Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases
Broad Institute
JUNE 24, 2025
was led by physician-scientists Kiran Musunuru and Rebecca Ahrens-Nicklas at the Children’s Hospital of Philadelphia and U. Together, these technologies have entered at least 19 clinical trials, with clinical results from seven of those trials reported so far — all showing that the base edit or prime edit resulted in patient benefit.
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