Pioneering CRISPR Treatment Brings Hope to Thousands

CRISPR has been used to help hundreds of people in clinical trials--but KJ, a baby boy with a rare liver disease who received a CRISPR-based treatment, is not just another story of a personalized treatment. Instead of a liver transplant, a procedure with life-long medical consequences even if a donor for the newborn could be found, KJ received a tailor-made gene editing treatment that would fix his unique genetic mistake, and sparked a public-private partnership that hopes to help so many more.

KJ still has a mild form of the urea cycle disease, but he is a healthy and happy toddler. And the people who made this possible don't want to stop there. Dr. Kiran Musunuru, the geneticist who spearheaded the treatment development process, and Dr Rebecca Aherns-Nicklas, the physician who administered the dose, believe that this type of treatment needs to be made available to the hundreds of people who also have diseases that are caused by genetic mistakes that might be amenable to gene editing. 

The task is not small. Efforts from across the country, as well as significant resources, were needed to produce KJ's treatment. Although almost miraculous, the approach that was used had to be modified and scaled in order to help so many others.

Musunuru and Aherns-Nicklas are planning to ask the FDA for a formal clinical trial to take the scaled process to patients with seven different urea cycle disorders and hope to show that base editing can be a platform technology that provides treatments in a resource effective way. The hope is that CRISPR treatments could become not only for the lucky few, but rather one more way of healing many.

Original story: STAT10