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Fig. 2 | Stem Cell Research & Therapy

Fig. 2

From: Low-density lipoprotein receptor-deficient hepatocytes differentiated from induced pluripotent stem cells allow familial hypercholesterolemia modeling, CRISPR/Cas-mediated genetic correction, and productive hepatitis C virus infection

Fig. 2

CRISPR/Cas9-mediated insertion of LDLR expression cassette at the AAVS1 genomic site. a Design of the LDLR expression cassette in the AAVS1 SA-2A-puro-pA donor plasmid that contains sequences homologous to the sequences flanking the AAVS1 double strand break (DSB) induced by CRISPR/Cas9 (5′ and 3′ Arms). Abbreviations: AAVS1, adeno-associated virus integration site 1, APOA2, apolipoprotein A2; pA, polyadenylation tail; Puro(R), puromycin resistance gene; SA, splice acceptor site; WPRE, woodchuck posttranslational regulatory element. b Junction PCR analyses showing correct integration of the cassette in 15/16 FH-iPSC clones. c PCR analysis corresponding to AAVS1 targeted locus, showing absence of integration of the correcting cassette in one of the alleles

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Stem Cell Research & Therapy

ISSN: 1757-6512