For generations, Sickle Cell Disease has been a devastating illness affecting millions of people around the world, ...

Like countless Americans urged to take medicine every day to control high cholesterol, University of Pennsylvania cardiologist Kiran Musunuru frequently forgets to take his pills. But unlike most ...

Tweaked lipid nanoparticle delivery system showed early safety ...

Cardiac gene therapy was once viewed as a promising breakthrough before high-profile clinical setbacks dampened expectations. Now, advances in gene delivery, viral vectors and molecular targeting are ...

Treatments aim to bulk up failing hearts to restore their vigour – but can the field move on from past controversy?

Dr. Marty Makary noted it had "record speed approval," the fastest approval of any gene therapy device combination in U.S. history.

A key claim by the company MedTherapy is a major reduction in production timelines. While gene therapies typically take weeks ...

In a worldwide first, a one-and-done gene therapy has been approved to treat a form of hereditary deafness. Many of the ...

Doctors have linked a boy's tumor to a gene therapy, but say the rare risks need to be weighed against the profound benefits such medicines can offer.

A one-time gene therapy for treating hypercholesterolaemia could lower up to 62 per cent 'bad' cholesterol in the blood, with ...

Eli Lillys VERVE-102 gene therapy shows early trial success, cutting LDL cholesterol by up to 62 percent in high risk ...

While gene therapy for cystic fibrosis is still in the research phase, researchers aim to correct the defective gene responsible for the disease. Current research is promising. Cystic fibrosis (CF) is ...