Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes-Reference-Cited by-同舟云学术

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Comprehensive transcriptome-wide analysis of spliceopathy correction of myotonic dystrophy using CRISPR-Cas9 in iPSCs-derived cardiomyocytes

Published:2022-01 Issue:1 Volume:30 Page:75-91
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Dastidar Sumitava,Majumdar Debanjana,Tipanee Jaitip,Singh Kshitiz,Klein Arnaud F.,Furling Denis,Chuah Marinee K.,VandenDriessche Thierry

Funder

Vrije Universiteit Brussel

Publisher

Elsevier BV

Subject

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

Reference86 articles.

1. Loss of muscleblind-like 1 results in cardiac pathology and persistence of embryonic splice isoforms;Dixon;Sci. Rep.,2015

2. Modelling inherited cardiac disease using human induced pluripotent stem cell-derived cardiomyocytes: Progress, pitfalls, and potential;van Mil;Cardiovasc. Res.,2018

3. Therapeutic approaches for dominant muscle diseases: Highlight on myotonic dystrophy;Klein;Curr. Gene Ther.,2015

4. Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy;Lin;Hum. Mol. Genet.,2006

5. Molecular mechanisms in DM1—A focus on foci;Pettersson;Nucleic Acids Res.,2015

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3. Biallelic variants in SNUPN cause a limb girdle muscular dystrophy with myofibrillar-like features;Brain;2024-02-15

4. Cardiac involvement in patient-specific induced pluripotent stem cells of myotonic dystrophy type 1: unveiling the impact of voltage-gated sodium channels;Frontiers in Physiology;2023-09-18

5. Pluripotent Stem Cells in Disease Modeling and Drug Discovery for Myotonic Dystrophy Type 1;Cells;2023-02-10

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