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Evidence-based path to newborn screening for Duchenne muscular dystrophy. <i>Ann. Neurol.<\/i>, <b>71<\/b>, 304\u2013313 (2012).","DOI":"10.1002\/ana.23528"},{"key":"3","doi-asserted-by":"crossref","unstructured":"3) Mort M, Ivanov D, Cooper DN, Chuzhanova NA. A meta-analysis of nonsense mutations causing human genetic disease. <i>Hum. Mutat.<\/i>, <b>29<\/b>, 1037\u20131047 (2008).","DOI":"10.1002\/humu.20763"},{"key":"4","doi-asserted-by":"crossref","unstructured":"4) Ozawa E, Yoshida M, Suzuki A, Mizuno Y, Hagiwara Y, Noguchi S. Dystrophin-associated proteins in muscular dystrophy. <i>Hum. Mol. Genet.<\/i>, <b>4<\/b> <b>(<\/b>suppl_1<b>)<\/b>, 1711\u20131716 (1995).","DOI":"10.1093\/hmg\/4.suppl_1.1711"},{"key":"5","doi-asserted-by":"crossref","unstructured":"5) Allamand V, Bidou L, Arakawa M, Floquet C, Shiozuka M, Paturneau-Jouas M, Gartioux C, Butler-Browne GS, Mouly V, Rousset JP, Matsuda R, Ikeda D, Guicheney P. Drug-induced readthrough of premature stop codons leads to the stabilization of laminin alpha2 chain mRNA in CMD myotubes. <i>J. Gene Med.<\/i>, <b>10<\/b>, 217\u2013224 (2008).","DOI":"10.1002\/jgm.1140"},{"key":"6","doi-asserted-by":"crossref","unstructured":"6) Howard M, Frizzell RA, Bedwell DM. Aminoglycoside antibiotics restore CFTR function by overcoming premature stop mutations. <i>Nat. Med.<\/i>, <b>2<\/b>, 467\u2013469 (1996).","DOI":"10.1038\/nm0496-467"},{"key":"7","doi-asserted-by":"crossref","unstructured":"7) Martin R, Mogg AE, Heywood LA, Nitschke L, Burke JF. Aminoglycoside suppression at UAG, UAA and UGA codons in Escherichia coli and human tissue culture cells. <i>Mol. Gen. Genet.<\/i>, <b>217<\/b>, 411\u2013418 (1989).","DOI":"10.1007\/BF02464911"},{"key":"8","doi-asserted-by":"crossref","unstructured":"8) Prokhorova I, Altman RB, Djumagulov M, Shrestha JP, Urzhumtsev A, Ferguson A, Chang CT, Yusupov M, Blanchard SC, Yusupova G. Aminoglycoside interactions and impacts on the eukaryotic ribosome. <i>Proc. Natl. Acad. Sci. U.S.A.<\/i>, <b>114<\/b>, E10899\u2013E10908 (2017).","DOI":"10.1073\/pnas.1715501114"},{"key":"9","doi-asserted-by":"crossref","unstructured":"9) Barton-Davis ER, Cordier L, Shoturma DI, Leland SE, Sweeney HL. Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of <i>mdx<\/i> mice. <i>J. Clin. Invest.<\/i>, <b>104<\/b>, 375\u2013381 (1999).","DOI":"10.1172\/JCI7866"},{"key":"10","doi-asserted-by":"crossref","unstructured":"10) Mingeot-Leclercq MP, Tulkens PM. Aminoglycosides: nephrotoxicity. <i>Antimicrob. Agents Chemother.<\/i>, <b>43<\/b>, 1003\u20131012 (1999).","DOI":"10.1128\/AAC.43.5.1003"},{"key":"11","doi-asserted-by":"crossref","unstructured":"11) Hutchin T, Cortopassi G. Proposed molecular and cellular mechanism for aminoglycoside ototoxicity. <i>Antimicrob. Agents Chemother.<\/i>, <b>38<\/b>, 2517\u20132520 (1994).","DOI":"10.1128\/AAC.38.11.2517"},{"key":"12","unstructured":"12) Welch EM, Barton ER, Zhuo J, <i>et al.<\/i> PTC124 targets genetic disorders caused by nonsense mutations. <i>Nature<\/i>, <b>447<\/b>, 87\u201391 (2007)."},{"key":"13","unstructured":"13) \u201cTranslarna: EMA re-confirms non-renewal of authorisation of Duchenne muscular dystrophy medicine.\u201d: &lt;https:\/\/www.ema.europa.eu\/en\/news\/translarna-ema-re-confirms-non-renewal-authorisation-duchenne-muscular-dystrophy-medicine&gt;, accessed 30 October, 2025."},{"key":"14","doi-asserted-by":"crossref","unstructured":"14) Trzaska C, Amand S, Bailly C, <i>et al.<\/i> 2,6-Diaminopurine as a highly potent corrector of UGA nonsense mutations. <i>Nat. Commun.<\/i>, <b>11<\/b>, 1509 (2020).","DOI":"10.1038\/s41467-020-15140-z"},{"key":"15","doi-asserted-by":"crossref","unstructured":"15) Bidou L, Bugaud O, Merer G, Coupet M, Hatin I, Chirkin E, Karri S, Demais S, Fran\u00e7ois P, Cintrat J-C, Namy O. 2-Guanidino-quinazoline promotes the readthrough of nonsense mutations underlying human genetic diseases. <i>Proc. Natl. Acad. Sci. U.S.A.<\/i>, <b>119<\/b>, e2122004119 (2022).","DOI":"10.1073\/pnas.2122004119"},{"key":"16","doi-asserted-by":"crossref","unstructured":"16) Hamada M, Takeuchi T, Kondo S, Ikeda Y, Naganawa H, Maeda K, Okami Y, Umezawa H. A new antibiotic, negamycin. <i>J. Antibiot.<\/i>, <b>23<\/b>, 170\u2013171 (1970).","DOI":"10.7164\/antibiotics.23.170"},{"key":"17","doi-asserted-by":"crossref","unstructured":"17) Arakawa M, Shiozuka M, Nakayama Y, Hara T, Hamada M, Kondo S, Ikeda D, Takahashi Y, Sawa R, Nonomura Y, Sheykholeslami K, Kondo K, Kaga K, Kitamura T, Suzuki-Miyagoe Y, Takeda S, Matsuda R. Negamycin restores dystrophin expression in skeletal and cardiac muscles of <i>mdx<\/i> mice. <i>J. Biochem.<\/i>, <b>134<\/b>, 751\u2013758 (2003).","DOI":"10.1093\/jb\/mvg203"},{"key":"18","doi-asserted-by":"crossref","unstructured":"18) Taguchi A, Hamada K, Kotake M, Shiozuka M, Nakaminami H, Pillaiyar T, Takayama K, Yakushiji F, Noguchi N, Usui T, Matsuda R, Hayashi Y. Discovery of natural products possessing selective eukaryotic readthrough activity: 3-<i>epi<\/i>-deoxynegamycin and its leucine adduct. <i>ChemMedChem<\/i>, <b>9<\/b>, 2233\u20132237 (2014).","DOI":"10.1002\/cmdc.201402208"},{"key":"19","doi-asserted-by":"crossref","unstructured":"19) Kondo S, Yoshida K, Ikeda T, Iinuma K, Honma Y, Hamada M, Umezawa H. 3-<i>epi<\/i>-deoxynegamycin and leucyl-3-<i>epi<\/i>-deoxynegamycin produced by <i>Streptomyces<\/i>. <i>J. Antibiot.<\/i>, <b>30<\/b>, 1137\u20131139 (1977).","DOI":"10.7164\/antibiotics.30.1137"},{"key":"20","doi-asserted-by":"crossref","unstructured":"20) Hamada K, Taguchi A, Kotake M, Aita S, Murakami S, Takayama K, Yakushiji F, Hayashi Y. Structure-activity relationship studies of 3-<i>epi<\/i>-deoxynegamycin derivatives as potent readthrough drug candidates. <i>ACS Med. Chem. Lett.<\/i>, <b>6<\/b>, 689\u2013694 (2015).","DOI":"10.1021\/acsmedchemlett.5b00121"},{"key":"21","doi-asserted-by":"crossref","unstructured":"21) Hamada K, Omura N, Taguchi A, Baradaran-Heravi A, Kotake M, Arai M, Takayama K, Taniguchi A, Roberge M, Hayashi Y. New negamycin-based potent readthrough derivative effective against TGA-type nonsense mutations. <i>ACS Med. Chem. Lett.<\/i>, <b>10<\/b>, 1450\u20131456 (2019).","DOI":"10.1021\/acsmedchemlett.9b00273"},{"key":"22","doi-asserted-by":"crossref","unstructured":"22) Mizuno A, Matsui K, Shuto S. From peptides to peptidomimetics: a strategy based on the structural features of cyclopropane. <i>Chem. Eur. J.<\/i>, <b>23<\/b>, 14394\u201314409 (2017).","DOI":"10.1002\/chem.201702119"},{"key":"23","doi-asserted-by":"crossref","unstructured":"23) Omura N, Taguchi A, Kuwahara T, Hamada K, Watanabe M, Nakakuki M, Konno S, Takayama K, Taniguchi A, Nomura T, Shuto S, Hayashi Y. Development of conformationally restricted negamycin derivatives for potent readthrough activity. <i>ACS Med. Chem. Lett.<\/i>, <b>14<\/b>, 1807\u20131814 (2023).","DOI":"10.1021\/acsmedchemlett.3c00424"},{"key":"24","doi-asserted-by":"crossref","unstructured":"24) Manuvakhova M, Keeling K, Bedwell DM. Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system. <i>RNA<\/i>, <b>6<\/b>, 1044\u20131055 (2000).","DOI":"10.1017\/S1355838200000716"},{"key":"25","doi-asserted-by":"crossref","unstructured":"25) Cridge AG, Crowe-Mcauliffe C, Mathew SF, Tate WP. Eukaryotic translational termination efficiency is influenced by the 3\u2032 nucleotides within the ribosomal mRNA channel. <i>Nucleic Acids Res.<\/i>, <b>46<\/b>, 1927\u20131944 (2018).","DOI":"10.1093\/nar\/gkx1315"},{"key":"26","doi-asserted-by":"crossref","unstructured":"26) Howard MT, Shirts BH, Petros LM, Flanigan KM, Gesteland RF, Atkins JF. Sequence specificity of aminoglycoside-induced stop codon readthrough: potential implications for treatment of Duchenne muscular dystrophy. <i>Ann. Neurol.<\/i>, <b>48<\/b>, 164\u2013169 (2000).","DOI":"10.1002\/1531-8249(200008)48:2<164::AID-ANA5>3.3.CO;2-2"},{"key":"27","doi-asserted-by":"crossref","unstructured":"27) Floquet C, Hatin I, Rousset J-P, Bidou L. Statistical analysis of readthrough levels for nonsense mutations in mammalian cells reveals a major determinant of response to gentamicin. <i>PLoS Genet.<\/i>, <b>8<\/b>, e1002608 (2012).","DOI":"10.1371\/journal.pgen.1002608"},{"key":"28","doi-asserted-by":"crossref","unstructured":"28) Namy O, Hatin I, Rousset JP. Impact of the six nucleotides downstream of the stop codon on translation termination. <i>EMBO Rep.<\/i>, <b>2<\/b>, 787\u2013793 (2001).","DOI":"10.1093\/embo-reports\/kve176"},{"key":"29","doi-asserted-by":"crossref","unstructured":"29) Keeling KM, Bedwell DM. Clinically relevant aminoglycosides can suppress disease-associated premature stop mutations in the <i>IDUA<\/i> and <i>P53<\/i> cDNAs in a mammalian translation system. <i>J. Mol. Med.<\/i>, <b>80<\/b>, 367\u2013376 (2002).","DOI":"10.1007\/s00109-001-0317-z"},{"key":"30","doi-asserted-by":"crossref","unstructured":"30) Bidou L, Hatin I, Perez N, Allamand V, Panthier JJ, Rousset JP. Premature stop codons involved in muscular dystrophies show a broad spectrum of readthrough efficiencies in response to gentamicin treatment. <i>Gene Ther.<\/i>, <b>11<\/b>, 619\u2013627 (2004).","DOI":"10.1038\/sj.gt.3302211"},{"key":"31","doi-asserted-by":"crossref","unstructured":"31) Bonetti B, Fu L, Moon J, Bedwell DM. The efficiency of translation termination is determined by a synergistic interplay between upstream and downstream sequences in <i>Saccharomyces cerevisiae<\/i>. <i>J. Mol. 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