Half of all babies born with CPS1 deficiency don’t live longer than one week. A biomedical breakthrough could someday make that frightening statistic a thing of the past. In May 2025, a team of doctors and scientists from Pennsylvania reported that they’d delivered the first-ever patient-specific gene editing treatment to a baby diagnosed with CPS1 deficiency. In August 2025, the child celebrated his first birthday.
This was but one of several remarkable advances shared during the Genome Engineering: CRISPR Frontiers meeting held August 12-16 at Cold Spring Harbor Laboratory (CSHL). Over the course of that meeting, we sat down with some of the field’s leading minds, including the scientists behind the aforementioned breakthrough, Rebecca Ahrens-Nicklas and Kiran Musunuru. Together, they offered fascinating insights into what CRISPR may mean for the future of science and medicine.
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Read the related story: The CRISPR revolution continues