期刊
STEM CELL RESEARCH
卷 42, 期 -, 页码 -出版社
ELSEVIER
DOI: 10.1016/j.scr.2019.101677
关键词
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资金
- Fundacao para a Ciencia e a Tecnologia [PTDC/BIM-MED/3363/2014, iNOVA4Health -UID/Multi/04462/2013]
- Fundacao para a Ciencia e Tecnologia/Ministerio da Educacao e Ciencia
- FEDER under the PT2020 Partnership Agreement
- Fundação para a Ciência e a Tecnologia [PTDC/BIM-MED/3363/2014] Funding Source: FCT
Human induced pluripotent stem cells (hiPSCs) from individual patient basis are considered a powerful resource to model human diseases. However, to study complex multigenic diseases such as Congenital Heart Disease, it is crucial to generate perfect isogenic controls to understand gene singularity and contribution. Here, we report the engendering of an isogenic hiPSC line with homozygous correction of c.455G > A alteration in the DAND5 gene, using CRISPR/Cas9 technology. The characterization of a clone of this cell line demonstrates normal karyotype, pluripotent state, and potential to differentiate in vitro towards endoderm, mesoderm, and ectoderm.
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