Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9-Reference-Cited by-同舟云学术

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Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9

Published:2018-11 Issue:11 Volume:26 Page:2617-2630
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Wang Yanlin,Hao Lei,Wang Hongcai,Santostefano Katherine,Thapa Arjun,Cleary John,Li Hui,Guo Xiuming,Terada Naohiro,Ashizawa Tetsuo,Xia Guangbin

Funder

NIH/NIAMS

Office of Research at the Health Science Center of University of New Mexico

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference74 articles.

1. RNA toxicity and foci formation in microsatellite expansion diseases;Zhang;Curr. Opin. Genet. Dev.,2017

2. Trinucleotide repeat disorders;Orr;Annu. Rev. Neurosci.,2007

3. Genome-editing Technologies for Gene and Cell Therapy;Maeder;Mol. Ther.,2016

4. Genome Editing Techniques and Their Therapeutic Applications;Calos;Clin. Pharmacol. Ther.,2017

5. Therapeutic gene editing: delivery and regulatory perspectives;Shim;Acta Pharmacol. Sin.,2017

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1. Harnessing the evolving CRISPR/Cas9 for precision oncology;Journal of Translational Medicine;2024-08-08

2. Immortalized human myotonic dystrophy type 1 muscle cell lines to address patient heterogeneity;iScience;2024-06

3. Precise editing of pathogenic nucleotide repeat expansions in iPSCs using paired prime editor;Nucleic Acids Research;2024-04-25

4. Cas9 nickase-mediated contraction of CAG/CTG repeats at multiple disease loci;2024-02-21

5. Therapeutic Targeting of the GSK3β-CUGBP1 Pathway in Myotonic Dystrophy;International Journal of Molecular Sciences;2023-06-26

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