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Rescue of premature aging defects in Cockayne syndrome stem cells by CRISPR/Cas9-mediated gene correction

Protein Cell. 2020; 
Wang S, , Min Z, Ji Q, Geng L, Su Y, Liu Z, Hu H, Wang L, Zhang W, , , Suzuiki K, Huang Y, Zhang P, Tang TS, , Qu J, , Yu Y, Liu GH, , , Qiao J, .
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Gene Synthesis … The primer sequences are listed in Table S1 b Endotoxin detection Endotoxin in the supernatant of the conditioned medium was detected with the ToxinSensor Gel Clot Endotoxin Assay Kit (GenScript, Cat No L00351) according to the manufacturer's protocol … Get A Quote

摘要

Cockayne syndrome (CS) is a rare autosomal recessive inherited disorder characterized by a variety of clinical features, including increased sensitivity to sunlight, progressive neurological abnormalities, and the appearance of premature aging. However, the pathogenesis of CS remains unclear due to the limitations of current disease models. Here, we generate integration-free induced pluripotent stem cells (iPSCs) from fibroblasts from a CS patient bearing mutations in CSB/ERCC6 gene and further derive isogenic gene-corrected CS-iPSCs (GC-iPSCs) using the CRISPR/Cas9 system. CS-associated phenotypic defects are recapitulated in CS-iPSC-derived mesenchymal stem cells (MSCs) and neural stem cells (NSCs)... More

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