Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases
Broad Institute
JUNE 24, 2025
Muldoon, a baby boy born without the ability to process dietary protein properly, had become the first person to be treated with a customized gene editing therapy. wasn’t the first patient to receive base editing therapy. He needed a personalized, one-of-a-kind therapy.
Let's personalize your content