Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells

CRISPR/Cas9 is an attractive platform to potentially correct dominant genetic diseases by gene editing with unprecedented precision. In the current proof-of-principle study, we explored the use of CRISPR/Cas9 for gene-editing in myotonic dystrophy type-1 (DM1), an autosomal-dominant muscle disorder,...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Dastidar, Sumitava, Ardui, Simon, Singh, Kshitiz, Majumdar, Debanjana, Nair, Nisha, Fu, Yanfang, Reyon, Deepak, Samara, Ermira, Gerli, Mattia F M, Klein, Arnaud F, De Schrijver, Wito, Tipanee, Jaitip, Seneca, Sara, Tulalamba, Warut, Wang, Hui, Chai, Yoke Chin, In’t Veld, Peter, Furling, Denis, Tedesco, Francesco Saverio, Vermeesch, Joris R, Joung, J Keith, Chuah, Marinee K, VandenDriessche, Thierry
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2018
Assuntos:
Genome Integrity, Repair and Replication
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6144820/
https://ncbi.nlm.nih.gov/pubmed/29947794
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gky548
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