Literature DB >> 19506240

Nonsense suppression activity of PTC124 (ataluren).

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

Abstract

Mesh：

Substances：

Year:  2009        PMID： 19506240      PMCID： PMC2700894          DOI： 10.1073/pnas.0901936106

Source DB:  PubMed          Journal:  Proc Natl Acad Sci U S A        ISSN： 0027-8424            Impact factor:   11.205


× No keyword cloud information.
  5 in total

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

Authors:  Ming Du; Xiaoli Liu; Ellen M Welch; Samit Hirawat; Stuart W Peltz; David M Bedwell
Journal:  Proc Natl Acad Sci U S A       Date:  2008-02-06       Impact factor: 11.205

2.  PTC124 targets genetic disorders caused by nonsense mutations.

Authors:  Ellen M Welch; Elisabeth R Barton; Jin Zhuo; Yuki Tomizawa; Westley J Friesen; Panayiota Trifillis; Sergey Paushkin; Meenal Patel; Christopher R Trotta; Seongwoo Hwang; Richard G Wilde; Gary Karp; James Takasugi; Guangming Chen; Stephen Jones; Hongyu Ren; Young-Choon Moon; Donald Corson; Anthony A Turpoff; Jeffrey A Campbell; M Morgan Conn; Atiyya Khan; Neil G Almstead; Jean Hedrick; Anna Mollin; Nicole Risher; Marla Weetall; Shirley Yeh; Arthur A Branstrom; Joseph M Colacino; John Babiak; William D Ju; Samit Hirawat; Valerie J Northcutt; Langdon L Miller; Phyllis Spatrick; Feng He; Masataka Kawana; Huisheng Feng; Allan Jacobson; Stuart W Peltz; H Lee Sweeney
Journal:  Nature       Date:  2007-04-22       Impact factor: 49.962

3.  Characterization of chemical libraries for luciferase inhibitory activity.

Authors:  Douglas S Auld; Noel T Southall; Ajit Jadhav; Ronald L Johnson; David J Diller; Anton Simeonov; Christopher P Austin; James Inglese
Journal:  J Med Chem       Date:  2008-03-26       Impact factor: 7.446

4.  Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression.

Authors:  Douglas S Auld; Natasha Thorne; William F Maguire; James Inglese
Journal:  Proc Natl Acad Sci U S A       Date:  2009-02-10       Impact factor: 11.205

5.  Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.

Authors:  Eitan Kerem; Samit Hirawat; Shoshana Armoni; Yasmin Yaakov; David Shoseyov; Michael Cohen; Malka Nissim-Rafinia; Hannah Blau; Joseph Rivlin; Micha Aviram; Gary L Elfring; Valerie J Northcutt; Langdon L Miller; Batsheva Kerem; Michael Wilschanski
Journal:  Lancet       Date:  2008-08-20       Impact factor: 79.321
  5 in total
  23 in total

1.  Translation drives mRNA quality control.

Authors:  Christopher J Shoemaker; Rachel Green
Journal:  Nat Struct Mol Biol       Date:  2012-06-05       Impact factor: 15.369

Review 2.  Cystic fibrosis chronic rhinosinusitis: a comprehensive review.

Authors:  Mohamad R Chaaban; Alexandra Kejner; Steven M Rowe; Bradford A Woodworth
Journal:  Am J Rhinol Allergy       Date:  2013 Sep-Oct       Impact factor: 2.467

3.  Targeting the activin type IIB receptor to improve muscle mass and function in the mdx mouse model of Duchenne muscular dystrophy.

Authors:  Emidio E Pistilli; Sasha Bogdanovich; Marcus D Goncalves; Rexford S Ahima; Jennifer Lachey; Jasbir Seehra; Tejvir Khurana
Journal:  Am J Pathol       Date:  2011-03       Impact factor: 4.307

4.  Nonsense suppressor therapies rescue peroxisome lipid metabolism and assembly in cells from patients with specific PEX gene mutations.

Authors:  Patricia K Dranchak; Erminia Di Pietro; Ann Snowden; Nathan Oesch; Nancy E Braverman; Steven J Steinberg; Joseph G Hacia
Journal:  J Cell Biochem       Date:  2011-05       Impact factor: 4.429

Review 5.  Suppression of nonsense mutations as a therapeutic approach to treat genetic diseases.

Authors:  Kim M Keeling; David M Bedwell
Journal:  Wiley Interdiscip Rev RNA       Date:  2011-07-06       Impact factor: 9.957

6.  Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124.

Authors:  Douglas S Auld; Scott Lovell; Natasha Thorne; Wendy A Lea; David J Maloney; Min Shen; Ganesha Rai; Kevin P Battaile; Craig J Thomas; Anton Simeonov; Robert P Hanzlik; James Inglese
Journal:  Proc Natl Acad Sci U S A       Date:  2010-03-01       Impact factor: 11.205

Review 7.  Therapeutics based on stop codon readthrough.

Authors:  Kim M Keeling; Xiaojiao Xue; Gwen Gunn; David M Bedwell
Journal:  Annu Rev Genomics Hum Genet       Date:  2014-04-18       Impact factor: 8.929

8.  Aminoglycoside-Induced Premature Stop Codon Read-Through of Mucopolysaccharidosis Type I Patient Q70X and W402X Mutations in Cultured Cells.

Authors:  Makoto Kamei; Karissa Kasperski; Maria Fuller; Emma J Parkinson-Lawrence; Litsa Karageorgos; Valery Belakhov; Timor Baasov; John J Hopwood; Doug A Brooks
Journal:  JIMD Rep       Date:  2013-11-06

Review 9.  Ataluren as an agent for therapeutic nonsense suppression.

Authors:  Stuart W Peltz; Manal Morsy; Ellen M Welch; Allan Jacobson
Journal:  Annu Rev Med       Date:  2012-11-28       Impact factor: 13.739

10.  Firefly luciferase in chemical biology: a compendium of inhibitors, mechanistic evaluation of chemotypes, and suggested use as a reporter.

Authors:  Natasha Thorne; Min Shen; Wendy A Lea; Anton Simeonov; Scott Lovell; Douglas S Auld; James Inglese
Journal:  Chem Biol       Date:  2012-08-24
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.