A team of doctors at the Children's Hospital of Philadelphia and Penn Medicine treated a baby with a gene editing technology ...

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to a well-tolerated safety profile, which has been heralded by analysts as ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects ...

A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized ...

The patient received 3E15 vector genomes of gene therapy NGN-401 on Nov. 5 as part of the high-dose arm of Neurogene’s ongoing phase 1/2 open-label, pediatric study. She developed a fever before ...

We can give a gene therapy or what we call gene transfer therapy, where we actually transplant the missing gene into the body,” he said. It isn’t a cure, but it may slow the damage.

An infant in Philadelphia has been successfully treated with a customized Crispr gene-editing therapy for a rare and deadly disease, doctors reported Thursday—a breakthrough in the futuristic ...

A baby in Pennsylvania has become the first recipient of an experimental genetic therapy that targets a rare urea cycle disorder. The treatment, which involves customizing the gene editing process ...

Reacting positively to the the rare Carbamoyl phosphate synthetase (CPSI) deficiency gene editing therapy treatment, the baby has made medical history benefitting 1.3 million other babies along ...

Luckily, KJ's parents, Nicole and Kyle Muldoon, connected with Dr. Rebecca Ahrens-Nicklas, director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program (GTIMD) at Children’s ...

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 ...

Within six months, the team at Children’s Hospital of Philadelphia and Penn Medicine, along with their partners, created a therapy designed to correct KJ’s faulty gene. They used CRISPR ...