CRISPR Saves a Baby’s Life

Biotechnology is like Star Wars’, “Force”: It has a dark side and a light side.

CRISPR, the gene-editing technique that can alter any cell and life-form on the planet, exemplifies the point. It can be deployed to alter a bird flu virus to kill multitudes. It can be used for eugenics manipulations. And, in theory, it can save the lives of people afflicted with genetic diseases.

That seems to have just happened. Baby KJ’s life was apparently saved or extended — at least for now — using the technique to treat a genetically caused liver condition. From the Stat story:

For the first time, scientists say they have reached into the genome of a severely ill child and rewritten the unique misspelling in his DNA.

The results, published in the New England Journal of Medicine on Thursday, are a landmark in the 50-year quest to read and repair the code of life. The boy, a now 9.5-month-old named KJ, was diagnosed days after birth with an ultra-rare disease that impairs his liver’s ability to process ammonia, which can build up and cause permanent brain damage or death.

KJ had been living in the hospital, waiting until he was old enough to receive a liver transplant. Instead, at 6 months, doctors administered the first dose of the gene editing treatment. They have since been able to loosen a strict low-protein diet and reduce his daily ammonia-lowering medications by half.

Plans for a transplant have, for the moment, been scrapped, although investigators remain cautious about declaring success.

This is terrific news and offers great hope for treating many other genetic conditions.

The pertinent questions are: Do we have the wisdom to reject “dark side” dystopian uses of CRISPR and other powerful biotechnologies and stay on the “light side”? And do we have the international ethical cohesion to enact enforceable restrictions to better ensure that happens?