Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1-Reference-Cited by-同舟云学术

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Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1

Published:2023-06 Issue: Volume:32 Page:622-636
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

El Boujnouni Najoua,van der Bent M. Leontien,Willemse Marieke,’t Hoen Peter A.C.,Brock Roland,Wansink Derick G.^ORCID

Funder

Prinses Beatrix Spierfonds

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference73 articles.

1. Antisense oligonucleotides: a new therapeutic principle;Uhlmann;Chem. Rev.,1990

2. Pharmacology of antisense drugs;Bennett;Annu. Rev. Pharmacol. Toxicol.,2017

3. FDA approval of nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides;Aartsma-Rus;Nucleic Acid Therapeut.,2017

4. Evaluation of 2’-modified oligonucleotides containing 2’-deoxy gaps as antisense inhibitors of gene expression;Monia;J. Biol. Chem.,1993

5. Kinetic characteristics of Escherichia coli RNase H1: cleavage of various antisense oligonucleotide-RNA duplexes;Crooke;Biochem. J.,1995

Cited by 3 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Therapeutic Antisense Oligonucleotides in Oncology: From Bench to Bedside;Cancers;2024-08-23

2. Antisense oligonucleotide therapeutic approach for Timothy syndrome;Nature;2024-04-24

3. All roads lead to cure: Diversity of oligonucleotides in DM1 therapy;Molecular Therapy - Nucleic Acids;2023-06

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