Gene therapy pioneer Jim Wilson and researcher Kiran Musunuru are using contrasting approaches to overcome “irrational ...

The field of gene editing recently got a major boost from Massachusetts-based Beam Therapeutics, and the company’s Durham ...

The arc of biotechnology is one of breakthroughs and struggles, writes Tim Hunt, CEO of the Alliance for Regenerative ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

“Gene therapy is we collect the cells through stem cell collection, which requires you being placed on a large machine for collection, where you receive mobilized," she said. "This helps push ...

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with ...

Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw ...

Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition. For more than 15 years ...

A one-time injection of a novel gene therapy was able to save mice and a newborn calf from succumbing to maple syrup urine disease (MSUD), suggesting the approach could one day be used to treat ...