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Genetic Modulation of RNA Splicing with a CRISPR-Guided Cytidine Deaminase

This protocol uses lipofectamine to deliver base editors (i.e., dCas9 and AIDx fusion protein) and sgRNA expression vectors into Duchenne Muscular Dystrophy (DMD) patient-derived human induced pluripotent stem cells (hiPSCs). This protocol details mutation of the 5′ splice site of DMD exon50 with TA...

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Detalles Bibliográficos
Autores principales: Ma, Yunqing, Yuan, Juanjuan, Chang, Xing
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7580208/
https://www.ncbi.nlm.nih.gov/pubmed/33111067
http://dx.doi.org/10.1016/j.xpro.2019.100005