Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy
Author:
Funder
Horizon 2020 Marie Skłodowska-Curie Actions
Prinses Beatrix Spierfonds
Spieren voor Spieren
Publisher
Elsevier BV
Subject
Drug Discovery,Molecular Medicine
Reference49 articles.
1. Facioscapulohumeral dystrophy: the path to consensus on pathophysiology;Tawil;Skelet. Muscle,2014
2. A unifying genetic model for facioscapulohumeral muscular dystrophy;Lemmers;Science,2010
3. DUX-family transcription factors regulate zygotic genome activation in placental mammals;De Iaco;Nat. Genet.,2017
4. Conserved roles of mouse DUX and human DUX4 in activating cleavage-stage genes and MERVL/HERVL retrotransposons;Hendrickson;Nat. Genet.,2017
5. DUX4 activates germline genes, retroelements, and immune mediators: implications for facioscapulohumeral dystrophy;Geng;Dev. Cell,2012
Cited by 11 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. Deciphering D4Z4 CpG methylation gradients in fascioscapulohumeral muscular dystrophy using nanopore sequencing;Genome Research;2023-09
2. A Systemically Administered Unconjugated Antisense Oligonucleotide Targeting DUX4 Improves Muscular Injury and Motor Function in FSHD Model Mice;Biomedicines;2023-08-22
3. The FSHD jigsaw: are we placing the tiles in the right position?;Current Opinion in Neurology;2023-06-14
4. Influence of DUX4 Expression in Facioscapulohumeral Muscular Dystrophy and Possible Treatments;International Journal of Molecular Sciences;2023-05-30
5. 268th ENMC workshop - Genetic diagnosis, clinical classification, outcome measures, and biomarkers in Facioscapulohumeral Muscular Dystrophy (FSHD): Relevance for clinical trials;Neuromuscular Disorders;2023-05
1.学者识别学者识别
2.学术分析学术分析
3.人才评估人才评估
"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370
www.globalauthorid.com
TOP
Copyright © 2019-2024 北京同舟云网络信息技术有限公司 京公网安备11010802033243号 京ICP备18003416号-3