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 ...

Drug development takes years, time KJ did not have. But his doctor, Rebecca Ahrens-Nicklas, a metabolic-disease expert at the ...

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

Within hours of his birth last August, Baby KJ was so sick, his parents didn’t know if he’d live long enough to get to play with his three older siblings in Clifton Heights, just outside Philadelphia.

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 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.

A blood test revealed toxic levels of ammonia building up in KJ’s body, threatening to damage his brain — a sign of an ...

The global CRISPR Market, valued at US$2.90 billion in 2024, is forecasted to grow at a robust CAGR of 11.2%, reaching US$3.21 billion in 2025 and an impressive US$5.47 billion by 2030. The growing ...