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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Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy
Finkel, Richard S, Flanigan, Kevin M, Wong, Brenda, Bönnemann, Carsten, Sampson, Jacinda, Sweeney, H Lee, Reha, Allen, Northcutt, Valerie J, Elfring, Gary, Barth, Jay, Peltz, Stuart W
Published in PloS one (11.12.2013)
Published in PloS one (11.12.2013)
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The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study
McDonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart W.
Published in Muscle & nerve (01.09.2013)
Published in Muscle & nerve (01.09.2013)
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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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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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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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Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening Platform
Cao, Liangxian, Weetall, Marla, Bombard, Jenelle, Qi, Hongyan, Arasu, Tamil, Lennox, William, Hedrick, Jean, Sheedy, Josephine, Risher, Nicole, Brooks, Peter C, Trifillis, Panayiota, Trotta, Christopher, Moon, Young-Choon, Babiak, John, Almstead, Neil G, Colacino, Joseph M, Davis, Thomas W, Peltz, Stuart W
Published in PloS one (16.12.2016)
Published in PloS one (16.12.2016)
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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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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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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
Published in Antimicrobial Agents and Chemotherapy (01.07.2013)
Published in Antimicrobial Agents and Chemotherapy (01.07.2013)
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Derepression of the Her-2 uORF is mediated by a novel post-transcriptional control mechanism in cancer cells
Mehta, Anuradha, Trotta, Christopher R, Peltz, Stuart W
Published in Genes & development (15.04.2006)
Published in Genes & development (15.04.2006)
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Global Analysis of Pub1p Targets Reveals a Coordinate Control of Gene Expression through Modulation of Binding and Stability
Duttagupta, Radharani, Tian, Bin, Wilusz, Carol J., Khounh, Danny T., Soteropoulos, Patricia, Ouyang, Ming, Dougherty, Joseph P., Peltz, Stuart W.
Published in Molecular and Cellular Biology (01.07.2005)
Published in Molecular and Cellular Biology (01.07.2005)
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Cleavage of pre-tRNAs by the splicing endonuclease requires a composite active site
Li, Hong, Peltz, Stuart W, Paushkin, Sergey V, Trotta, Christopher R, Patel, Meenal
Published in Nature (18.05.2006)
Published in Nature (18.05.2006)
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Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation
Weetall, Marla, Davis, Thomas, Elfring, Gary, Northcutt, Valerie, Cao, Liangxian, Moon, Young-Choon, Riebling, Peter, Dali, Mandar, Hirawat, Samit, Babiak, John, Colacino, Joseph, Almstead, Neil, Spiegel, Robert, Peltz, Stuart W.
Published in Clinical pharmacology in drug development (01.07.2016)
Published in Clinical pharmacology in drug development (01.07.2016)
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The Human Immunodeficiency Virus Type 1 Ribosomal Frameshifting Site Is an Invariant Sequence Determinant and an Important Target for Antiviral Therapy
BISWAS, Preetha, XI JIANG, PACCHIA, Annmarie L, DOUGHERTY, Joseph P, PELTZ, Stuart W
Published in Journal of Virology (01.02.2004)
Published in Journal of Virology (01.02.2004)
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