Scientists in Philadelphia have, for the first time, created a life-saving solution for a 9-month-old baby—one that could now ...

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

After displaying symptoms of a rare metabolic disease ... was born with inherited genetic mutations that cause a urea cycle disorder called carbamoyl phosphate synthetase 1 (CPS1) deficiency ...

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 ...

A n infant with a life-threatening and incurable genetic disease has become the first human to successfully receive a ...

This causes symptoms to show up shortly after birth ... therapies designed to address a patient's unique mutation. The duo ...

G ene therapy has always held enormous promise to correct genetic diseases, but turning that potential into treatments has been challenging.

For Uditi’s parents, reading about use of CRISPR tech, or ‘genetic scissors’, to treat baby KJ, diagnosed with severe genetic ...

Doctors have successfully used a customised form of gene editing to ease the symptoms of a rare and ... helped another baby with a similar urea cycle disorder, allowing that child to stop ...

CAMP4 Therapeutics announced promising results from its SYNGAP1 and Urea Cycle Disorders (UCDs) programs at the 28th Annual Meeting of the American Society of Gene and Cell Therapy. In ...