Editing Human Stem Cell Genes Could Let Patients Grow Their Own Cures

Thursday, October 13, 2011 - 12:00 in Biology & Nature

Human Genome Sequence Editing the arrangement of a sequence of these letters corrected a genetic mutation in stem cells, a breakthrough combination of stem cell therapy and genetic modification. Wikimedia Commons For the first time, scientists using a combination of gene-editing technologies have corrected mutations in a patient's own induced stem cells. The breakthrough could pave the way toward reprogramming a person's own cells to cure genetic diseases, rather than using transplanted organs and drug therapies. Researchers led by two institutions in the UK corrected a mutation in cells derived from a patient with a metabolic liver disease. Stem cells - embryonic ones and induced pluripotent ones - can turn into any type of cell, so they hold promise for treating a host of disorders. They can come with unwanted mutations, however. For one thing, induced pluripotent stem cells (iPS cells) would contain the same genetic defects as the rest...

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