Making Magic Happen: How Scientists are Editing DNA to Remove Potentially Fatal Genes

By Jane Farrell

Scientists have achieved a landmark breakthrough by editing DNA in human embryos to rid them of a potentially fatal gene

As reported in the journal Nature, the researchers from Oregon and Health Sciences University focused on hypertrophic cardiomyopathy, a common genetic heart disease. By editing the DNA of embryos carrying that gene, the scientists said that the embryo’s descendants would also be free of the illness. (The photo, above, released by OHSU, shows the editing process.)

In a release from the university, senior author Shoukhrat Miltalipov said, “Every generation on would carry this repair because we’ve removed the gene-carrying variant from that family’s lineage. By using this technique, it’s possible to reduce the burden of this inheritable disease.” Mitalipov directs the Center for Embryonic Cell and Gene Therapy at OHSU in Portland.

This is the first time scientists have successfully tested the method on donated clinical-quality human eggs.

In its news release, the university said that the technique the scientists used could be applied to “thousands of inherited genetic disorders affecting millions of people worldwide.” Additionally, it could increase the success of in vitro fertilization by increasing the number of healthy embryos. The gene-editing technique, described in the study, was done in concert with IVF.

The technique would be performed on people with known disease-causing genetic mutations, the release said.

While the success of the technique represents an astonishing breakthrough, it also raises the specter of “designer babies” whose DNA would be modified to include qualities like greater intelligence. That debate is likely to continue. DNA editing is very far from being used in a clinical setting.

The scientists acknowledged the controversy, with Daniel Dorsa, Ph.D., senior vice president for research at OHSU, saying,

“The ethical considerations of moving this technology to clinical trials are complex and deserve significant public engagement becfore we can answer the broader questions of whether it’s in humanity’s interest to alter human genes for future generations.”

In addition to OHSU, co-authors on the study represented the Center for Genome Engineering, within the Institute for Basic Science, and Seoul National University in South Korea; the Salk Institute for Biological Studies in La Jolla, California; and BGI-Qingdao and Shenzhen Engineering Laboratory for Innovative Molecular Diagnostics in China.