A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design ...

Since the development of CRISPR gene editing, leaders in the field have foreseen a future in which CRISPR could be used to repair any genetic disease ? including rare "N-of-1" diseases caused by ...

In a world first, scientists used CRISPR to fix a baby's unique genetic mutation. The report offers hope for personalized ...

An infant named KJ has made history as the first patient to be treated with an in vivo CRISPR gene editing therapy designed ...

The one-off treatment highlights the promise of a new type of gene editing and the challenges of using it to treat extremely ...

A Delaware County infant with a rare metabolic disease, CPS1 deficiency, was treated at CHOP with a custom CRISPR therapy.

A baby born with a rare and dangerous genetic disease is thriving after getting an experimental gene editing treatment made ...

Get our top picks of the posters at AACR 2025 and learn more about the method behind them and the techniques used.