This isn’t just another milestone in DNA editing technology. It’s a landmark moment that signals the beginning of a new era: personalized, genetically-targeted medicine. These are treatments created for a specific person, for their unique mutation, for their precise diagnosis. This is the future of medicine, and it’s happening today.
I have been enthusiastically following the development of Cas9 technology since 2012—the very protein that kicked off the era of precision DNA editing. The world later came to know this field as CRISPR, or more simply, “genetic scissors.”
At a certain stage of my IT career, I was involved in projects related to CRISPR technologies as part of a collaboration with pharmaceutical companies. My team was responsible for implementing tasks at the intersection of IT and biotechnology. They were challenging, critical, and fascinating projects. That’s why the story of KJ Muldoon holds special significance for me, as someone personally and professionally invested in this field.
Today is a historic achievement. The world’s first personalized CRISPR therapy has not only been developed but has already saved a child with an extremely rare genetic disorder. A technology that we were discussing on a theoretical level just a decade ago is now working in vivo—and giving a child a chance at life.
The Breakthrough Case: Who is KJ Muldoon?
Infant KJ Muldoon was suffering from a rare genetic disorder that impaired his body’s ability to eliminate ammonia—a toxic substance that causes devastating brain damage. A liver transplant was not an option.
In response, scientists urgently developed a single-dose gene therapy based on CRISPR, created personally for him. After the drug was administered, the boy’s condition improved dramatically. He is now recovering and will soon be able to go home.