From: Oregon Health & Science University
Published August 2, 2017 04:57 PM

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

Scientists have demonstrated an effective way of using a gene-editing tool to correct a disease-causing gene mutation in human embryos and stop it from passing to future generations.

The new technique uses the gene-editing tool CRISPR to target a mutation in nuclear DNA that causes hypertrophic cardiomyopathy, a common genetic heart disease that can cause sudden cardiac death and heart failure. The research, published Aug. 2 in the journal Nature, demonstrates a new method for repairing a disease-causing mutation and preventing it from being inherited by succeeding generations. This is the first time scientists have successfully tested the method on donated clinical-quality human eggs.

"Every generation on would carry this repair because we've removed the disease-causing gene variant from that family's lineage," said senior author Shoukhrat Mitalipov, Ph.D., who directs the Center for Embryonic Cell and Gene Therapy at OHSU. "By using this technique, it's possible to reduce the burden of this heritable disease on the family and eventually the human population."

Continue reading at Oregon Health & Science University (OHSU)

Image: This sequence of images shows the development of embryos after co-injection of a gene-correcting enzyme and sperm from a donor with a genetic mutation known to cause hypertrophic cardiomyopathy. A new study, published today in the journal Nature, demonstrates an effective method of repairing a disease-causing mutation from the moment of fertilization, preventing it from being passed to future generations. (Credit: OHSU)

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