A mother has chosen to return to her home country with her 11-year-old daughter, who has a rare genetic disorder, after being ...

Researchers have used a personalised base-editing gene therapy to successfully treat an infant with carbamoyl-phosphate ...

Angelman Syndrome is a rare genetic condition that effects one in every 20,000 kids. Clinic at Children's Hospital Colorado ...

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other ...

A nonprofit called the n-Lorem Foundation is working on developing an individualized treatment that targets the genetic cause of rare diseases.

Researchers at UC Berkeley’s Innovative Genomics Institute, or IGI, developed a revolutionary treatment using CRISPR gene ...

Fargo’s Aldevron helped create a custom gene-editing therapy that cured a baby of urea cycle disorder, a rare and potentially ...

He’s among the first to be successfully treated with a custom therapy that seeks to fix a tiny but critical error in his ...

Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...

Doctors announced this week that they have treated a newborn baby with a rare genetic disease using the world’s first personalized gene editing therapy. Geoff Bennett discussed the treatment and its ...