A Deoxyribonucleic Acid Decoy Trapping DUX4 for the Treatment of Facioscapulohumeral Muscular Dystrophy-Reference-Cited by-同舟云学术

A Deoxyribonucleic Acid Decoy Trapping DUX4 for the Treatment of Facioscapulohumeral Muscular Dystrophy

Published:2020-12 Issue: Volume:22 Page:1191-1199
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Mariot Virginie,Joubert Romain,Marsollier Anne-Charlotte,Hourdé Christophe,Voit Thomas,Dumonceaux Julie

Funder

National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London

Wellcome Trust Institutional Strategic Support Fund

NIHR GOSH BRC

FHSD Society

Association Française contre les Myopathies

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference44 articles.

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2. Facioscapulohumeral muscular dystrophies;Wagner;Continuum (Minneap. Minn.),2019

3. Facioscapulohumeral dystrophy: incomplete suppression of a retrotransposed gene;Snider;PLoS Genet.,2010

4. Facioscapulohumeral muscular dystrophy: consequences of chromatin relaxation;van der Maarel;Curr. Opin. Neurol.,2012

5. A unifying genetic model for facioscapulohumeral muscular dystrophy;Lemmers;Science,2010

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1. Oligonucleotide Therapies for Facioscapulohumeral Muscular Dystrophy: Current Preclinical Landscape;International Journal of Molecular Sciences;2024-08-21

2. Facioscapulohumeral muscular dystrophy: the road to targeted therapies;Nature Reviews Neurology;2023-01-10

3. Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy;Human Gene Therapy;2022-09-01

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5. Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit DUX4 Expression;Biomedicines;2022-07-07