Scientists have identified mutated immune cell clones that could point to improved treatment for refractory celiac disease.

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia ...

KJ Muldoon was born with a devastating medical problem. Racing to solve it, doctors may have launched a 'roadmap' for a new type of treatment.

Scientists are confident people with rare genetic diseases can be cured after the technology saved a baby's life.

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

A baby known as KJ is the first person in the world to receive a customized CRISPR therapy designed to fix a specific ...

A new gene editor takes advantage of CRISPR-associated proteins to insert whole genes into the genome, scientists report.

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

Sitting in my doctor's examination room, I was surprised when she told me, "Genetics don't really matter for chronic disease.

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

A study published in The New England Journal of Medicine looks at a new CRISPR gene therapy for children with a rare genetic disease. Dr Alena Pance, Senior Lecturer in Genetics, University of ...

Elysia Aves explores why drug outcomes vary so widely between patients and what this means for the future of pharmacology ...