Literature DB >> 16997553

Redesign of aminoglycosides for treatment of human genetic diseases caused by premature stop mutations.

Igor Nudelman1, Annie Rebibo-Sabbah, Dalia Shallom-Shezifi, Mariana Hainrichson, Ido Stahl, Tamar Ben-Yosef, Timor Baasov.   

Abstract

A series of new derivatives of the clinically used aminoglycoside antibiotic paromomycin were designed, synthesized, and their ability to read-through premature stop codon mutations was examined in both in vitro translation system and ex vivo mammalian cultured cells. One of these structures, a pseudo-trisaccharide derivative, showed notably higher stop codon read-through activity in cultured cells compared to those of paromomycin and gentamicin.

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Year:  2006        PMID: 16997553     DOI: 10.1016/j.bmcl.2006.09.013

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


  28 in total

1.  Increased selectivity toward cytoplasmic versus mitochondrial ribosome confers improved efficiency of synthetic aminoglycosides in fixing damaged genes: a strategy for treatment of genetic diseases caused by nonsense mutations.

Authors:  Jeyakumar Kandasamy; Dana Atia-Glikin; Eli Shulman; Katya Shapira; Michal Shavit; Valery Belakhov; Timor Baasov
Journal:  J Med Chem       Date:  2012-11-29       Impact factor: 7.446

2.  When Proteins Start to Make Sense: Fine-tuning Aminoglycosides for PTC Suppression Therapy.

Authors:  Moran Shalev; Timor Baasov
Journal:  Medchemcomm       Date:  2014-08-01       Impact factor: 3.597

3.  A review of patents (2011-2015) towards combating resistance to and toxicity of aminoglycosides.

Authors:  Nishad Thamban Chandrika; Sylvie Garneau-Tsodikova
Journal:  Medchemcomm       Date:  2015-11-19       Impact factor: 3.597

4.  The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse.

Authors:  Dan Wang; Valery Belakhov; Jeyakumar Kandasamy; Timor Baasov; Su-Chen Li; Yu-Teh Li; David M Bedwell; Kim M Keeling
Journal:  Mol Genet Metab       Date:  2011-10-19       Impact factor: 4.797

Review 5.  Comprehensive review of chemical strategies for the preparation of new aminoglycosides and their biological activities.

Authors:  Nishad Thamban Chandrika; Sylvie Garneau-Tsodikova
Journal:  Chem Soc Rev       Date:  2018-02-19       Impact factor: 54.564

6.  The aminoglycoside geneticin permits translational readthrough of the CTNS W138X nonsense mutation in fibroblasts from patients with nephropathic cystinosis.

Authors:  Emma J Brasell; LeeLee Chu; Reyhan El Kares; Jung Hwa Seo; Robin Loesch; Diana M Iglesias; Paul Goodyer
Journal:  Pediatr Nephrol       Date:  2018-11-09       Impact factor: 3.714

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

Review 8.  Nonsense-mediated decay in genetic disease: friend or foe?

Authors:  Jake N Miller; David A Pearce
Journal:  Mutat Res Rev Mutat Res       Date:  2014-05-28       Impact factor: 5.657

9.  Nonaminoglycoside compounds induce readthrough of nonsense mutations.

Authors:  Liutao Du; Robert Damoiseaux; Shareef Nahas; Kun Gao; Hailiang Hu; Julianne M Pollard; Jimena Goldstine; Michael E Jung; Susanne M Henning; Carmen Bertoni; Richard A Gatti
Journal:  J Exp Med       Date:  2009-09-21       Impact factor: 14.307

10.  Reawakening retrocyclins: ancestral human defensins active against HIV-1.

Authors:  Nitya Venkataraman; Amy L Cole; Piotr Ruchala; Alan J Waring; Robert I Lehrer; Olga Stuchlik; Jan Pohl; Alexander M Cole
Journal:  PLoS Biol       Date:  2009-04-28       Impact factor: 8.029

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