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Development of novel aminoglycoside (NB54) with reduced toxicity and enhanced suppression of disease-causing premature stop mutations.
Nudelman I, Rebibo-Sabbah A, Cherniavsky M, Belakhov V, Hainrichson M, Chen F, Schacht J, Pilch DS, Ben-Yosef T, Baasov T. Nudelman I, et al. J Med Chem. 2009 May 14;52(9):2836-45. doi: 10.1021/jm801640k. J Med Chem. 2009. PMID: 19309154 Free PMC article.
Repairing faulty genes by aminoglycosides: development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations.
Nudelman I, Glikin D, Smolkin B, Hainrichson M, Belakhov V, Baasov T. Nudelman I, et al. Bioorg Med Chem. 2010 Jun 1;18(11):3735-46. doi: 10.1016/j.bmc.2010.03.060. Epub 2010 Mar 27. Bioorg Med Chem. 2010. PMID: 20409719
Designer aminoglycosides: the race to develop improved antibiotics and compounds for the treatment of human genetic diseases.
Hainrichson M, Nudelman I, Baasov T. Hainrichson M, et al. Among authors: nudelman i. Org Biomol Chem. 2008 Jan 21;6(2):227-39. doi: 10.1039/b712690p. Epub 2007 Oct 9. Org Biomol Chem. 2008. PMID: 18174989 Review.
Redesign of aminoglycosides for treatment of human genetic diseases caused by premature stop mutations.
Nudelman I, Rebibo-Sabbah A, Shallom-Shezifi D, Hainrichson M, Stahl I, Ben-Yosef T, Baasov T. Nudelman I, et al. Bioorg Med Chem Lett. 2006 Dec 15;16(24):6310-5. doi: 10.1016/j.bmcl.2006.09.013. Epub 2006 Sep 25. Bioorg Med Chem Lett. 2006. PMID: 16997553
In vitro and ex vivo suppression by aminoglycosides of PCDH15 nonsense mutations underlying type 1 Usher syndrome.
Rebibo-Sabbah A, Nudelman I, Ahmed ZM, Baasov T, Ben-Yosef T. Rebibo-Sabbah A, et al. Among authors: nudelman i. Hum Genet. 2007 Nov;122(3-4):373-81. doi: 10.1007/s00439-007-0410-7. Epub 2007 Jul 25. Hum Genet. 2007. PMID: 17653769
Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54.
Rowe SM, Sloane P, Tang LP, Backer K, Mazur M, Buckley-Lanier J, Nudelman I, Belakhov V, Bebok Z, Schwiebert E, Baasov T, Bedwell DM. Rowe SM, et al. Among authors: nudelman i. J Mol Med (Berl). 2011 Nov;89(11):1149-61. doi: 10.1007/s00109-011-0787-6. Epub 2011 Jul 22. J Mol Med (Berl). 2011. PMID: 21779978 Free PMC article.
Ex vivo treatment with a novel synthetic aminoglycoside NB54 in primary fibroblasts from Rett syndrome patients suppresses MECP2 nonsense mutations.
Vecsler M, Ben Zeev B, Nudelman I, Anikster Y, Simon AJ, Amariglio N, Rechavi G, Baasov T, Gak E. Vecsler M, et al. Among authors: nudelman i. PLoS One. 2011;6(6):e20733. doi: 10.1371/journal.pone.0020733. Epub 2011 Jun 13. PLoS One. 2011. PMID: 21695138 Free PMC article.
Readthrough of nonsense mutations in Rett syndrome: evaluation of novel aminoglycosides and generation of a new mouse model.
Brendel C, Belakhov V, Werner H, Wegener E, Gärtner J, Nudelman I, Baasov T, Huppke P. Brendel C, et al. Among authors: nudelman i. J Mol Med (Berl). 2011 Apr;89(4):389-98. doi: 10.1007/s00109-010-0704-4. Epub 2010 Dec 1. J Mol Med (Berl). 2011. PMID: 21120444 Free PMC article.
Beneficial read-through of a USH1C nonsense mutation by designed aminoglycoside NB30 in the retina.
Goldmann T, Rebibo-Sabbah A, Overlack N, Nudelman I, Belakhov V, Baasov T, Ben-Yosef T, Wolfrum U, Nagel-Wolfrum K. Goldmann T, et al. Among authors: nudelman i. Invest Ophthalmol Vis Sci. 2010 Dec;51(12):6671-80. doi: 10.1167/iovs.10-5741. Epub 2010 Jul 29. Invest Ophthalmol Vis Sci. 2010. PMID: 20671281
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