Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases
Broad Institute
JUNE 24, 2025
It also needed to be delivered directly to the liver cells in his body, rather than to cells in a lab, and this was achieved by packaging the base editor in a lipid nanoparticle, a delivery system conceptually similar to the one used to deliver COVID vaccines to billions of people.
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