Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including our reproductive cells. While most of these defects have no or only ...

The boy, son of Saurabh Grover and Nikita Kohli from Karnal in Haryana, was diagnosed with a urea cycle defect, a rare genetic condition in which the body is unable to process urea as it should ...

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care ...

How does CMP-CPS-001 work in Urea Cycle Disorders? CMP-CPS-001 helps reduce ... near normal range after a single dose Rescue of motor defects and spatial learning defects following two doses ...

PHILADELPHIA (WPVI) -- An infant, who was born with a deadly disease, is now thriving, thanks to a medical first performed at ...

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

In a groundbreaking achievement, doctors have successfully treated a baby boy with a life-threatening genetic disorder using ...

Turned out, KJ was born with carbamoyl phosphate synthetase 1 (CPS1), an inherited genetic disease known as a urea cycle disorder ... his genetic code to fix his defect. "This is an encouraging ...

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 3 doses of tailor-made treatment, an infant with a rare, life-threatening urea cycle disorder is thriving ...

A tailored CRISPR base-editing therapy was given for the first time to an infant who was born with a rare genetic disease, ...