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.

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.

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

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific gene mutation, doctors say.

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

Researchers at Kyushu University in Japan have pinpointed the X-linked mutation behind orange fur in house cats.

The “meow-tation” that gives ginger cats their distinct fur has been discovered. A small deletion in a gene on the ...