For the first time in American history, scientists have successfully used a gene-editing tool to correct a disease-causing mutation in human DNA with the goal of ending its heredity for future generations.
Until now, the CRISPR gene-editing tool had never been used for human testing in the United States. But, according to a new report published in Nature, researchers at Oregon Health and Science University were able to edit the DNA in donated embryos to remove the gene for a heart condition that is the most common cause of sudden death in young athletes.
CRISPR, which stands for clustered regularly interspaced short palindromic repeats, uses an enzyme to track down specific sets of gene mutations that cause certain diseases. When CRISPR removes the mutation from the embryo’s DNA, it effectively brings an end to the disease being passed down that family line.
“Every generation on would carry this repair because we’ve removed the disease-causing gene variant from that family’s lineage,” said the study’s senior author and scientist Shoukhrat Mitalipov. “By using this technique, it’s possible to reduce the burden of this heritable disease on the family and eventually the human population.”
The targeted disease, hypertrophic cardiomyopathy, affects 1 in every 500 people worldwide. It is entirely undetectable and devoid of symptoms until the host basically goes into cardiac arrest.