Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including ...

Ammonia is made in the colon and small intestine. It then moves to the liver to be changed into urea, a waste product. In healthy people, the urea is removed from the body through the kidneys and ...

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.

In medical breakthrough, a baby diagnosed with rare genetic disorder is thriving after customized CRISPR gene editing therapy in Philadelphia.

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

After spending over 300 days at CHOP, KJ Muldoon has been released from the hospital after receiving personalized CRISPR gene ...

Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a ...

Rela Hospital, a globally renowned leader in liver transplant surgery, has achieved a historic milestone by becoming the ...

The pair hoped to make progress against urea-cycle disorders – inherited ... and diet can help reduce the risk of ammonia overloads and a liver transplant can fix most problems, she said ...

The baby received three infusions containing billions of microscopic gene-editors that homed in on a mutation in his liver ... a urea cycle disorder. The condition causes toxic levels of ammonia ...