Dr. Kiran Musunuru and Dr. Rebecca Ahrens-Nicklas developed and administered the world’s first fully personalized CRISPR gene-editing therapy for an infant born with a rare genetic disorder. Working with collaborators including Nobel laureate Jennifer Doudna, they designed a treatment tailored to the patient’s unique mutation in record time. The successful therapy demonstrated that gene editing could be customized for a single individual rather than a broad patient population. It marked a historic advance in precision medicine and opened the door to highly personalized genomic treatments for previously untreatable diseases.
