PTC124 targets genetic disorders caused by nonsense mutations (Q34003720)

30 July 2017

title

PTC124 targets genetic disorders caused by nonsense mutations (English)

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30 July 2017

Ellen M Welch

1

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30 July 2017

Elisabeth R Barton

2

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30 July 2017

Jin Zhuo

3

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Yuki Tomizawa

4

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30 July 2017

Westley J Friesen

5

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Panayiota Trifillis

6

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Sergey Paushkin

7

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Meenal Patel

8

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Christopher R Trotta

9

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Seongwoo Hwang

10

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Richard G Wilde

11

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Gary Karp

12

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30 July 2017

James Takasugi

13

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30 July 2017

Guangming Chen

14

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30 July 2017

Stephen Jones

15

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30 July 2017

Hongyu Ren

16

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30 July 2017

Young-Choon Moon

17

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30 July 2017

Donald Corson

18

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Anthony A Turpoff

19

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Jeffrey A Campbell

20

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M Morgan Conn

21

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Atiyya Khan

22

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Neil G Almstead

23

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Jean Hedrick

24

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Anna Mollin

25

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Nicole Risher

26

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Marla Weetall

27

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Shirley Yeh

28

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Arthur A Branstrom

29

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30 July 2017

Joseph M Colacino

30

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John Babiak

31

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William D Ju

32

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Samit Hirawat

33

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Valerie J Northcutt

34

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Langdon L Miller

35

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Phyllis Spatrick

36

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30 July 2017

Feng He

37

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30 July 2017

Masataka Kawana

38

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30 July 2017

Huisheng Feng

39

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30 July 2017

Allan Jacobson

40

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30 July 2017

Stuart W Peltz

41

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30 July 2017

H Lee Sweeney

42

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30 July 2017

language of work or name

English

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publication date

22 April 2007

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30 July 2017

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30 July 2017

volume

447

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30 July 2017

issue

7140

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30 July 2017

page(s)

87-91

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When the message goes awry: disease-producing mutations that influence mRNA content and performance.

30 July 2017

cites work

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https://api.crossref.org/works/10.1038%2FNATURE05756

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Pharmacologic therapy for stop mutations: how much CFTR activity is enough?

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Five Percent of Normal Cystic Fibrosis Transmembrane Conductance Regulator mRNA Ameliorates the Severity of Pulmonary Disease in Cystic Fibrosis

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Nonsense-mediated mRNA decay: splicing, translation and mRNP dynamics

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Early nonsense: mRNA decay solves a translational problem

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7 January 2021

Upf1p control of nonsense mRNA translation is regulated by Nmd2p and Upf3p

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7 January 2021

Genetic and biochemical characterization of mutations in the ATPase and helicase regions of the Upf1 protein

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7 January 2021

Identification and characterization of mutations in the UPF1 gene that affect nonsense suppression and the formation of the Upf protein complex but not mRNA turnover

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

The role of Upf proteins in modulating the translation read-through of nonsense-containing transcripts

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Evidence that systemic gentamicin suppresses premature stop mutations in patients with cystic fibrosis

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Gentamicin-Induced Correction of CFTR Function in Patients with Cystic Fibrosis andCFTRStop Mutations

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

The efficiency of translation termination is determined by a synergistic interplay between upstream and downstream sequences in Saccharomyces cerevisiae.

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Translational termination efficiency in mammals is influenced by the base following the stop codon.

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Sequence specificity of aminoglycoside-induced stop condon readthrough: potential implications for treatment of Duchenne muscular dystrophy

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7 January 2021

Dystrophin protects the sarcolemma from stresses developed during muscle contraction

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Interrelationships of the pathways of mRNA decay and translation in eukaryotic cells

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Nonsense surveillance regulates expression of diverse classes of mammalian transcripts and mutes genomic noise

1 reference

https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

A faux 3'-UTR promotes aberrant termination and triggers nonsense-mediated mRNA decay

1 reference

https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers

1 reference

https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Genome-wide analysis of mRNAs regulated by the nonsense-mediated and 5' to 3' mRNA decay pathways in yeast

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

RefSeq and LocusLink: NCBI gene-centered resources

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

An mRNA surveillance mechanism that eliminates transcripts lacking termination codons

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Exosome-mediated recognition and degradation of mRNAs lacking a termination codon

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Release factor RF-3 GTPase activity acts in disassembly of the ribosome termination complex

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https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Defective point mutants of the encephalomyocarditis virus internal ribosome entry site can be complemented in trans

1 reference

https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

A segment of the 5' nontranslated region of encephalomyocarditis virus RNA directs internal entry of ribosomes during in vitro translation

1 reference

https://api.crossref.org/works/10.1038%2FNATURE05756

7 January 2021

Identifiers

DOI

10.1038/NATURE05756

1 reference

Dimensions Publication ID

30 July 2017

1053507775

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