Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases
Broad Institute
JUNE 24, 2025
Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. Some of these treatments, like K.J.’s, The team that treated K.J. Today, K.J.
Let's personalize your content