Design and In Vitro Evaluation of Splice-Switching Oligonucleotides Bearing Locked Nucleic Acids, Amido-Bridged Nucleic Acids, and Guanidine-Bridged Nucleic Acids.
amido-bridged nucleic acids (AmNAs)
bridged nucleic acids (BNAs)
chemical modification
dystrophin
exon skipping
guanidine-bridged nucleic acids (GuNAs)
locked nucleic acids (LNAs)
splice-switching oligonucleotide
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
29 Mar 2021
29 Mar 2021
Historique:
received:
15
02
2021
revised:
20
03
2021
accepted:
26
03
2021
entrez:
3
4
2021
pubmed:
4
4
2021
medline:
2
6
2021
Statut:
epublish
Résumé
Our group previously developed a series of bridged nucleic acids (BNAs), including locked nucleic acids (LNAs), amido-bridged nucleic acids (AmNAs), and guanidine-bridged nucleic acids (GuNAs), to impart specific characteristics to oligonucleotides such as high-affinity binding and enhanced enzymatic resistance. In this study, we designed a series of LNA-, AmNA-, and GuNA-modified splice-switching oligonucleotides (SSOs) with different lengths and content modifications. We measured the melting temperature (
Identifiants
pubmed: 33805378
pii: ijms22073526
doi: 10.3390/ijms22073526
pmc: PMC8037388
pii:
doi:
Substances chimiques
DMD protein, human
0
Dystrophin
0
Nucleic Acids
0
Oligonucleotides
0
locked nucleic acid
0
Guanidine
JU58VJ6Y3B
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Japan Society for the Promotion of Science
ID : 15J05689
Organisme : Japan Agency for Medical Research and Development
ID : JP20am0101084
Organisme : Japan Agency for Medical Research and Development
ID : JP18am0301004
Organisme : Japan Agency for Medical Research and Development
ID : JP20am0401003
Références
Chem Commun (Camb). 2014 Jan 18;50(5):575-7
pubmed: 24270219
PLoS One. 2017 Jul 24;12(7):e0181065
pubmed: 28742140
Org Biomol Chem. 2015 Mar 28;13(12):3757-65
pubmed: 25690587
Nucleic Acids Res. 2020 Jan 24;48(2):802-816
pubmed: 31802121
Hum Gene Ther. 2014 Jul;25(7):587-98
pubmed: 24826963
Sci Rep. 2019 Apr 15;9(1):6078
pubmed: 30988454
J Med Chem. 1993 Apr 2;36(7):831-41
pubmed: 8464037
Nat Rev Drug Discov. 2020 Oct;19(10):673-694
pubmed: 32782413
Hum Gene Ther. 2004 Aug;15(8):749-57
pubmed: 15319032
J Hum Genet. 2018 Mar;63(3):365-375
pubmed: 29339778
Nucleic Acid Ther. 2014 Feb;24(1):37-47
pubmed: 24171481
Nucleic Acids Res. 2014 Jul;42(12):8174-87
pubmed: 24935206
Nucleic Acids Res. 2016 Aug 19;44(14):6549-63
pubmed: 27288447
J Clin Invest. 2014 Sep;124(9):4067-81
pubmed: 25105368
Mol Ther. 2006 Oct;14(4):471-5
pubmed: 16854630
Chem Commun (Camb). 2016 Nov 10;52(92):13467-13470
pubmed: 27790668
Chembiochem. 2012 Nov 26;13(17):2513-6
pubmed: 23081931
Methods Mol Biol. 2018;1828:395-411
pubmed: 30171556
BMC Bioinformatics. 2012 Jun 18;13:134
pubmed: 22708584
Mol Ther Nucleic Acids. 2017 Dec 15;9:155-161
pubmed: 29246294
Oligonucleotides. 2005 Dec;15(4):284-97
pubmed: 16396622
Expert Rev Mol Med. 2019 Oct 02;21:e5
pubmed: 31576784
Am J Pathol. 2012 Aug;181(2):392-400
pubmed: 22683468
Mol Ther. 2008 Jul;16(7):1316-22
pubmed: 18461057
Nucleic Acids Res. 2020 Apr 6;48(6):2853-2865
pubmed: 32103257
Mol Ther. 2009 Mar;17(3):554-61
pubmed: 19142179
Chem Commun (Camb). 2019 Jun 11;55(48):6850-6853
pubmed: 31123731
Org Biomol Chem. 2020 Dec 7;18(46):9461-9472
pubmed: 33179694
PLoS One. 2018 May 16;13(5):e0197373
pubmed: 29768479
Methods Mol Biol. 2012;867:143-67
pubmed: 22454060
Nat Chem Biol. 2012 Apr 15;8(6):555-61
pubmed: 22504300