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and siRNA-based therapeutics[J]. Drug Discov Today, 2017, erebroventricular administration of a 2'-O-methyl phosphoroth-
22(5): 823-833. ioate antisense oligonucleotide results in activation of the in-
[8] Alhamadani F, Zhang K, Parikh R, et al. Adverse drug reac- nate immune system in mouse brain[J]. Nucleic Acid Ther,
tions and toxicity of the food and drug administration-approved 2018, 28(2): 63-73.
antisense oligonucleotide drugs[J]. Drug Metab Dispos, 2022, [26] Shen LJ, Frazer-Abel A, Reynolds PR, et al. Mechanistic under-
50(6): 879-887. standing for the greater sensitivity of monkeys to antisense
[9] Frazier KS. Antisense oligonucleotide therapies: the promise oligonucleotide-mediated complement activation compared with
and the challenges from a toxicologic pathologist’s humans[J]. J Pharmacol Exp Ther, 2014, 351(3): 709-717.
perspective[J]. Toxicol Pathol, 2015, 43(1): 78-89. [27] Mansoor M, Melendez AJ. Advances in antisense oligonu-
[10] Zuany-Amorim C, Hastewell J, Walker C. Toll-like receptors as cleotide development for target identification, validation, and as
potential therapeutic targets for multiple diseases[J]. Nat Rev novel therapeutics[J]. Gene Regul Syst Bio, 2008, 2: 275-295.
Drug Discov, 2002, 1(10): 797-807. [28] Shadid M, Badawi M, Abulrob A. Antisense oligonucleotides:
[11] Partridge W, Burel SA, Ferng A, et al. Correlations between absorption, distribution, metabolism, and excretion[J]. Expert
preclinical BJAB assay ranking of antisense drugs and clinical Opin Drug Metab Toxicol, 2021, 17(11): 1281-1292.
trial adverse events[J]. Clin Transl Sci, 2023, 16(4): 575-580. [29] Shen W, De Hoyos CL, Migawa MT, et al. Chemical modifica-
[12] Agrawal S, Kandimalla ER. Antisense and siRNA as agonists of tion of PS-ASO therapeutics reduces cellular protein-binding
toll-like receptors[J]. Nat Biotechnol, 2004, 22(12): 1533-1537. and improves the therapeutic index[J]. Nat Biotechnol, 2019,
[13] Kim Y. Drug discovery perspectives of antisense oligonu- 37(6): 640-650.
cleotides[J]. Biomol Ther (Seoul), 2023, 31(3): 241-252. [30] Roberts TC, Langer R, Wood MJA. Advances in oligonu-
[14] Takakusa H, Iwazaki N, Nishikawa M, et al. Drug metabolism cleotide drug delivery[J]. Nat Rev Drug Discov, 2020, 19(10):
and pharmacokinetics of antisense oligonucleotide therapeutics: 673-694.
typical profiles, evaluation approaches, and points to consider [31] Nielsen PE, Egholm M, Berg RH, et al. Sequence-selective
compared with small molecule drugs[J]. Nucleic Acid Ther, recognition of DNA by strand displacement with a thymine-sub-
2023, 33(2): 83-94. stituted polyamide[J]. Science, 1991, 254(5037): 1497-1500.
[15] Vinjamuri BP, Pan JY, Peng P. A review on commercial [32] Inoue H, Hayase Y, Iwai S, et al. Sequence-dependent hydroly-
oligonucleotide drug products[J]. J Pharm Sci, 2024, 113(7): sis of RNA using modified oligonucleotide splints and RNase
1749-1768. H[J]. FEBS Lett, 1987, 215(2): 327-330.
[16] Quemener AM, Bachelot L, Forestier A, et al. The powerful [33] Aartsma-Rus A, Takeda S. A historical perspective on the de-
world of antisense oligonucleotides: From bench to bedside[J]. velopment of antisense oligonucleotide treatments for Duchenne
Wiley Interdiscip Rev RNA, 2020, 11(5): e1594. muscular dystrophy and spinal muscular atrophy[J]. J Neuro-
[17] Kurreck J. Antisense technologies[J]. Eur J Biochem, 2003, muscul Dis, 2025: 22143602251317422.
270(8): 1628-1644. [34] Goyenvalle A, Jimenez-Mallebrera C, van Roon W, et al. Con-
[18] Dhuri K, Bechtold C, Quijano E, et al. Antisense oligonu- siderations in the preclinical assessment of the safety of anti-
cleotides: an emerging area in drug discovery and sense oligonucleotides[J]. Nucleic Acid Ther, 2023, 33(1): 1-16.
development[J]. J Clin Med, 2020, 9(6): 2004. [35] Gapmers: Methods and Protocols | SpringerLink[EB/OL].
[19] Yanai H, Chiba S, Ban T, et al. Suppression of immune re- [2025-06-22]. https://link.springer.com/book/10.1007/978-1-
sponses by nonimmunogenic oligodeoxynucleotides with high 0716-0771-8.
affinity for high-mobility group box proteins (HMGBs)[J]. Proc [36] Huang S, Hao XY, Li YJ, et al. Nonviral delivery systems for
Natl Acad Sci U S A, 2011, 108(28): 11542-11547. antisense oligonucleotide therapeutics[J]. Biomater Res, 2022,
[20] Mansour HM, El-Khatib AS. Oligonucleotide-based therapeu- 26(1): 49.
tics for neurodegenerative disorders: focus on antisense oligonu- [37] Samaridou E, Heyes J, Lutwyche P. Lipid nanoparticles for nu-
cleotides[J]. Eur J Pharmacol, 2025, 998: 177529. cleic acid delivery: Current perspectives[J]. Adv Drug Deliv
[21] Graham MJ, Lee RG, Brandt TA, et al. Cardiovascular and Rev, 2020, 154/155: 37-63.
metabolic effects of ANGPTL3 antisense oligonucleotides[J]. N [38] Kong WW, Wei YN, Dong ZR, et al. Role of size, surface
Engl J Med, 2017, 377(3): 222-232. charge, and PEGylated lipids of lipid nanoparticles (LNPs) on
[22] Anderson BA, Freestone GC, Low A, et al. Towards next gen- intramuscular delivery of mRNA[J]. J Nanobiotechnol, 2024,
eration antisense oligonucleotides: mesylphosphoramidate mod- 22(1): 553.
ification improves therapeutic index and duration of effect of [39] Nair JK, Attarwala H, Sehgal A, et al. Impact of enhanced
gapmer antisense oligonucleotides[J]. Nucleic Acids Res, 2021, metabolic stability on pharmacokinetics and pharmacodynam-
49(16): 9026-9041. ics of GalNAc-siRNA conjugates[J]. Nucleic Acids Res, 2017,
[23] Çakan E, Lara OD, Szymanowska A, et al. Therapeutic anti- 45(19): 10969-10977.
sense oligonucleotides in oncology: from bench to bedside[J]. [40] Adams D, Gonzalez-Duarte A, O’Riordan WD, et al. Patisiran,
Cancers (Basel), 2024, 16(17): 2940. an RNAi therapeutic, for hereditary transthyretin
[24] Ramasamy T, Ruttala HB, Munusamy S, et al. Nano drug deliv- amyloidosis[J]. N Engl J Med, 2018, 379(1): 11-21.
ery systems for antisense oligonucleotides (ASO) [41] Garrelfs SF, Frishberg Y, Hulton SA, et al. Lumasiran, an
therapeutics[J]. J Control Release, 2022, 352: 861-878. RNAi therapeutic for primary hyperoxaluria type 1[J]. N Engl J
[25] Toonen LJA, Casaca-Carreira J, Pellisé-Tintoré M, et al. Intrac- Med, 2021, 384(13): 1216-1226.