A Lifesaving CRISPR Treatment

LIFE'S ABILITY TO COPY billions of distinct letters in a genome is an absolute biological wonder—but mistakes are made. Genetic disorders and birth defects occur in one in every 33 babies in the U.S., according to the Centers for Disease Control and Prevention.

- By -D.O.

For all of history, a person born with a disorder just had to live with it, and depending on the defect, those lives could be brutally short.

But in 2025, that changed forever. In a groundbreaking announcement, detailed in a study published in The New England Journal of Medicine, experts from institutions around the U.S. saved the life of a patient named KJ, who was born with a rare genetic disorder. To pull off this incredible feat, doctors employed the world's first custom in vivo (i.e., inside a living organism) CRISPR gene therapy. This technique could help alleviate pain for millions.