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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...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2020
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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 |