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

Treated with an individualized gene-editing therapy that corrects mutations directly on the genome, a patient shows improvement from a rare metabolic disease.

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

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as ...

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

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

One of the most commonly used gene-delivery methods relies on engineered viruses to insert stretches of genetic material into a cell’s genome. Although effective, these viruses tend to insert their ...

Treatment seems to have been effective, but it is not clear whether such bespoke therapies can be widely applied.