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. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.
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