The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month ...

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 ...

This technique streamlines quality control in gene therapy vector production, potentially accelerating manufacturing and improving clinical outcomes. This summary was automatically generated using ...

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

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 ...

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

A child diagnosed with a rare genetic disorder was successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia and Penn Medicine in a ...

The 28th American Society of Gene & Cell Therapy (ASGCT) meeting kicks off in mid-May in New Orleans. The annual event, which is on pace toward a goal of 8,000 attendees, will be a whirlwind of ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later ...

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 ...