KJ Muldoon was born with a devastating medical problem. Racing to solve it, doctors may have launched a 'roadmap' for a new type of treatment.

Doctors at CHOP and Penn Medicine successfully used gene editing to treat an infant with a rare disorder in what they're ...

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a ...

An infant named KJ has made history as the first patient to be treated with an in vivo CRISPR gene editing therapy designed ...

In a groundbreaking application of genetic science, physicians and researchers at Children’s Hospital of Philadelphia (CHOP) ...

A baby born with a rare and dangerous genetic disease is thriving after getting an experimental gene editing treatment made ...

A blood test revealed toxic levels of ammonia building up in KJ’s body, threatening to damage his brain — a sign of an ...

Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances ...

Mayo Clinic's Center for Regenerative Biotherapeutics has 40 manufacturing engineers in Rochester and Florida. Their work ...

Discover Cellectis SA's innovative CAR T-cell therapies targeting heme malignancies & upcoming 2025 catalysts.

Randee’s cancer had metastasized. When her doctor tested her for the PIK3CA gene mutation, she was able to make informed ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec.