One Scientist's Bold Vision to Make on-Demand Treatments Routine for Life-Threatening Rare Genetic Diseases

One scientist's bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

In May 2025, researchers announced that K.J. 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. 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.

Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases By Leah Eisenstadt June 24, 2025 Breadcrumb Home Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases Broad core member and gene editing pioneer David Liu describes a framework that could enable the treatment of 1,000 patients with personalized gene editing therapies by …