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CRISPR Editing of GLI1 in Human ESCs

Using a CRISPR/Cas9-based strategy, researchers led by Vasiliy Galat and Philip M Iannaccone (Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA) edited out a regulatory region of the first intron of the GLI1 gene in human embryonic stem cells (ESCs). As this region contains highly conserved GLI1 binding sites, editing significantly lowered GLI1 transcription in the heterozygous state and reduced GLI1 expression to barely detectable levels in the homozygous state. Galat et al. note that alterations to GLI1 expression significantly decreased ectodermal, mesodermal, and endodermal marker expression and resulted in significant differentiation defects. For all the details, see STEM CELLS now!