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

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

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

Afiliação

Dastidar S; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, 1090 Brussels, Belgium.
Majumdar D; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, 1090 Brussels, Belgium.
Tipanee J; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, 1090 Brussels, Belgium.
Singh K; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, 1090 Brussels, Belgium.
Klein AF; Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, F-75013 Paris, France.
Furling D; Sorbonne Université, Inserm, Institut de Myologie, Centre de Recherche en Myologie, F-75013 Paris, France.
Chuah MK; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, 1090 Brussels, Belgium; Center for Molecular & Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, 3000 Leuven, Belgium. Electronic address: marinee.chuah@vub.be.
VandenDriessche T; Department of Gene Therapy & Regenerative Medicine, Vrije Universiteit Brussel, 1090 Brussels, Belgium; Center for Molecular & Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, 3000 Leuven, Belgium. Electronic address: thierry.vandendriessche@vub.be.

Mol Ther ; 30(1): 75-91, 2022 01 05.

Article em En | MEDLINE | ID: mdl-34371182

Assuntos

Células-Tronco Pluripotentes Induzidas; Distrofia Miotônica; Processamento Alternativo; Sistemas CRISPR-Cas; Proteínas de Ligação a Calmodulina/genética; Humanos; Células-Tronco Pluripotentes Induzidas/metabolismo; Miócitos Cardíacos/metabolismo; Distrofia Miotônica/genética; Distrofia Miotônica/terapia; Miotonina Proteína Quinase/genética; Proteínas de Ligação a RNA/genética; Proteínas de Ligação a RNA/metabolismo; Transcriptoma; Expansão das Repetições de Trinucleotídeos/genética

Palavras-chave

CRISPR ribonucleoprotein; alternate splicing; cardiomyogenic differentiation; gene editing; induced pluripotent stem cells; myotonic dystrophy; ribonuclear foci; spliceopathy; transcriptome

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Células-Tronco Pluripotentes Induzidas / Distrofia Miotônica Limite: Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Bélgica

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