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Dispos."},{"key":"10.1016\/j.ymthe.2023.01.005_bib99","doi-asserted-by":"crossref","first-page":"644","DOI":"10.1038\/s41419-022-05075-2","article-title":"RNA-based therapeutics: an overview and prospectus","volume":"13","author":"Zhu","year":"2022","journal-title":"Cell Death Dis."},{"key":"10.1016\/j.ymthe.2023.01.005_bib100","doi-asserted-by":"crossref","first-page":"2929","DOI":"10.1016\/S0960-894X(01)80842-8","article-title":"Study of antisense oligonucleotide phosphorothioates containing segments of oligodeoxynucleotides and 2\u2032-o- methyloligoribonucleotides","volume":"4","author":"Metelev","year":"1994","journal-title":"Bioorg. Med. Chem. 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Med."},{"key":"10.1016\/j.ymthe.2023.01.005_bib108","doi-asserted-by":"crossref","first-page":"16051","DOI":"10.1038\/nrdp.2016.51","article-title":"alpha1-Antitrypsin deficiency","volume":"2","author":"Greene","year":"2016","journal-title":"Nat. Rev. Dis. Primers"},{"key":"10.1016\/j.ymthe.2023.01.005_bib109","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1177\/0192623314551840","article-title":"Antisense oligonucleotide therapies: the promise and the challenges from a toxicologic pathologist's perspective","volume":"43","author":"Frazier","year":"2015","journal-title":"Toxicol. 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Sci."},{"key":"10.1016\/j.ymthe.2023.01.005_bib117","doi-asserted-by":"crossref","first-page":"1293","DOI":"10.1093\/nar\/29.6.1293","article-title":"Fully modified 2' MOE oligonucleotides redirect polyadenylation","volume":"29","author":"Vickers","year":"2001","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.ymthe.2023.01.005_bib118","doi-asserted-by":"crossref","first-page":"1468","DOI":"10.1093\/hmg\/ddw015","article-title":"Antisense targeting of 3' end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach","volume":"25","author":"Marsollier","year":"2016","journal-title":"Hum. Mol. 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Biol."},{"key":"10.1016\/j.ymthe.2023.01.005_bib122","doi-asserted-by":"crossref","first-page":"1062","DOI":"10.1016\/j.omtn.2020.07.037","article-title":"CRISPR start-loss: a novel and practical alternative for gene silencing through base-editing-induced start codon mutations","volume":"21","author":"Chen","year":"2020","journal-title":"Mol. Ther. 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