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 ...
The NIH defines a disease as rare if it affects fewer than 200,000 Americans, or roughly 1 in every 1,000 (as stated on the Global Genes website, a non-profit advocating for rare disease globally). And if your child is that one, finding out why, and figuring out how to fix ...
