Study in Nature demonstrates method for repairing genes in human embryos that prevents inherited diseases

Oregon Health & Science University| Wed Aug 02 11:07:41 EDT 2017
Study in Nature demonstrates method for repairing genes in human embryos that prevents inherited diseases

Shoukhrat Mitalipov, Ph.D., reported the successful removal of a lethal genetic defect in human embryos.

The gene-editing technique described in this study could one day provide an avenue for people with known heritable disease-causing genetic mutations to eliminate the risk of passing the disease to their children. The study also demonstrated a way to overcome a crucial problem in genome editing. Known as mosaicism, the problem occurs when not all cells in a multicellular embryo are repaired and some cells still carry a mutation. These could ultimately find its way into an offspring’s DNA, rendering the repair moot.

The new study is the first to demonstrate that the technique can be used in human embryos to convert mutant genes back to normal.

You may have seen some speculative reporting in the media about this research. OHSU honors our agreements with peer-reviewed journals to hold news of research until it is published. Two events made possible the premature media coverage. An individual not affiliated with OHSU, but familiar with the research, spoke without permission to a reporter. That reporter then found photos and video associated with the research that were briefly published prematurely on an OHSU website. Those images were removed and protocols have been changed to prevent this happening in the future. We worked closely with Nature to hold communications until publication of the paper, helping make possible thoughtful scientific writing about the paper.

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