Understanding the science behind the gene editing treatment for Baby KJ

A workflow for the rapid development of N-of-1 gene editing treatments

TL;DR: There are many other such inborn errors. Unfortunately, KJ’s story is not unique. For much of human history, we have had no option to help. But, now we can. Can we develop n-of-1 treatment for rare inborn errors of metabolism and give it to patients before they develop irreversible effects from their disease? We have the technology (base editors,…