Gene therapy is at the cutting edge of medicine, with exciting new products being developed and used to treat a wide variety ...

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

Doctors have successfully used gene-editing therapy to treat an infant born with a rare disorder. Experts believe the ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

Mass photometry enables rapid, purification-free quantification of rAAV vectors, offering a faster solution for gene therapy ...

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

“This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which ...

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 ...

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment May 15, 2025 — A research team has developed and safely delivered a personalized gene ...

The phase 3 EMBARK clinical trial provided exploratory analysis supporting the efficacy of this gene therapy. Advanced imaging techniques like QMR are crucial for assessing treatment efficacy in ...