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Literature DB >> 24022133

Doubts raised over 'read-through' Duchenne drug mechanism.

Cormac Sheridan.

Abstract

Entities: Disease

Mesh：

Substances：
Oxadiazoles
ataluren

Year: 2013 PMID： 24022133 DOI： 10.1038/nbt0913-771

Source DB: PubMed Journal: Nat Biotechnol ISSN： 1087-0156 Impact factor: 54.908

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5 in total

1. A coincidence reporter-gene system for high-throughput screening.

Authors: Ken C-C Cheng; James Inglese
Journal: Nat Methods Date: 2012-10 Impact factor: 28.547

2. 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

3. 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

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. A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays.

Authors: Stuart P McElroy; Toshifumi Nomura; Leah S Torrie; Emma Warbrick; Ulrike Gartner; Gavin Wood; W H Irwin McLean
Journal: PLoS Biol Date: 2013-06-25 Impact factor: 8.029

5 in total

8 in total

1. Characterization of new-generation aminoglycoside promoting premature termination codon readthrough in cancer cells.

Authors: Laure Bidou; Olivier Bugaud; Valery Belakhov; Timor Baasov; Olivier Namy
Journal: RNA Biol Date: 2017-02-01 Impact factor: 4.652

2. EMA reconsiders 'read-through' drug against Duchenne muscular dystrophy following appeal.

Authors: Asher Mullard
Journal: Nat Biotechnol Date: 2014-08 Impact factor: 54.908

Review 3. Small molecules as therapeutic agents for inborn errors of metabolism.

Authors: Leslie Matalonga; Laura Gort; Antonia Ribes
Journal: J Inherit Metab Dis Date: 2016-12-13 Impact factor: 4.982

Review 4. Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.

Authors: Luca Bello; Elena Pegoraro
Journal: Acta Myol Date: 2016-12

5. Effect of Readthrough Treatment in Fibroblasts of Patients Affected by Lysosomal Diseases Caused by Premature Termination Codons.

Authors: Leslie Matalonga; Ángela Arias; Frederic Tort; Xènia Ferrer-Cortés; Judit Garcia-Villoria; Maria Josep Coll; Laura Gort; Antonia Ribes
Journal: Neurotherapeutics Date: 2015-10 Impact factor: 7.620