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

CTG repeat expansion (CTG(exp)) is associated with aberrant alternate splicing that contributes to cardiac dysfunction in myotonic dystrophy type 1 (DM1). Excision of this CTG(exp) repeat using CRISPR-Cas resulted in the disappearance of punctate ribonuclear foci in cardiomyocyte-like cells derived...

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Detalles Bibliográficos
Autores principales: Dastidar, Sumitava, Majumdar, Debanjana, Tipanee, Jaitip, Singh, Kshitiz, Klein, Arnaud F., Furling, Denis, Chuah, Marinee K., VandenDriessche, Thierry
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8753376/
https://www.ncbi.nlm.nih.gov/pubmed/34371182
http://dx.doi.org/10.1016/j.ymthe.2021.08.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8753376/
https://www.ncbi.nlm.nih.gov/pubmed/34371182
http://dx.doi.org/10.1016/j.ymthe.2021.08.004

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