Expanded DNA and RNA Trinucleotide Repeats in Myotonic Dystrophy Type 1 Select Their Own Multitarget, Sequence-Selective Inhibitors.
Journal
Biochemistry
ISSN: 1520-4995
Titre abrégé: Biochemistry
Pays: United States
ID NLM: 0370623
Informations de publication
Date de publication:
22 09 2020
22 09 2020
Historique:
pubmed:
29
8
2020
medline:
17
3
2021
entrez:
29
8
2020
Statut:
ppublish
Résumé
There are few methods available for the rapid discovery of multitarget drugs. Herein, we describe the template-assisted, target-guided discovery of small molecules that recognize d(CTG) in the expanded d(CTG·CAG) sequence and its r(CUG) transcript that cause myotonic dystrophy type 1. A positive cross-selection was performed using a small library of 30 monomeric alkyne- and azide-containing ligands capable of producing >5000 possible di- and trimeric click products. The monomers were incubated with d(CTG)
Identifiants
pubmed: 32856901
doi: 10.1021/acs.biochem.0c00472
pmc: PMC7793557
mid: NIHMS1654201
doi:
Substances chimiques
Small Molecule Libraries
0
RNA
63231-63-0
DNA
9007-49-2
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
3463-3472Subventions
Organisme : NIGMS NIH HHS
ID : P41 GM103399
Pays : United States
Organisme : NIAMS NIH HHS
ID : R01 AR069645
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32 GM070421
Pays : United States
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