When a rare genetic mutation left baby KJ without any treatment options, scientists and regulators confronted a profound ethical question: should medicine move fast enough to save a single child? For geneticist Vanessa Almendro-Navarro, PhD, KJ’s case represented both an urgent humanitarian mission and a glimpse into the future of personalized medicine. In this interview, she explains how her team created a bespoke gene-editing therapy for KJ, what it reveals about the rise of one-patient medicines, and how unprecedented collaboration is reshaping the boundaries of science and ethics.
Produced and hosted by Jonathan D. Grinstein, PhD
Audio mixed and mastered by David Mosely
Brought to you by Inside Precision Medicine (SAGE Publishing)
Hosted on Acast. See acast.com/privacy for more information.
Behind the Breakthroughs