Du Z, Qin Y. Dyslipidemia and cardiovascular disease: current knowledge, existing challenges, and new opportunities for management strategies. J Clin Med. 2023;12(1):363. https://doi.org/10.3390/jcm12010363.
Hedayatnia M, Asadi Z, Zare-Feyzabadi R, Yaghooti-Khorasani M, Ghazizadeh H, Ghaffarian-Zirak R, Nosrati-Tirkani A, Mohammadi-Bajgiran M, Rohban M, Sadabadi F, Rahimi HR, Ghalandari M, Ghaffari MS, Yousefi A, Pouresmaeili E, Besharatlou MR, Moohebati M, Ferns GA, Esmaily H, Ghayour-Mobarhan M. Dyslipidemia and cardiovascular disease risk among the MASHAD study population. Lipids Health Dis. 2020;19(1):42. https://doi.org/10.1186/s12944-020-01204-y.
Article CAS PubMed Google Scholar
Zodda D, Giammona R, Schifilliti S. Treatment strategy for dyslipidemia in cardiovascular disease prevention: focus on old and new drugs. Pharmacy (Basel). 2018;6(1):10. https://doi.org/10.3390/pharmacy6010010.
Sizar O, Khare S, Patel P, Talati R. Statin medications. Treasure Island: StatPearls Publishing; 2024.
Bardolia C, Amin NS, Turgeon J. Emerging non-statin treatment options for lowering low-density lipoprotein cholesterol. Front Cardiovasc Med. 2021;17(8): 789931. https://doi.org/10.3389/fcvm.2021.789931.
Tyagi S, Gupta P, Saini AS, Kaushal C, Sharma S. The peroxisome proliferator-activated receptor: a family of nuclear receptors role in various diseases. J Adv Pharm Technol Res. 2011;2(4):236–40. https://doi.org/10.4103/2231-4040.90879.
Article CAS PubMed Google Scholar
Gouni-Berthold I, Berthold H. Antisense oligonucleotides for the treatment of dyslipidemia. Curr Pharm Des. 2011;17(9):950–60. https://doi.org/10.2174/138161211795428830.
Article CAS PubMed Google Scholar
Crooke ST. Molecular mechanisms of antisense oligonucleotides. Nucleic Acid Ther. 2017;27:70–7. https://doi.org/10.1089/nat.2016.0656.
Article CAS PubMed 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. https://doi.org/10.1146/annurev.pharmtox.010909.105654.
Article CAS PubMed Google Scholar
Dias N, Stein CA. Antisense oligonucleotides: basic concepts and mechanisms. Mol Cancer Ther. 2002;1:347–55.
Agrawal S, Kandimalla ER. Antisense therapeutics: Is it as simple as complementary base recognition? Mol Med Today. 2000;6:72–81. https://doi.org/10.1016/s1357-4310(99)01638-x.
Article CAS PubMed Google Scholar
Chan JH, Lim S, Wong WF. Antisense oligonucleotides: from design to therapeutic application. Clin Exp Pharmacol Physiol. 2006;33:533–40. https://doi.org/10.1111/j.1440-1681.2006.04403.x.
Article CAS PubMed Google Scholar
Lauffer MC, van Roon-Mom W, Aartsma-Rus A, N = 1 Collaborative. Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders. Commun Med (Lond). 2024;4(1):6. https://doi.org/10.1038/s43856-023-00419-1.
Article CAS PubMed Google Scholar
Goyenvalle A, Babbs A, Wright J, Wilkins V, Powell D, Garcia L, et al. Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping. Hum Mol Genet. 2012;21:2559–71. https://doi.org/10.1093/hmg/dds082.
Article CAS PubMed Google Scholar
Prakash TP, Graham MJ, Yu J, Carty R, Low A, Chappell A, Schmidt K, Zhao C, Aghajan M, Murray HF. Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice. Nucleic Acids Res. 2014;42:8796–807. https://doi.org/10.1093/nar/gku531.
Article CAS PubMed Google Scholar
Dhuri K, Bechtold C, Quijano E, Pham H, Gupta A, Vikram A, Bahal R. Antisense oligonucleotides: an emerging area in drug discovery and development. J Clin Med. 2020;9:2004. https://doi.org/10.3390/jcm9062004.
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. 1978;75:280–4. https://doi.org/10.1073/pnas.75.1.280.
Article CAS PubMed Google Scholar
Eckstein F. Phosphorothioates, essential components of therapeutic oligonucleotides. Nucleic Acid Ther. 2014;24:374–87. https://doi.org/10.1089/nat.2014.0506.
Article CAS PubMed Google Scholar
Sasso JM, Ambrose BJB, Tenchov R, Datta RS, Basel MT, DeLong RK, et al. The progress and promise of RNA medicine—an arsenal of targeted treatments. J Med Chem. 2022;65:6975–7015. https://doi.org/10.1021/acs.jmedchem.2c00024.
Article CAS PubMed Google Scholar
Ersöz E, Demir-Dora D. Unveiling the potential of antisense oligonucleotides: mechanisms, therapies, and safety insights. Drug Dev Res. 2024. https://doi.org/10.1002/ddr.22187.
Nurmohamed NS, Navar AM, Kastelein JJP. New and emerging therapies for reduction of LDL-cholesterol and apolipoprotein B. J Am Coll Cardiol. 2021;77:1564–75. https://doi.org/10.1016/j.jacc.2020.11.079.
Article CAS PubMed Google Scholar
Finkel RS, Mercuri E, Darras BT, Connolly AM, Kuntz NL, Kirschner J. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377:1723–32.
Article CAS PubMed Google Scholar
Raal FJ, Santos RD, Blom DJ, Marais AD, Charng M-J, Cromwell WC, Lachmann RH, Gaudet D, Tan JL. Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet. 2010;375:998–1006. https://doi.org/10.1016/s0140-6736(10)60284-x.
Article CAS PubMed Google Scholar
Gaudet D, Gipe DA, Pordy R, Ahmad Z, Cuchel M, Shah PK, Sasiela WJ, Chan K, Brisson D. ANGPTL3 inhibition in homozygous familial hypercholesterolemia. N Engl J Med. 2017;377:296–7. https://doi.org/10.1056/NEJMc1705994.
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM. Eteplirsen for the treatment of duchenne muscular dystrophy. Ann Neurol. 2013;74:637–47. https://doi.org/10.1002/ana.23982.
Article CAS PubMed Google Scholar
Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH III, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, McEvoy BW, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T. Inotersen treatment for patients with hereditary transthyretin amyloidosis. N Engl J Med. 2018;379:22–31. https://doi.org/10.1056/nejmoa1716793.
Article CAS PubMed Google Scholar
Tsimikas S, Viney NJ, Hughes SG, Singleton W, Graham MJ, Baker BF, Burkey JL, Yang Q, Marcovina SM, Geary RS, Crooke RM, Witztum JL. Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebo-controlled phase 1 study. Lancet. 2015;386:1472–83. https://doi.org/10.1016/s0140-6736(15)61252-1.
Article CAS PubMed Google Scholar
Graham MJ, Lee RG, Bell TA III, Fu W, Mullick AE, Alexander VJ, Singleton W, Viney N, Geary R, Su J, Baker BF, Burkey J, Crooke ST, Crooke RM. Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans. Circ Res. 2013;112:1479–90. https://doi.org/10.1161/circresaha.111.300367.
Article CAS PubMed Google Scholar
Witztum JL, Gaudet D, Freedman SD, Alexander VJ, Digenio A, Williams KR, Yang Q, Hughes SG, Geary RS, Arca M, Stroes ESG, Bergeron J, Soran H, Civeira F, Hemphill L, Tsimikas S, Blom DJ, O’Dea L, Bruckert E. Volanesorsen and triglyceride levels
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