{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T19:45:41Z","timestamp":1792179941597,"version":"4.4.0"},"reference-count":86,"publisher":"MDPI AG","issue":"13","license":[{"start":{"date-parts":[[2019,7,6]],"date-time":"2019-07-06T00:00:00Z","timestamp":1562371200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100008385","name":"Fondazione per la Ricerca sulla Fibrosi Cistica","doi-asserted-by":"publisher","award":["FFC#3\/2017"],"award-info":[{"award-number":["FFC#3\/2017"]}],"id":[{"id":"10.13039\/501100008385","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Fondazione 3\u00b0 Pilastro Internazionale","award":["2018"],"award-info":[{"award-number":["2018"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>This review focuses on the use of oxadiazoles as translational readthrough-inducing drugs (TRIDs) to rescue the functional full-length protein expression in mendelian genetic diseases caused by nonsense mutations. These mutations in specific genes generate premature termination codons (PTCs) responsible for the translation of truncated proteins. After a brief introduction on nonsense mutations and their pathological effects, the features of various classes of TRIDs will be described discussing differences or similarities in their mechanisms of action. Strategies to correct the PTCs will be presented, particularly focusing on a new class of Ataluren-like oxadiazole derivatives in comparison to aminoglycosides. Additionally, recent results on the efficiency of new candidate TRIDs in restoring the production of the cystic fibrosis transmembrane regulator (CFTR) protein will be presented. Finally, a prospectus on complementary strategies to enhance the effect of TRIDs will be illustrated together with a conclusive paragraph about perspectives, opportunities, and caveats in developing small molecules as TRIDs.<\/jats:p>","DOI":"10.3390\/ijms20133329","type":"journal-article","created":{"date-parts":[[2019,7,8]],"date-time":"2019-07-08T03:01:31Z","timestamp":1562554891000},"page":"3329","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":49,"title":["Strategies against Nonsense: Oxadiazoles as Translational Readthrough-Inducing Drugs (TRIDs)"],"prefix":"10.3390","volume":"20","author":[{"given":"Ambra","family":"Campofelice","sequence":"first","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 Degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Lentini","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 Degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4760-7179","authenticated-orcid":false,"given":"Aldo","family":"Di Leonardo","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 Degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4463-8156","authenticated-orcid":false,"given":"Raffaella","family":"Melfi","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 Degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5059-8686","authenticated-orcid":false,"given":"Marco","family":"Tutone","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 Degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1992-2024","authenticated-orcid":false,"given":"Andrea","family":"Pace","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 Degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5885-164X","authenticated-orcid":false,"given":"Ivana","family":"Pibiri","sequence":"additional","affiliation":[{"name":"Dipartimento di Scienze e Tecnologie Biologiche, Chimiche e Farmaceutiche (STEBICEF), Universit\u00e0 Degli Studi di Palermo, Viale delle Scienze Ed. 16-17, 90128 Palermo, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2019,7,6]]},"reference":[{"key":"ref_1","first-page":"415","article-title":"Central dogma, selfish DNA and noncoding Rnas: A historical perspective","volume":"84","author":"Lakhotia","year":"2018","journal-title":"Proc. Indian Natl. Sci. Acad."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"561","DOI":"10.1038\/227561a0","article-title":"Central Dogma of Molecular Biology","volume":"227","author":"Crick","year":"1970","journal-title":"Nature"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1016\/j.synbio.2017.09.003","article-title":"Transcription control engineering and applications in synthetic biology","volume":"2","author":"Engstrom","year":"2017","journal-title":"Synth. Syst. Biotechnol."},{"key":"ref_4","doi-asserted-by":"crossref","unstructured":"Litwack, G. (2018). Protein Biosynthesis. Human Biochemistry, Publisher. Chapter 11.","DOI":"10.1016\/B978-0-12-383864-3.00011-9"},{"key":"ref_5","unstructured":"Rimoin, D., Pyeritz, R., and Bruce, K. (2013). Human Gene Mutation in Inherited Disease: Molecular Mechanisms and Clinical Consequences. Emery Rimoin\u2019s Princ Pract Med Genet, Elsevier. [6th ed.]. Chapter 7."},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1146\/annurev-med-120611-144851","article-title":"Ataluren as an Agent for Therapeutic Nonsense Suppression","volume":"64","author":"Peltz","year":"2013","journal-title":"Annu. Rev. Med."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"175","DOI":"10.5483\/BMBRep.2017.50.4.015","article-title":"Nonsense-mediated mRNA decay at the crossroads of many cellular pathways","volume":"50","author":"Lejeune","year":"2017","journal-title":"BMB Rep."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"79","DOI":"10.4172\/1948-593X.1000086","article-title":"Repurposing Amlexanox as a \u2018Run the Red Light Cure-All\u2019 with Read-Through\u2014A \u2018No-Nonsense\u2019 Approach to Personalised Medicine","volume":"5","author":"Loudon","year":"2013","journal-title":"J. Bioanal. Biomed."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"1285","DOI":"10.1021\/acsmedchemlett.8b00472","article-title":"New in Vitro Assay Measuring Direct Interaction of Nonsense Suppressors with the Eukaryotic Protein Synthesis Machinery","volume":"9","author":"Ng","year":"2018","journal-title":"ACS Med. Chem. Lett."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1055\/s-0038-1675626","article-title":"Diagnostic Pathway to Nonsense Mutation Dystrophinopathy: A Tertiary-Center, Retrospective Experience","volume":"50","year":"2019","journal-title":"Neuropediatrics"},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1038\/sj.ejhg.5200599","article-title":"Premature termination mutations in exon 3 of the SMN1 gene are associated with exon skipping and a relatively mild SMA phenotype","volume":"9","author":"Sossi","year":"2001","journal-title":"Eur. J. Hum. Genet."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1038\/s10038-018-0553-5","article-title":"A novel mutation in VRK1 associated with distal spinal muscular atrophy","volume":"64","author":"Li","year":"2019","journal-title":"J. Hum. Genet."},{"key":"ref_13","first-page":"759","article-title":"Mice with missense and nonsense NF1 mutations display divergent phenotypes compared with human neurofibromatosis type I","volume":"9","author":"Li","year":"2016","journal-title":"Dis. Model. Mech."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"5774","DOI":"10.1093\/hmg\/ddu291","article-title":"New syndrome with retinitis pigmentosa is caused by nonsense mutations in retinol dehydrogenase RDH11","volume":"23","author":"Xie","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"76","DOI":"10.1016\/j.ajo.2018.12.009","article-title":"Time Course of Disease Progression of PRPF31-mediated Retinitis Pigmentosa","volume":"200","author":"Kiser","year":"2019","journal-title":"Am. J. Ophthalmol."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"668","DOI":"10.1016\/j.bbadis.2017.12.014","article-title":"Amlexanox provides a potential therapy for nonsense mutations in the lysosomal storage disorder Aspartylglucosaminuria","volume":"1864","author":"Banning","year":"2018","journal-title":"Biochim. Biophys. Acta Mol. Basis Dis."},{"key":"ref_17","first-page":"3441","article-title":"Clinical diagnosis and genetic counseling of atypical ataxia-telangiectasia in a Chinese family","volume":"19","author":"Cao","year":"2019","journal-title":"Mol. Med. Rep."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"294","DOI":"10.3103\/S0095452718040023","article-title":"Novel Mutation in the MECP2 Gene Identified in a Group of Rett Syndrome Patients from Ukraine","volume":"52","author":"Chernushyn","year":"2018","journal-title":"Cytol. Genet."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1002\/ajh.25025","article-title":"Ataluren-driven restoration of Shwachman-Bodian-Diamond syndrome protein function in Shwachman-Diamond syndrome bone marrow cells","volume":"93","author":"Bezzerri","year":"2017","journal-title":"Am. J. Hematol."},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1089\/hum.2010.067","article-title":"PTC124-Mediated Translational Readthrough of a Nonsense Mutation Causing Usher Syndrome Type 1C","volume":"22","author":"Goldmann","year":"2011","journal-title":"Hum. Gene Ther."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"3043","DOI":"10.1182\/blood-2005-03-1307","article-title":"Aminoglycoside Suppression of Nonsense Mutations in Severe Hemophilia","volume":"106","author":"James","year":"2005","journal-title":"Blood"},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"1092","DOI":"10.1039\/C4MD00081A","article-title":"When Proteins Start to Make Sense: Fine-tuning Aminoglycosides for PTC Suppression Therapy","volume":"5","author":"Shalev","year":"2014","journal-title":"Medchemcomm"},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"667","DOI":"10.1016\/j.molmed.2012.09.004","article-title":"Cancer syndromes and therapy by stop-codon readthrough","volume":"18","author":"Santos","year":"2012","journal-title":"Trends Mol. Med."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s10020-018-0024-7","article-title":"Advances in therapeutic use of a drug-stimulated translational readthrough of premature termination codons","volume":"24","author":"Dabrowski","year":"2018","journal-title":"Mol. Med."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1080\/23808993.2016.1146077","article-title":"Precise treatment of cystic fibrosis\u2013current treatments and perspectives for using CRISPR","volume":"1","author":"Colemeadow","year":"2016","journal-title":"Expert Rev. Precis. Med. Drug Dev."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"10","DOI":"10.1016\/j.jcf.2017.11.018","article-title":"Gene editing & stem cells","volume":"17","author":"Harrison","year":"2018","journal-title":"J. Cyst. Fibros."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1016\/j.stem.2013.11.002","article-title":"Functional repair of CFTR by CRISPR\/Cas9 in intestinal stem cell organoids of cystic fibrosis patients","volume":"13","author":"Schwank","year":"2013","journal-title":"Cell Stem Cell"},{"key":"ref_28","doi-asserted-by":"crossref","first-page":"453","DOI":"10.1016\/j.omtn.2018.05.010","article-title":"VSV-G-Enveloped Vesicles for Traceless Delivery of CRISPR-Cas9","volume":"12","author":"Montagna","year":"2018","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"236","DOI":"10.1016\/j.ejmech.2015.06.038","article-title":"Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives","volume":"101","author":"Pibiri","year":"2015","journal-title":"Eur. J. Med. Chem."},{"key":"ref_30","doi-asserted-by":"crossref","unstructured":"Midgley, J. A breakthrough in readthrough? Could geneticin lead the way to effective treatment for cystinosis nonsense mutations?. Pediatr. Nephrol., 2019.","DOI":"10.1007\/s00467-018-4173-2"},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1007\/s40259-016-0157-6","article-title":"Targeting Nonsense Mutations in Diseases with Translational Read-Through-Inducing Drugs (TRIDs)","volume":"30","author":"Penner","year":"2016","journal-title":"BioDrugs"},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"1044","DOI":"10.1017\/S1355838200000716","article-title":"Aminoglycoside antibiotics mediate context-dependent suppression of termination codons in a mammalian translation system","volume":"6","author":"Manuvakhova","year":"2000","journal-title":"RNA"},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1038\/nature05756","article-title":"PTC124 targets genetic disorders caused by nonsense mutations","volume":"447","author":"Welch","year":"2007","journal-title":"Nature"},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1016\/j.ejmech.2016.06.048","article-title":"Exploring the readthrough of nonsense mutations by non-acidic Ataluren analogues selected by ligand-based virtual screening","volume":"122","author":"Pibiri","year":"2016","journal-title":"Eur. J. Med. Chem."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1016\/j.ejmech.2018.09.057","article-title":"Rescuing the CFTR protein function: Introducing 1,3,4-oxadiazoles as translational readthrough inducing drugs","volume":"159","author":"Pibiri","year":"2018","journal-title":"Eur. J. Med. Chem."},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1371\/journal.pone.0206158","article-title":"The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential","volume":"13","author":"Friesen","year":"2018","journal-title":"PLoS ONE"},{"key":"ref_37","doi-asserted-by":"crossref","unstructured":"Leubitz, A., Frydman-Marom, A., Sharpe, N., van Duzer, J., Campbell, K.C.M., and Vanhoutte, F. (2019). Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers. Clin. Pharmacol. Drug Dev., 1\u201311.","DOI":"10.1002\/cpdd.647"},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"378","DOI":"10.1080\/15476286.2017.1285480","article-title":"Characterization of new-generation aminoglycoside promoting premature termination codon readthrough in cancer cells","volume":"14","author":"Bidou","year":"2017","journal-title":"RNA Biol."},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"355","DOI":"10.3892\/ijmm.2014.1809","article-title":"Therapeutic suppression of premature termination codons: Mechanisms and clinical considerations (Review)","volume":"34","author":"Karijolich","year":"2014","journal-title":"Int. J. Mol. Med."},{"key":"ref_40","doi-asserted-by":"crossref","unstructured":"Floquet, C., Hatin, I., Rousset, J.P., and Bidou, L. (2012). Statistical analysis of readthrough levels for nonsense mutations in mammalian cells reveals a major determinant of response to gentamicin. PLoS Genet., 8.","DOI":"10.1371\/journal.pgen.1002608"},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"805","DOI":"10.1165\/rcmb.2013-0282OC","article-title":"Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor","volume":"50","author":"Xue","year":"2014","journal-title":"Am. J. Respir. Cell Mol. Biol."},{"key":"ref_42","first-page":"211","article-title":"1, 2, 3-Oxadiazoles","volume":"Volume 5","author":"Katritzky","year":"2008","journal-title":"Comprehensive Heterocyclic Chemistry III"},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1016\/bs.aihch.2015.05.001","article-title":"Recent Advances in the Chemistry of 1, 2, 4-Oxadiazoles","volume":"Volume 116","author":"Scriven","year":"2015","journal-title":"Advances Heterocyclic Chemistry"},{"key":"ref_44","doi-asserted-by":"crossref","first-page":"4337","DOI":"10.1039\/b908937c","article-title":"The new era of 1, 2, 4-oxadiazoles","volume":"7","author":"Pace","year":"2009","journal-title":"Org. Biomol. Chem."},{"key":"ref_45","first-page":"229","article-title":"1, 2, 5-Oxadiazoles","volume":"Volume 4","author":"Katritzky","year":"2009","journal-title":"Comprehensive Heterocyclic Chemistry II"},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"1805","DOI":"10.1080\/00397911.2017.1360911","article-title":"Updates on synthesis and biological activities of 1, 3, 4-oxadiazole: A review","volume":"47","author":"Salahuddin","year":"2017","journal-title":"Synth. Commun."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"8596","DOI":"10.1039\/C6TC03003C","article-title":"1, 3, 4-Oxadiazoles as luminescent materials for organic light emitting diodes via cross-coupling reactions","volume":"4","author":"Paun","year":"2016","journal-title":"J. Mater. Chem. C"},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"936","DOI":"10.1007\/s10593-017-2154-1","article-title":"Rearrangements of 1, 2, 4-Oxadiazole: \u201cOne Ring to Rule Them All\u201d","volume":"53","author":"Pace","year":"2017","journal-title":"Chem. Heterocycl. Compd."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"22894","DOI":"10.1039\/c4ra01502a","article-title":"Perfluorocarbon functionalized hyaluronic acid derivatives as oxygenating systems for cell culture","volume":"4","author":"Palumbo","year":"2014","journal-title":"RSC Adv."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"14306","DOI":"10.1039\/c2cp42467c","article-title":"Oxadiazolyl-pyridines and perfluoroalkyl-carboxylic acids as building blocks for protic ionic liquids: Crossing the thin line between ionic and hydrogen bonded materials","volume":"14","author":"Pibiri","year":"2012","journal-title":"Phys. Chem. Chem. Phys."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"1935","DOI":"10.1002\/jhet.2509","article-title":"Synthesis of Fluorinated Bent-Core Mesogens (BCMs) Containing the 1, 2, 4-Oxadiazole Ring","volume":"53","author":"Calabrese","year":"2016","journal-title":"J. Heterocycl. Chem."},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"3044","DOI":"10.1039\/c2ob07024c","article-title":"Synthesis of fluorinated oxadiazoles with gelation and oxygen storage ability","volume":"10","author":"Guarcello","year":"2012","journal-title":"Org. Biomol. Chem."},{"key":"ref_53","doi-asserted-by":"crossref","unstructured":"Pibiri, I., Beneduci, A., Carraro, M., Causin, V., Casella, G., Corrente, G.A., Chidichimo, G., Pace, A., Riccobono, A., and Saielli, G. (2019). Mesomorphic and electrooptical properties of viologens based on non-symmetric alkyl\/polyfluoroalkyl functionalization and on an oxadiazolyl-extended bent core. J. Mater. Chem. C.","DOI":"10.1039\/C9TC01697J"},{"key":"ref_54","first-page":"603","article-title":"Review on Energetic Compounds Based on 1, 2, 4-Oxadiazoles","volume":"25","author":"Huang","year":"2017","journal-title":"Hanneng Cailiao\/Chin. J. Energetic"},{"key":"ref_55","doi-asserted-by":"crossref","unstructured":"Fouad, F.S., Ness, T., Wang, K., Ruth, C.E., Britton, S., and Twieg, R.J. (2019). Biphenylyl-1, 2, 4-oxadiazole based liquid crystals\u2013synthesis, mesomorphism, effect of lateral monofluorination. Liq. Cryst.","DOI":"10.1080\/02678292.2019.1623335"},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"441","DOI":"10.1016\/j.ejmech.2012.02.002","article-title":"Synthesis and preliminary antibacterial evaluation of Linezolid-like 1, 2, 4-oxadiazole derivatives","volume":"50","author":"Musumeci","year":"2012","journal-title":"Eur. J. Med. Chem."},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"3672","DOI":"10.1016\/j.tetlet.2017.08.020","article-title":"Room-temperature synthesis of pharmaceutically important carboxylic acids bearing the 1, 2, 4-oxadiazole moiety","volume":"58","author":"Tarasenko","year":"2017","journal-title":"Tetrahedron Lett."},{"key":"ref_58","doi-asserted-by":"crossref","unstructured":"Tarasenko, M., Sidneva, V., Belova, A., Romanycheva, A., Sharonova, T., Baykov, S., Shetnev, A., Kofanov, E., and Kuznetsov, M.A. (2018). An efficient synthesis and antimicrobial evaluation of 5-alkenyl-and 5-styryl-1, 2, 4-oxadiazoles. Arkivoc, 458\u2013470.","DOI":"10.24820\/ark.5550190.p010.760"},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"1817","DOI":"10.1021\/jm2013248","article-title":"Oxadiazoles in Medicinal Chemistry","volume":"55","author":"Hogner","year":"2012","journal-title":"J. Med. Chem."},{"key":"ref_60","first-page":"3758","article-title":"The recent introduction in therapy of a new antitussive drug: Oxolamine","volume":"52","author":"Rottini","year":"1961","journal-title":"Minerva Med."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"5843","DOI":"10.1021\/jm800245z","article-title":"Discovery of Raltegravir, a Potent, Selective Orally Bioavailable HIV-Integrase Inhibitor for the Treatment of HIV-AIDS Infection","volume":"51","author":"Summa","year":"2008","journal-title":"J. Med. Chem."},{"key":"ref_62","doi-asserted-by":"crossref","first-page":"624","DOI":"10.1358\/dof.2009.034.08.1400202","article-title":"Zibotentan","volume":"34","author":"James","year":"2009","journal-title":"Drugs Fut."},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"430","DOI":"10.1177\/0091270006297140","article-title":"Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single- and Multiple-Dose Administration to Healthy Male and Female Adult Volunteers","volume":"47","author":"Hirawat","year":"2007","journal-title":"J. Clin. Pharmacol."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1016\/j.nmd.2014.11.011","article-title":"European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene","volume":"25","author":"Haas","year":"2015","journal-title":"Neuromuscul. Disord."},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1016\/S2213-2600(14)70100-6","article-title":"Ataluren for the treatment of nonsense-mutation cystic fibrosis: A randomised, double-blind, placebo-controlled phase 3 trial","volume":"2","author":"Kerem","year":"2014","journal-title":"Lancet Respir. Med."},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"12353","DOI":"10.1073\/pnas.1615548113","article-title":"Proposing a mechanism of action for ataluren","volume":"113","author":"Siddiqui","year":"2016","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"3585","DOI":"10.1073\/pnas.0813345106","article-title":"Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression","volume":"106","author":"Auld","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"4878","DOI":"10.1073\/pnas.0909141107","article-title":"Molecular basis for the high-affinity binding and stabilization of firefly luciferase by PTC124","volume":"107","author":"Auld","year":"2010","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"3038","DOI":"10.1073\/pnas.1424127112","article-title":"Nonsense suppression by near-cognate tRNAs employs alternative base pairing at codon positions 1 and 3","volume":"112","author":"Roy","year":"2015","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"12508","DOI":"10.1073\/pnas.1605336113","article-title":"Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression","volume":"113","author":"Roy","year":"2016","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1021\/acsmedchemlett.8b00558","article-title":"Deciphering the Nonsense Readthrough Mechanism of Action of Ataluren: An in Silico Compared Study","volume":"10","author":"Tutone","year":"2019","journal-title":"ACS Med. Chem. Lett."},{"key":"ref_72","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1021\/mp400230s","article-title":"Toward a Rationale for the PTC124 (Ataluren) Promoted Readthrough of Premature Stop Codons: A Computational Approach and GFP-Reporter Cell-Based Assay","volume":"11","author":"Lentini","year":"2014","journal-title":"Mol. Pharm."},{"key":"ref_73","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1016\/S0165-022X(97)00028-6","article-title":"Ussing chamber for high-frequency transmural impedance analysis of epithelial tissues","volume":"35","author":"Gitter","year":"1997","journal-title":"J. Biochem. Biophys. Methods"},{"key":"ref_74","doi-asserted-by":"crossref","first-page":"1073","DOI":"10.1177\/002215549804600911","article-title":"Simultaneous Visualization of the Yellow and Green Forms of the Green Fluorescent Protein in Living Cells","volume":"46","author":"Baumann","year":"1998","journal-title":"J. Histochem. Cytochem."},{"key":"ref_75","first-page":"1","article-title":"Rescue of nonsense mutations by amlexanox in human cells","volume":"7","author":"Beghyn","year":"2012","journal-title":"Orphanet J. Rare Dis."},{"key":"ref_76","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1159\/000233861","article-title":"Mechanism of the action of Amoxanox (AA-673) an Orally Active Antiallergic Agent","volume":"78","author":"Saijo","year":"1985","journal-title":"Int. Archs Allergy Appl. Immun."},{"key":"ref_77","doi-asserted-by":"crossref","unstructured":"Meng, W., Dong, Y., Liu, J., Wang, Z., Zhong, X., Chen, R., Zhou, H., Lin, M., Jiang, L., and Gao, F. (2009). A clinical evaluation of amlexanox oral adhesive pellicles in the treatment of recurrent aphthous stomatitis and comparison with amlexanox oral tablets: A randomized, placebo controlled, blinded, multicenter clinical trial. Trials, 10.","DOI":"10.1186\/1745-6215-10-30"},{"key":"ref_78","doi-asserted-by":"crossref","first-page":"567","DOI":"10.1261\/rna.060236.116","article-title":"The nucleoside analog clitocine is a potent and efficacious readthrough agent","volume":"23","author":"Friesen","year":"2017","journal-title":"RNA"},{"key":"ref_79","doi-asserted-by":"crossref","unstructured":"Lueck, J.D., Yoon, J.S., Perales-Puchalt, A., Mackey, A.L., Infield, D.T., Behlke, M.A., Pope, M.R., Weiner, D.B., Skach, W.R., and McCray, P.B. (2019). Engineered transfer RNAs for suppression of premature termination codons. Nat. Commun., 10.","DOI":"10.1038\/s41467-019-08329-4"},{"key":"ref_80","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1038\/nature10913","article-title":"A new understanding of the decoding principle on the ribosome","volume":"484","author":"Demeshkina","year":"2012","journal-title":"Nature"},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"13333","DOI":"10.1073\/pnas.1307365110","article-title":"Identification of the molecular attributes required for aminoglycoside activity against Leishmania","volume":"110","author":"Shalev","year":"2013","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_82","doi-asserted-by":"crossref","unstructured":"Keeling, K., Wang, D., Dai, Y., Murugesan, S., Chenna, B., Clark, J., Belakhov, V., Kandasamy, J., Velu, S., and Baasov, T. (2013). Attenuation of Nonsense-Mediated mRNA Decay Enhances In Vivo Nonsense Suppression. PLoS ONE, 8.","DOI":"10.1371\/journal.pone.0060478"},{"key":"ref_83","doi-asserted-by":"crossref","unstructured":"Wat, D. (2015). Therapy for Cystic Fibrosis Caused by Nonsense Mutations. Cystic Fibrosis in the Light of New Research, Intech Open. Chapter 13.","DOI":"10.5772\/59523"},{"key":"ref_84","doi-asserted-by":"crossref","unstructured":"Keeling, K.M. (2016). Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases. Diseases, 4.","DOI":"10.3390\/diseases4040032"},{"key":"ref_85","doi-asserted-by":"crossref","first-page":"552","DOI":"10.1016\/j.tig.2008.08.010","article-title":"Introducing sense into nonsense in treatments of human genetic diseases","volume":"24","author":"Linde","year":"2008","journal-title":"Trends Genet."},{"key":"ref_86","doi-asserted-by":"crossref","first-page":"e01963","DOI":"10.1016\/j.heliyon.2019.e01963","article-title":"Caffeine boosts Ataluren\u2019s readthrough activity","volume":"5","author":"Lentini","year":"2019","journal-title":"Heliyon"}],"container-title":["International Journal of Molecular Sciences"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1422-0067\/20\/13\/3329\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,11]],"date-time":"2025-10-11T13:03:11Z","timestamp":1760187791000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1422-0067\/20\/13\/3329"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,7,6]]},"references-count":86,"journal-issue":{"issue":"13","published-online":{"date-parts":[[2019,7]]}},"alternative-id":["ijms20133329"],"URL":"https:\/\/doi.org\/10.3390\/ijms20133329","relation":{},"ISSN":["1422-0067"],"issn-type":[{"value":"1422-0067","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,7,6]]}}}