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Clustered, regularly interspaced short palindromic repeats (CRISPR) editing to rapidly generate stem cell models for disease

A comparison of genome editing technologies suggests CRISPR-based systems could be more efficient for generating stem cell models for disease than systems that use transcription activator-like effector nucleases (TALENs). In a human pluripotent stem cell line, CRISPR systems introduced mutant alleles at 7 genomic loci with an efficiency of 51%-79%, whereas TALENs had an efficiency of 0%-34%. Next steps include determining the off-target effects of CRISPR-based genome editing.

SciBX 6(16); doi:10.1038/scibx.2013.399
Published online April 25, 2013

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Ding, Q. et al. Cell Stem Cell; published online April 4, 2013;
Contact: Kiran Musunuru, Harvard University, Cambridge, Mass.