Ataluren as an agent for therapeutic nonsense suppression
Peltz, Stuart W, Morsy, Manal, Welch, Ellen M, Jacobson, Allan
Published in Annual review of medicine (14.01.2013)
Published in Annual review of medicine (14.01.2013)
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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
Published in Proceedings of the National Academy of Sciences - PNAS (01.11.2016)
Published in Proceedings of the National Academy of Sciences - PNAS (01.11.2016)
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The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines
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.
Published in Virus research (15.01.2021)
Published in Virus research (15.01.2021)
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Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial
Kerem, Eitan, 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, Gary L, Welch, Ellen M, Branstrom, Arthur, Spiegel, Robert J, Peltz, Stuart W, Ajayi, Temitayo, Rowe, Steven M
Published in The lancet respiratory medicine (01.07.2014)
Published in The lancet respiratory medicine (01.07.2014)
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The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential
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
Published in PloS one (25.10.2018)
Published in PloS one (25.10.2018)
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PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model
Du, Ming, Liu, Xiaoli, Welch, Ellen M, Hirawat, Samit, Peltz, Stuart W, Bedwell, David M
Published in Proceedings of the National Academy of Sciences - PNAS (12.02.2008)
Published in Proceedings of the National Academy of Sciences - PNAS (12.02.2008)
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SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
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
Published in Science (American Association for the Advancement of Science) (08.08.2014)
Published in Science (American Association for the Advancement of Science) (08.08.2014)
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Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder
Voronin, Gregory, Narasimhan, Jana, Gittens, Jamila, Sheedy, Josephine, Lipari, Philip, Peters, Melinda, DeMarco, Steven, Cao, Liangxian, Varganov, Yakov, Kim, Min Jung, Pear, Lisset, Fotouh, Eman, Sinha, Supriya, Ray, Balmiki, Wu, Michael C., Yalamanchili, Padmaja, Southgate, Christopher, Pick, Joseph, Saadipour, Khalil, Jung, Stephen, Lee, Jeanee, Mollin, Anna, Welch, Ellen M., Wu, Zhijian, Weetall, Marla
Published in Molecular therapy (02.10.2024)
Published in Molecular therapy (02.10.2024)
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Guanidino quinazolines and pyrimidines promote readthrough of premature termination codons in cells with native nonsense mutations
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
Published in Bioorganic & medicinal chemistry letters (15.11.2022)
Published in Bioorganic & medicinal chemistry letters (15.11.2022)
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PTC124 targets genetic disorders caused by nonsense mutations
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
Published in Nature (03.05.2007)
Published in Nature (03.05.2007)
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Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset
Feng, Zhihua, Ling, Karen K Y, Zhao, Xin, Zhou, Chunyi, Karp, Gary, Welch, Ellen M, Naryshkin, Nikolai, Ratni, Hasane, Chen, Karen S, Metzger, Friedrich, Paushkin, Sergey, Weetall, Marla, Ko, Chien-Ping
Published in Human molecular genetics (01.03.2016)
Published in Human molecular genetics (01.03.2016)
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In vitro metabolism, pharmacokinetics and drug interaction potentials of emvododstat, a DHODH inhibitor
Ma, Jiyuan, Kaushik, Diksha, Yeh, Shirley, Northcutt, Valerie, Babiak, John, Risher, Nicole, Weetall, Marla, Moon, Young-Choon, Welch, Ellen M., Molony, Lachlan, O'Keefe, Kylie, Kong, Ronald
Published in Xenobiotica (01.02.2022)
Published in Xenobiotica (01.02.2022)
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Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers
Hirawat, Samit, Welch, Ellen M, Elfring, Gary L, Northcutt, Valerie J, Paushkin, Sergey, Hwang, Seongwoo, Leonard, Eileen M, Almstead, Neil G, Ju, William, Peltz, Stuart W, Miller, Langdon L
Published in Journal of clinical pharmacology (01.04.2007)
Published in Journal of clinical pharmacology (01.04.2007)
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Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy
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
Published in Human molecular genetics (15.05.2016)
Published in Human molecular genetics (15.05.2016)
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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
Published in RNA (Cambridge) (01.04.2017)
Published in RNA (Cambridge) (01.04.2017)
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In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren
Kong, Ronald, Ma, Jiyuan, Hwang, Seongwoo, Moon, Young‐Choon, Welch, Ellen M., Weetall, Marla, Colacino, Joseph M., Almstead, Neil, Babiak, John, Goodwin, Elizabeth
Published in Pharmacology research & perspectives (01.04.2020)
Published in Pharmacology research & perspectives (01.04.2020)
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Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression
BINGJING WANG, ZHAOHUI YANG, BRISSON, Becky K, HUISHENG FENG, ZHIQIAN ZHANG, WELCH, Ellen M, PELTZ, Stuart W, BARTON, Elisabeth R, BROWN, Robert H, SWEENEY, H. Lee
Published in Journal of applied physiology (1985) (01.09.2010)
Published in Journal of applied physiology (1985) (01.09.2010)
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