sheedy, Josephine, Yeh, Shirley, Petruska, Janet, Narasimhan, Jana, Dakka, Amal, Welch, Ellen M., Karp, Gary, Chen, Karen S., Metzger, Friedrich, Ratni, Hasane, Lotti, Francesco, Tisdale, Sarah, Naryshkin, Nikolai A., Pellizzoni, Livio, Paushkin, Sergey, Ko, Chien-Ping, Weetall, Marla
Argitaratua izan da Hum Mol Genet (2016)

The nucleoside analog clitocine is a potent and efficacious readthrough agent

nork 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.
Argitaratua izan da RNA (2017)

The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential

nork 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.
Argitaratua izan da PLoS One (2018)

The DHODH Inhibitor PTC299 Arrests SARS-CoV-2 Replication and Suppresses Induction of Inflammatory Cytokines

nork Luban, Jeremy, Sattler, Rachel, Mühlberger, Elke, Graci, Jason D., Cao, Liangxian, Weetall, Marla, Trotta, Christopher, Colacino, Joseph M., Bavari, Sina, Strambio-De-Castillia, Caterina, Suder, Ellen L., Wang, Yetao, Soloveva, Veronica, Cintron-Lue, Katherine, Naryshkin, Nikolai A., Pykett, Mark, Welch, Ellen M., O’Keefe, Kylie, Kong, Ronald, Goodwin, Elizabeth, Jacobson, Allan, Paessler, Slobodan, Peltz, Stuart
Argitaratua izan da bioRxiv (2020)

The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines

nork Luban, Jeremy, Sattler, Rachel A., Mühlberger, Elke, Graci, Jason D., Cao, Liangxian, Weetall, Marla, Trotta, Christopher, Colacino, Joseph M., Bavari, Sina, Strambio-De-Castillia, Caterina, Suder, Ellen L., Wang, Yetao, Soloveva, Veronica, Cintron-Lue, Katherine, Naryshkin, Nikolai A., Pykett, Mark, Welch, Ellen M., O’Keefe, Kylie, Kong, Ronald, Goodwin, Elizabeth, Jacobson, Allan, Paessler, Slobodan, Peltz, Stuart W.
Argitaratua izan da Virus Res (2021)

A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis

nork EitanKerem, Konstan, Michael W., De Boeck, Kris, Accurso, Frank J., Sermet-Gaudelus, Isabelle, Wilschanski, Michael, Elborn, J S, 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, Gary L., Welch, Ellen M., Branstrom, Arthur, Spiegel, Robert J., Peltz, Stuart W., Ajayi, Temitayo, Rowe, Steven M.
Argitaratua 2014