Repairing faulty genes by aminoglycosides: Development of new derivatives of geneticin (G418) with enhanced suppression of diseases-causing nonsense mutations

Bioorganic and Medicinal Chemistry

Volumn 18, Issue 11, 2010, Pages 3735-3746

  Nudelman, Igor ^a   Glikin, Dana ^a   Smolkin, Boris ^a   Hainrichson, Mariana ^a   Belakhov, Valery ^a   Baasov, Timor ^a  

^a TECHNION ISRAEL INSTITUTE OF TECHNOLOGY   (Israel)

Author keywords

Aminoglycosides; Cystic fibrosis; Genetic diseases; Nonsense mutations; Translational therapy

Indexed keywords

2',3 DIAMINO 1 N (4 AMINO 2 HYDROXYBUTANOYL) 6' METHYL PAROMAMINE; 2',3 DIAZIDO 1 N (4 AZIDO 2 HYDROXYBUTANOYL) 6' METHYL PAROMAMINE; 2',3 DIAZIDO 1 N [(TERT BUTOXY)CARBONYL] 6' METHYL PAROMAMINE; 3',4',6',6' TETRA O ACETATE 2,'3 DIAZIDO 1 N (4 AZIDO 2 O ACETATE BUTANOYL) 6' METHYL PAROMAMINE; 5 O (5 AZIDO 2,3 O DIBENZOYL 5 DEOXY BETA DEXTRO RIBOFURANOSE) 3',4',6',6', TETRA O ACETATE 1,2',3 DIAZIDO 1 N (4 AZIDO 2 O ACETATE BUTANOYL) 6' METHYL PAROMAMINE; 5 O (5 AZIDO 2,3 O DIBENZOYL 5 DEOXY BETA DEXTRO RIBOFURANOSE) 3',4',6',6', TETRA O ACETATE 1,2',3 TRIAZIDO 6' METHYL PAROMAMINE; 6' METHYL 1,2',3 TRIAZIDO PAROMAMINE; 6' METHYL 5 O (5 AMINO 5 DEOXY BETA DEXTRO RIBOFURANOSE) 2',1,3 TRIAMINO PAROMAMINE; 6' METHYL 5 O (5 AMINO 5 DEOXY BETA DEXTRO RIBOFURANOSE) 2',3 DIAMINO 1 N (4 AMINO 2 HYDROXYBUTANOYL)PAROMAMINE; 6' METHYL PAROMAMINE; AMINOGLYCOSIDE; AMINOGLYCOSIDE ANTIBIOTIC AGENT; ANTIBIOTIC G 418; ANTIINFECTIVE AGENT; DNA; GENTAMICIN; PAROMOMYCIN; TRISACCHARIDE; UNCLASSIFIED DRUG;

ARTICLE; BACTERICIDAL ACTIVITY; COMPETITIVE INHIBITION; CONTROLLED STUDY; CYSTIC FIBROSIS; DRUG CYTOTOXICITY; DRUG DESIGN; DRUG EFFICACY; DRUG STRUCTURE; DRUG SYNTHESIS; DUCHENNE MUSCULAR DYSTROPHY; EX VIVO STUDY; GENETIC DISORDER; HUMAN; HUMAN CELL; HURLER SYNDROME; IN VITRO STUDY; NONSENSE MUTATION; STOP CODON; TOXICITY TESTING; USHER SYNDROME;

AMINOGLYCOSIDES; ANIMALS; CODON, NONSENSE; CYSTIC FIBROSIS; DRUG DESIGN; GENETIC DISEASES, INBORN; GENETIC TECHNIQUES; GENTAMICINS; HUMANS; MUCOPOLYSACCHARIDOSIS I; MUSCULAR DYSTROPHY, DUCHENNE; USHER SYNDROMES;

EID: 77953133544     PISSN: 09680896     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.bmc.2010.03.060     Document Type: Article

References (50)

1. Keeling K.M., and Bedwell D.M. Curr. Pharmacogenomics 3 (2005) 259
2. Kellermayer R. Eur. J. Med. Genet. 49 (2006) 445
3. Zingman L.V., Park S., Olson T.M., Alekseev A.E., and Terzic A. Clin. Pharmacol. Ther. 81 (2007) 99
4. Burke J.F., and Mogg A.E. Nucleic Acids Res. 13 (1985) 6265
5. Bedwell D.M., Kaenjak A., Benos D.J., Bebok Z., Bubien J.K., Hong J., Tousson A., Clancy J.P., and Sorscher E. J. Nat. Med. 3 (1997) 1280
6. Howard M., Frizzell R.A., and Bedwell D.M. Nat. Med. 2 (1996) 467
7. Du M., Jones J.R., Lanier J., Keeling K.M., Lindsey J.R., Tousson A., Bebok Z., Whitsett J.A., Dey C.R., Colledge W.H., Evans M.J., Sorscher E.J., and Bedwell D.M. J. Mol. Med. 80 (2002) 595
8. Barton-Davis E.R., Cordier L., Shoturma D.I., Leland S.E., and Sweeney H.L. J. Clin. Invest. 104 (1999) 375
9. Wilschanski M., Yahav Y., Yaacov Y., Blau H., Bentur L., Rivlin J., Aviram M., Bdolah-Abram T., Bebok Z., Shushi L., Kerem B., and Kerem E. N. Eng. J. Med. 349 (2003) 1433
10. Politano L., Nigro G., Nigro V., Piluso G., Papparella S., Paciello O., and Comi L.I. Acta Myol. 22 (2003) 15
11. Zilberberg A., Lahav L., and Rosin-Arbesfeld R. Gut 59 (2010) 496
12. Brendel C., Klahold E., Gartner J., and Huppke P. Pediatr. Res. 65 (2009) 520
13. Keeling K.M., Brooks D.A., Hopwood J.J., Li P., Thompson J.N., and Bedwell D.M. Hum. Mol. Genet. 10 (2001) 291
14. Sangkuhl K., Schulz A., Rompler H., Yun J., Wess J., and Schoneberg T. Hum. Mol. Genet. 13 (2004) 893
15. Helip-Wooley A., Park M.A., Lemons R.M., and Thoene J.G. Mol. Genet. Metab. 75 (2002) 128
16. Grayson C., Chapple J.P., Willison K.R., Webster A.R., Hardcastle A.J., and Cheetham M.E. J. Med. Genet. 39 (2002) 62
17. Xi B., Guan F., and Lawrence D.S. J. Am. Chem. Soc. 126 (2004) 5660
18. Holbrook J.A., Neu-Yilik G., Hentze M.W., and Kulozik A.E. Nat. Genet. 36 (2004) 801
19. Aguiari G., Banzi M., Gessi S., Cai Y., Zeggio E., Manzati E., Piva R., Lambertini E., Ferrari L., Peters D.J., Lanza F., Harris P.C., Borea P.A., Somlo S., and Del Senno L. FASEB J. 18 (2004) 884
20. Kellermayer R., Szigeti R., Keeling K.M., Bedekovics T., and Bedwell D.M. J. Invest. Dermatol. 126 (2006) 229
21. Olson T.M., Alekseev A.E., Liu X.K., Park S., Zingman L.V., Bienengraeber M., Sattiraju S., Ballew J.D., Jahangir A., and Terzic A. Hum. Mol. Genet. 15 (2006) 2185
22. Porgpermdee S., Yu X., Takagi A., Mayuzumi N., Ogawa H., and Ikeda S. J. Dermatol. Sci. 40 (2005) 137
23. Karpati G., and Lochmuller H. Ann. Neurol. 49 (2001) 693
24. Carter A.P., Clemons W.M., Brodersen D.E., Morgan-Warren R.J., Wimberly B.T., and Ramakrishnan V. Nature 407 (2000) 340
25. Borovinskaya M.A., Pai R.D., Zhang W., Schuwirth B.S., Holton J.M., Hirokawa G., Kaji H., Kaji A., and Cate J.H. Nat. Struct. Mol. Biol. 14 (2007) 727
26. Kondo J., Hainrichson M., Nudelman I., Shallom-Shezifi D., Barbieri C.M., Pilch D.S., Westhof E., and Baasov T. ChemBioChem 8 (2007) 1700
27. Hainrichson M., Nudelman I., and Baasov T. Org. Biomol. Chem. 6 (2008) 227
28. Nudelman I., Rebibo-Sabbah A., Shallom-Shezifi D., Hainrichson M., Stahl I., Ben-Yosef T., and Baasov T. Bioorg. Med. Chem. Lett. 16 (2006) 6310
29. Nudelman I., Rebibo-Sabbah A., Cherniavsky M., Belakhov V., Hainrichson M., Chen F., Schacht J., Pilch D.S., Ben-Yosef T., and Baasov T. J. Med. Chem. 52 (2009) 2836
30. Rebibo-Sabbah A., Nudelman I., Ahmed Z.M., Baasov T., and Ben-Yosef T. Hum. Genet. 122 (2007) 373
31. Recht M.I., Fourmy D., Blanchard S.C., Dahlquist K.D., and Puglisi J.D. J. Mol. Biol. 262 (1996) 421
32. Recht M.I., Douthwaite S., and Puglisi J.D. EMBO J. 18 (1999) 3133
33. Pfister P., Hobbie S., Vicens Q., Bottger E.C., and Westhof E. ChemBioChem 4 (2003) 1078
34. Manuvakhova M., Keeling K., and Bedwell D.M. RNA (New York, NY) 6 (2000) 1044
35. Howard M.T., Shirts B.H., Petros L.M., Flanigan K.M., Gesteland R.F., and Atkins J.F. Ann. Neurol. 48 (2000) 164
36. Chernikov V.G., Terekhov S.M., Krokhina T.B., Shishkin S.S., Smirnova T.D., Kalashnikova E.A., Adnoral N.V., Rebrov L.B., Denisov-Nikol'skii Y.I., and Bykov V.A. Bull. Exp. Biol. Med. 135 (2003) 103
37. Sandoval R.M., Reilly J.P., Running W., Campos S.B., Santos J.R., Phillips C.L., and Molitoris B.A. J. Am. Soc. Nephrol. 17 (2006) 2697
38. Grentzmann G., Ingram J.A., Kelly P.J., Gesteland R.F., and Atkins J.F. RNA (New York, NY) 4 (1998) 479
39. Greenberg W.A., Priestley E.S., Sears P.S., Alper P.B., Rosenbohm C., Hendrix M., Hung S.C., and Wong C.H. J. Am. Chem. Soc. 121 (1999) 6527
40. Howard M.T., Anderson C.B., Fass U., Khatri S., Gesteland R.F., Atkins J.F., and Flanigan K.M. Ann. Neurol. 55 (2004) 422
41. Vicens Q., and Westhof E. Biopolymers 70 (2003) 42
42. Recht M.I., and Puglisi J.D. Antimicrob. Agents Chemother. 45 (2001) 2414
43. 2 group at 6′ position) conferred >200-1000-fold resistance in A1408G mutant.
44. Kondo J., and Westhof E. Nucleic Acids Res. 36 (2008) 2654
45. Bottger E.C., Springer B., Prammananan T., Kidan Y., and Sander P. EMBO Rep. 2 (2001) 318
46. Hobbie S.N., Bruell C.M., Akshay S., Kalapala S.K., Shcherbakov D., and Bottger E.C. Proc. Natl. Acad. Sci. U.S.A. 105 (2008) 3244
47. Alper P.B., Hendrix M., Sears P.S., and Wong C.H. J. Am. Chem. Soc. 120 (1998) 1965
48. Leatherbarrow, R. J. GraFit 5; Horley, U.K.: Erithacus Software Ltd, 2001.
49. NCCLS. National Committee for Clinical Laboratory Standards, Performance standards for antimicrobial susceptibility testing. Fifth information supplement: Approved Standard M100-S5; Villanova, Pa.: NCCLS, 1994.
50. Stark H.J., Baur M., Breitkreutz D., Mirancea N., and Fusenig N.E. J. Invest. Dermatol. 112 (1999) 681