Footprint-free gene mutation correction in induced pluripotent stem cell (iPSC) derived from recessive dystrophic epidermolysis bullosa (RDEB) using the CRISPR/Cas9 and piggyBac transposon system.

JOURNAL OF INVESTIGATIVE DERMATOLOGY(2020)

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摘要
•We corrected a gene mutation in RDEB-specific iPSC without any footprint.•CRISPR/Cas9 is an easy-designed and efficient tool for gene editing.•piggyBac transposon can remove the residual gene during gene editing process.•iPSC-derived keratinocytes after gene editing can provide proper type VII collagen.•Inherited disease-specific iPSC is a good cell source for gene/cell therapy.
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关键词
RDEB,iPSC,CRISPR,HR,MC-iPSC,KC,FB,gRNA,PAM,IRS,HA,SB,EB,BMP4,EGF
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