Landmark study removes deadly mutated genes from human embryos

U.S. and Korean scientists announced that they successfully edited defective genes that cause heart failure out of real-life human embryos Wednesday.

Paul Pate | Aug 04, 2017

U.S. and Korean scientists announced that they successfully edited defective genes that cause heart failure out of real-life human embryos Wednesday. The researchers, who carried out the procedure at Oregon Health and Science University and published a report on it in Nature, hope to develop it into a viable treatment that could prevent cystic fibrosis, breast cancer, and other illnesses and disorders that are passed on through DNA.

"This embryo gene correction method, if proven safe, can potentially be used to prevent transmission of genetic disease to future generations," said Paula Amato, a fertility specialist involved in the study.

Chinese researchers have edited human genes in a few small studies, but the Oregon team said that they are the first to conduct this procedure outside China. They used a gene-editing tool called Crispr-Cas9, which uses enzymes to locate a target gene on a chromosome, pull it out, and induce the cell to synthesize a new, healthy-functioning gene in its place.

In this procedure, they applied the Crispr-Cas9 to embryos made with the sperm of a man who had a mutant gene that causes hypertrophic cardiomyopathy, a disease that leads to cardiac arrest in young people.

The researchers made 58 embryos with the man's sperm and healthy eggs from female donors and found that half of the embryos carried the mutant gene. After the procedure, 72% of the embryos42 of the 58were free of it.

Gene editing is controversial, and the researchers note that legal obstacles may slow the research. Congress has barred the FDA from considering any clinical trials that involved edited embryos, and using gene-editing to create actual modified babies is illegal in many countries.

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