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. Some of these treatments, like K.J.’s, They plan to harness scientific, medical, regulatory, and manufacturing innovations to enable on-demand genetic treatments like K.J.’s
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