CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.nature.com/articles/s41434-022-00336-3.pdf
Reference24 articles.
1. Falzarano MS, Scotton C, Passarelli C, Ferlini A. Duchenne muscular dystrophy: from diagnosis to therapy. Molecules. 2015;20:18168–84.
2. Bladen CL, Salgado D, Monges S, Foncuberta ME, Kekou K, Kosma K, et al. The treat-nmd dmd global database: analysis of more than 7,000 duchenne muscular dystrophy mutations. Hum Mutat. 2015;36:395–402.
3. Matthews E, Brassington R, Kuntzer T, Jichi F, Manzur AY. Corticosteroids for the treatment of duchenne muscular dystrophy. Cochrane Database Syst Rev. 2016:CD003725.
4. Hsu PD, Lander ES, Zhang F. Development and applications of crispr-cas9 for genome engineering. Cell. 2014;157:1262–78.
5. Goell JH, Hilton IB. Crispr/cas-based epigenome editing: Advances, applications, and clinical utility. Trends Biotechnol. 2021;39:678–91.
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1. Correction of exon 2, exon 2–9 and exons 8–9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 system;Scientific Reports;2024-09-11
2. A bioinformatic analysis of gene editing off-target loci altered by common polymorphisms, using ‘PopOff’;Journal of the Royal Society of New Zealand;2024-05-09
3. Muscular Dystrophy: Underlying Cellular and Molecular Mechanisms and Various Nanotherapeutic Approaches for Muscular Dystrophy;Mechanism and Genetic Susceptibility of Neurological Disorders;2024
4. Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies;Human Gene Therapy;2023-05-01
5. CRISPR-Editing Therapy for Duchenne Muscular Dystrophy;Human Gene Therapy;2023-05-01
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