Literature DB >> 21873052

Synthesis and evaluation of compounds that induce readthrough of premature termination codons.

Michael E Jung1, Jin-Mo Ku, Liutao Du, Hailiang Hu, Richard A Gatti.   

Abstract

A structure-activity relationship (SAR) study was carried out to identify novel, small molecular weight compounds which induce readthrough of premature termination codons. In particular, analogs of RTC13, 1, were evaluated. In addition, hypothesizing that these compounds exhibit their activity by binding to the ribosome, we prepared the hybrid analogs 13 containing pyrimidine bases and these also showed good readthrough activity.
Copyright © 2011 Elsevier Ltd. All rights reserved.

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Year:  2011        PMID： 21873052     DOI： 10.1016/j.bmcl.2011.07.107

Source DB:  PubMed          Journal:  Bioorg Med Chem Lett        ISSN： 0960-894X            Impact factor:   2.823


  13 in total

1.  Generating SM(a)RTer compounds for translation termination suppression in A-T and other genetic disorders.

Authors:  Martin F Lavin
Journal:  Mol Ther       Date:  2013-09       Impact factor: 11.454

2.  SMRT compounds abrogate cellular phenotypes of ataxia telangiectasia in neural derivatives of patient-specific hiPSCs.

Authors:  Peiyee Lee; Nathan T Martin; Kotoka Nakamura; Soheila Azghadi; Mandana Amiri; Uri Ben-David; Susan Perlman; Richard A Gatti; Hailiang Hu; William E Lowry
Journal:  Nat Commun       Date:  2013       Impact factor: 14.919

Review 3.  SMRT compounds correct nonsense mutations in primary immunodeficiency and other genetic models.

Authors:  Richard A Gatti
Journal:  Ann N Y Acad Sci       Date:  2012-02       Impact factor: 5.691

4.  Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy.

Authors:  Refik Kayali; Jin-Mo Ku; Gregory Khitrov; Michael E Jung; Olga Prikhodko; Carmen Bertoni
Journal:  Hum Mol Genet       Date:  2012-06-12       Impact factor: 6.150

Review 5.  Suppression of premature termination codons as a therapeutic approach.

Authors:  Kim M Keeling; Dan Wang; Sara E Conard; David M Bedwell
Journal:  Crit Rev Biochem Mol Biol       Date:  2012-06-07       Impact factor: 8.250

6.  A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.

Authors:  Liutao Du; Michael E Jung; Robert Damoiseaux; Gladys Completo; Francesca Fike; Jin-Mo Ku; Shareef Nahas; Cijing Piao; Hailiang Hu; Richard A Gatti
Journal:  Mol Ther       Date:  2013-06-18       Impact factor: 11.454

Review 7.  High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomics.

Authors:  Thomas J J Gintjee; Alvin S H Magh; Carmen Bertoni
Journal:  Biology (Basel)       Date:  2014-11-14

8.  Efficacy of Postnatal In Vivo Nonsense Suppression Therapy in a Pax6 Mouse Model of Aniridia.

Authors:  Xia Wang; Kevin Gregory-Evans; Kishor M Wasan; Olena Sivak; Xianghong Shan; Cheryl Y Gregory-Evans
Journal:  Mol Ther Nucleic Acids       Date:  2017-05-08

9.  Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases.

Authors:  Kim M Keeling
Journal:  Diseases       Date:  2016-10-19

10.  Evaluation of Aminoglycoside and Non-Aminoglycoside Compounds for Stop-Codon Readthrough Therapy in Four Lysosomal Storage Diseases.

Authors:  Marta Gómez-Grau; Elena Garrido; Mónica Cozar; Víctor Rodriguez-Sureda; Carmen Domínguez; Concepción Arenas; Richard A Gatti; Bru Cormand; Daniel Grinberg; Lluïsa Vilageliu
Journal:  PLoS One       Date:  2015-08-19       Impact factor: 3.240
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