Chan JHP, Lim S, Wong WSF. Antisense oligonucleotides: from design to therapeutic application. Clin Exp Pharmacol Physiol. 2006;33:533–40.

Article  CAS  PubMed  Google Scholar 

Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus. Nat Rev Drug Discov. 2021;20:427–53.

Article  CAS  PubMed  Google Scholar 

Alama A, Barbieri F, Cagnoli M, Schettini G. Antisense oligonucleotides as therapeutic agents. Pharmacol Res. 1997;36:171–8.

Article  CAS  PubMed  Google Scholar 

Dias N, Stein CA. Antisense oligonucleotides: basic concepts and mechanisms. Mol Cancer Ther. 2002;1:347–55.

CAS  PubMed  Google Scholar 

Smith CIE, Zain R. Therapeutic oligonucleotides: State of the art. Annu Rev Pharmacol Toxicol. 2019;59:605–30.

Article  CAS  PubMed  Google Scholar 

Zamecnik PC, Stephenson ML. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc Natl Acad Sci USA. 1978;75:280–4.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Stephenson ML, Zamecnik PC. Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide. Proc Natl Acad Sci USA. 1978;75:285–8.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Donis-Keller H. Site specific enzymatic cleavage of RNA. Nucleic Acids Res. 1979;7:179–92.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Dominski Z, Kole R. Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. Proc Natl Acad Sci USA. 1993;90:8673–7.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Perry CM, Balfour JA. Fomivirsen. Drugs. 1999;57:375–80.

Article  CAS  PubMed  Google Scholar 

de Smet MD, Meenken CJ, van den Horn GJ. Fomivirsen—a phosphorothioate oligonucleotide for the treatment of CMV retinitis. Ocul Immunol Inflamm. 1999;7:189–98.

Article  PubMed  Google Scholar 

Roehr B. Fomivirsen approved for CMV retinitis. J Int Assoc Physicians AIDS Care. 1998;4:14–6.

CAS  PubMed  Google Scholar 

Wong E, Goldberg T. Mipomersen (kynamro): a novel antisense oligonucleotide inhibitor for the management of homozygous familial hypercholesterolemia. P T. 2014;39:119–22.

PubMed  PubMed Central  Google Scholar 

Chambergo-Michilot D, Alur A, Kulkarni S, Agarwala A. Mipomersen in familial hypercholesterolemia: an update on health-related quality of life and patient-reported outcomes. Vasc Health Risk Manag. 2022;18:73–80.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Baker DE. Eteplirsen. Hosp Pharm. 2017;52:302–5.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Lim KR, Maruyama R, Yokota T. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Dev Ther. 2017;11:533–45.

Article  CAS  Google Scholar 

Stein CA. Eteplirsen approved for Duchenne muscular dystrophy: the FDA faces a difficult choice. Mol Ther. 2016;24:1884–5.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, et al. Eteplirsen treatment for Duchenne muscular dystrophy: exon skipping and dystrophin production. Neurology. 2018;90:e2146–54.

Article  CAS  PubMed  Google Scholar 

Heo YA. Golodirsen: first approval. Drugs. 2020;80:329–33.

Article  PubMed  Google Scholar 

Aartsma-Rus A, Corey DR. The 10th oligonucleotide therapy approved: Golodirsen for Duchenne muscular dystrophy. Nucleic Acid Ther. 2020;30:67–70.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Anwar S, Yokota T. Golodirsen for Duchenne muscular dystrophy. Drugs Today (Barc). 2020;56:491–504.

Article  CAS  PubMed  Google Scholar 

Viltolarsen DS. First approval. Drugs. 2020;80:1027–31.

Article  Google Scholar 

Roshmi RR, Yokota T. Viltolarsen for the treatment of Duchenne muscular dystrophy. Drugs Today (Barc). 2019;55:627–39.

Article  CAS  PubMed  Google Scholar 

Roshmi RR, Yokota T. Viltolarsen: from preclinical studies to FDA approval. Methods Mol Biol. 2023;2587:31–41.

Article  CAS  PubMed  Google Scholar 

Casimersen SM. First approval. Drugs. 2021;81:875–9.

Article  Google Scholar 

Wilton-Clark H, Yokota T. Casimersen for Duchenne muscular dystrophy. Drugs Today (Barc). 2021;57:707–17.

Article  CAS  PubMed  Google Scholar 

Zakeri SE, Pradeep SP, Kasina V, Laddha AP, Manautou JE, Bahal R. Casimersen for the treatment of Duchenne muscular dystrophy. Trends Pharmacol Sci. 2022;43:607–8.

Article  CAS  PubMed  Google Scholar 

Neil EE, Bisaccia EK. Nusinersen: a novel antisense oligonucleotide for the treatment of spinal muscular atrophy. J Pediatr Pharmacol Ther. 2019;24:194–203.

PubMed  PubMed Central  Google Scholar 

Li Q. Nusinersen as a therapeutic agent for spinal muscular atrophy. Yonsei Med J. 2020;61:273–83.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Wurster CD, Ludolph AC. Nusinersen for spinal muscular atrophy. Ther Adv Neurol Disord. 2018;11:1756285618754459.

Article  PubMed  PubMed Central  Google Scholar 

Gales L. Tegsedi (inotersen): an antisense oligonucleotide approved for the treatment of adult patients with hereditary transthyretin amyloidosis. Pharmaceuticals (Basel). 2019;12:78.

Article  CAS  PubMed  Google Scholar 

Keam SJ. Inotersen: first global approval. Drugs. 2018;78:1371–6.

Article  CAS  PubMed  Google Scholar 

Blair HA. Tofersen: first approval. Drugs Today (Barc). 2023;83:1039–43.

CAS  Google Scholar 

Akcea's antisense drug rejection worries analysts. Nat Biotechnol. 2018; 36:911

Paik J, Duggan S. Volanesorsen: first global approval. Drugs. 2019;79:1349–54.

Article  CAS  PubMed  Google Scholar 

Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, et al. Patient-customized oligonucleotide therapy for a rare genetic disease. N Engl J Med. 2019;381:1644–52.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cappella M, Pradat PF, Querin G, Biferi MG. Beyond the traditional clinical trials for amyotrophic lateral sclerosis and the future impact of gene therapy. J Neuromuscul Dis. 2021;8:25–38.

Article  PubMed  PubMed Central  Google Scholar 

Synofzik M, van Roon-Mom WMC, Marckmann G, van Duyvenvoorde HA, Graessner H, Schüle R, et al. Preparing n-of-1 antisense oligonucleotide treatments for rare neurological diseases in Europe: genetic, regulatory, and ethical perspectives. Nucleic Acid Ther. 2022;32:83–94.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bennett CF, Swayze EE. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol. 2010;50:259–93.

Article  CAS  PubMed  Google Scholar 

Crooke ST, Liang XH, Baker BF, Crooke RM. Antisense technology: a review. J Biol Chem. 2021;296: 100416.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Crooke ST, Witztum JL, Bennett CF, Baker BF