Precision gene targeting in stem cells corrects disease-causing mutations

Using two distinct methods, researchers have successfully and consistently manipulated targeted genes in both human embryonic stem cells and induced pluripotent stem cells (adult cells that have been reprogrammed to an embryonic stem cell-like state). In one case, scientists employed proteins known as zinc finger nucleases to change a single base pair in the genome, allowing them either to insert or remove mutations known to cause early-onset Parkinson's disease.

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