Selective tissue targeting of synthetic nucleic acid drugs.
Journal
The Journal of clinical investigation
ISSN: 1558-8238
Titre abrégé: J Clin Invest
Pays: United States
ID NLM: 7802877
Informations de publication
Date de publication:
01 03 2019
01 03 2019
Historique:
pubmed:
29
1
2019
medline:
25
2
2020
entrez:
29
1
2019
Statut:
ppublish
Résumé
Antisense oligonucleotides (ASOs) are chemically synthesized nucleic acid analogs designed to bind to RNA by Watson-Crick base pairing. Following binding to the targeted RNA, the ASO perturbs RNA function by promoting selective degradation of the targeted RNA, altering RNA intermediary metabolism, or disrupting function of the RNA. Most antisense drugs are chemically modified to enhance their pharmacological properties and for passive targeting of the tissues of therapeutic interest. Recent advances in selective tissue targeting have resulted in a newer generation of ASO drugs that are more potent and better tolerated than previous generations, spawning renewed interest in identifying selective ligands that enhance targeted delivery of ASOs to tissues.
Identifiants
pubmed: 30688661
pii: 125228
doi: 10.1172/JCI125228
pmc: PMC6391085
doi:
pii:
Substances chimiques
Oligonucleotides, Antisense
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
915-925Références
Drug Dev Res. 2017 Sep;78(6):292-299
pubmed: 28786125
Nucleic Acid Ther. 2018 Apr;28(2):86-96
pubmed: 29437530
J Pharmacol Exp Ther. 2000 Feb;292(2):489-96
pubmed: 10640284
J Med Chem. 2016 Nov 10;59(21):9645-9667
pubmed: 27434100
Cell. 1983 Jan;32(1):277-87
pubmed: 6130851
Cell. 1984 Jul;37(3):789-800
pubmed: 6204769
Mol Ther. 2010 Jul;18(7):1357-64
pubmed: 20461061
Small. 2017 Apr;13(16):
pubmed: 28196309
ACS Nano. 2018 Mar 27;12(3):2138-2150
pubmed: 29320626
J Mol Biol. 2000 Jul 21;300(4):857-65
pubmed: 10891274
Nat Biotechnol. 2008 Apr;26(4):431-42
pubmed: 18376398
Bioorg Med Chem Lett. 2016 Aug 1;26(15):3690-3
pubmed: 27268307
Adv Drug Deliv Rev. 2015 Jun 29;87:46-51
pubmed: 25666165
J Biol Chem. 1994 Aug 26;269(34):21568-75
pubmed: 8063795
J Clin Invest. 1995 Apr;95(4):1814-23
pubmed: 7706488
Expert Opin Drug Metab Toxicol. 2009 Apr;5(4):381-91
pubmed: 19379126
Chembiochem. 2015 Apr 13;16(6):903-8
pubmed: 25786782
Int J Biochem Cell Biol. 2004 Nov;36(11):2137-43
pubmed: 15313461
Annu Rev Immunol. 2002;20:709-60
pubmed: 11861616
Nucleic Acids Res. 2014 Jul;42(13):8796-807
pubmed: 24992960
J Biol Chem. 2009 Feb 6;284(6):3777-83
pubmed: 19075021
J Biol Chem. 1993 Oct 5;268(28):20772-7
pubmed: 8407903
Glycoconj J. 1995 Jun;12(3):268-74
pubmed: 7496141
Chem Commun (Camb). 2012 Aug 25;48(66):8195-7
pubmed: 22614180
Mol Med. 2016 Oct;22:632-642
pubmed: 27455414
Kidney Int Suppl. 1998 Sep;67:S3-6
pubmed: 9736244
Pharmaceuticals (Basel). 2018 Apr 09;11(2):
pubmed: 29642542
Nucleic Acids Res. 2016 Apr 7;44(6):2782-94
pubmed: 26908652
Nature. 2004 Nov 11;432(7014):173-8
pubmed: 15538359
Angew Chem Int Ed Engl. 2012 Aug 20;51(34):8529-33
pubmed: 22782619
Nucleic Acids Res. 2015 Jan;43(2):1204-15
pubmed: 25550434
Atherosclerosis. 2005 Sep;182(1):1-15
pubmed: 15904923
J Biol Chem. 1971 Aug 10;246(15):4825-33
pubmed: 4327329
J Control Release. 2016 Sep 10;237:1-13
pubmed: 27369865
Nat Med. 2012 Jan 29;18(2):307-14
pubmed: 22286306
J Control Release. 2013 Dec 10;172(2):467-83
pubmed: 23742878
Proc Natl Acad Sci U S A. 2005 Nov 22;102(47):17125-9
pubmed: 16286643
Mol Pharm. 2014 Aug 4;11(8):2734-44
pubmed: 25020033
J Biol Chem. 1995 Feb 10;270(6):2620-7
pubmed: 7852327
Proc Natl Acad Sci U S A. 2012 Jul 24;109(30):11975-80
pubmed: 22773805
Med Princ Pract. 2016;25 Suppl 1:15-21
pubmed: 26111634
Sci Transl Med. 2017 Aug 2;9(401):
pubmed: 28768807
Pharmacol Rev. 2002 Dec;54(4):561-87
pubmed: 12429868
Biochim Biophys Acta. 1999 Dec 10;1489(1):117-30
pubmed: 10807002
Bioorg Med Chem. 2016 Jun 1;24(11):2397-409
pubmed: 27117693
Cell. 1980 Aug;21(1):79-93
pubmed: 7407914
Nucleic Acids Res. 2017 Nov 2;45(19):10969-10977
pubmed: 28981809
J Phys Chem C Nanomater Interfaces. 2012 Aug 30;116(34):18440-18450
pubmed: 22962627
Antisense Nucleic Acid Drug Dev. 1997 Jun;7(3):187-95
pubmed: 9212909
J Biol Chem. 1974 Sep 10;249(17):5536-43
pubmed: 4370480
Nucleic Acids Res. 2018 Feb 28;46(4):1601-1613
pubmed: 29361039
Mol Cell Ther. 2016 Feb 27;4:3
pubmed: 26925240
Nat Med. 1998 Apr;4(4):467-71
pubmed: 9546795
J Am Chem Soc. 2014 Dec 10;136(49):16958-61
pubmed: 25434769
Nucleic Acid Ther. 2014 Apr;24(2):101-13
pubmed: 24383421
Nucleic Acids Res. 2019 Feb 20;47(3):1070-1081
pubmed: 30535404
Nat Biotechnol. 2010 Feb;28(2):172-6
pubmed: 20081866
Bioconjug Chem. 2002 Nov-Dec;13(6):1220-5
pubmed: 12440856
Proc Natl Acad Sci U S A. 2013 Dec 10;110(50):20218-23
pubmed: 24284176
Nucleic Acid Ther. 2017 Aug;27(4):209-220
pubmed: 28448194
Adv Drug Deliv Rev. 2001 Jul 28;49(3):265-80
pubmed: 11551399
Physiol Rev. 1995 Jul;75(3):591-609
pubmed: 7624395
Cell. 2010 Apr 16;141(2):231-42
pubmed: 20403321
Physiology (Bethesda). 2012 Aug;27(4):237-47
pubmed: 22875454
Lancet. 2016 Nov 5;388(10057):2239-2253
pubmed: 27665230
Biochem Biophys Res Commun. 2015 Jan 2;456(1):257-61
pubmed: 25446080
Nucleic Acids Res. 2017 Dec 1;45(21):12388-12400
pubmed: 29069408
Nucleic Acids Res. 2013 Apr 1;41(6):3673-87
pubmed: 23396438
Mol Cell. 2008 Feb 29;29(4):428-40
pubmed: 18242112
Nucleic Acids Res. 2016 Aug 19;44(14):6518-48
pubmed: 27084936
Proc Natl Acad Sci U S A. 1983 Apr;80(8):2258-62
pubmed: 6300903
J Control Release. 2016 Oct 28;240:332-348
pubmed: 26774224
Biochemistry. 2018 Apr 10;57(14):2061-2064
pubmed: 29589907
Cell Tissue Res. 1998 May;292(2):395-410
pubmed: 9560481
Nat Biotechnol. 2007 Oct;25(10):1149-57
pubmed: 17873866
Expert Opin Pharmacother. 2006 Jun;7(8):1055-64
pubmed: 16722815
Nucleic Acids Res. 2017 Mar 17;45(5):2294-2306
pubmed: 28158620
Bioconjug Chem. 1997 Sep-Oct;8(5):762-5
pubmed: 9327143
J Biomater Sci Polym Ed. 2013;24(4):398-416
pubmed: 23565683
Am J Physiol Endocrinol Metab. 2000 Feb;278(2):E352-6
pubmed: 10662720
Mol Ther. 2017 Jan 4;25(1):71-78
pubmed: 28129130
Orphanet J Rare Dis. 2015 Sep 04;10:109
pubmed: 26338094
Mol Ther Nucleic Acids. 2016 Aug 02;5(8):e342
pubmed: 27483025
Annu Rev Pharmacol Toxicol. 2010;50:259-93
pubmed: 20055705
Nucleic Acid Ther. 2018 Jun;28(3):119-127
pubmed: 29425080
Annu Rev Biophys. 2013;42:217-39
pubmed: 23654304
J Org Chem. 2010 Mar 5;75(5):1569-81
pubmed: 20136157
Biochemistry. 2000 Jan 11;39(1):55-63
pubmed: 10625479
Nucleic Acids Res. 2016 Sep 6;44(15):7314-30
pubmed: 27378781
BioDrugs. 2018 Aug;32(4):297-309
pubmed: 29959665
Microsc Res Tech. 2002 Jun 1;57(5):269-88
pubmed: 12112439
Adv Drug Deliv Rev. 2016 Apr 1;99(Pt A):28-51
pubmed: 26456916
Nucleic Acid Ther. 2014 Dec;24(6):374-87
pubmed: 25353652
Nucleic Acids Res. 2018 Apr 20;46(7):3579-3594
pubmed: 29514240
Nucleic Acid Ther. 2016 Aug;26(4):223-35
pubmed: 27031383
Drug Discov Today. 2017 Oct;22(10):1547-1556
pubmed: 28627385
J Biol Chem. 1994 Oct 28;269(43):26801-5
pubmed: 7929417
Biochemistry. 1990 Oct 30;29(43):10009-18
pubmed: 2125488
Proc Natl Acad Sci U S A. 1996 Oct 15;93(21):11493-8
pubmed: 8876163
Angew Chem Int Ed Engl. 1998 Nov 2;37(20):2754-2794
pubmed: 29711117
ACS Chem Biol. 2015 May 15;10(5):1181-7
pubmed: 25730476
JAMA. 2017 Apr 11;317(14):1480
pubmed: 28399245
Cell Metab. 2018 Apr 3;27(4):714-739
pubmed: 29617640
Comp Hepatol. 2003 Jan 3;2(1):1
pubmed: 12537582
Nat Biotechnol. 2017 Mar;35(3):230-237
pubmed: 28244996
Nat Commun. 2015 Aug 10;6:7969
pubmed: 26258894
Sci Adv. 2018 Oct 17;4(10):eaat3386
pubmed: 30345352
Nucleic Acid Ther. 2013 Dec;23(6):369-78
pubmed: 24161045
J Biol Chem. 1980 Oct 10;255(19):9033-6
pubmed: 7410410
Endocrinology. 2014 Apr;155(4):1280-90
pubmed: 24467746
Nat Med. 2012 Dec;18(12):1847-56
pubmed: 23142820
J Pharmacol Exp Ther. 1996 May;277(2):923-37
pubmed: 8627575
Nucleic Acids Res. 2011 Jun;39(11):4795-807
pubmed: 21345934
Transl Neurosci. 2017 Jan 26;8:1-6
pubmed: 28400976
J Cell Biol. 1981 Sep;90(3):687-96
pubmed: 6270158