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News-in-brief / 05.6.15

New paper addresses “on-target” effects of engineering human pluripotent stem cells

By Broad Communications

Human pluripotent stem cells (hPSCs) are useful tools for studying disease, performing chemical screens, and looking for cellular therapies. But in these scenarios, studies with hPSCs are only as good as the genome engineering processes used to investigate them. The leading method for doing so is the CRISPR-Cas9 system, which can target one allele or another as it cuts DNA at specific points along the genome. The approach is extremely powerful for studying biology, but it can also introduce unintended mutations. So-called "off-target effects," or those mutations that happen in places other than the gene of interest, are well studied. But "on-target effects," which happen when the non-targeted allele is compromised, remain more of a mystery. In the journal Cell Reports, a new study led by Broad Institute Member Kevin Eggan, also of the Harvard Stem Cell Institute, presents a method for engineering hPSCs using CRISPR, but without these “on-target” effects.