The Director of the San Raffaele-Telethon Institute for Gene Therapy Luigi Naldini reflects on what a recent Nature study reveals and about the future of in vivo gene transfer “Our study provides an ...

As Chief Editor of one of the two journals in which Biopharma Dealmakers appears, I have a keen interest in the process of translating biomedical research into new drugs, including the partnerships ...

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

The special properties of methylcellulose foam could make it a vehicle for bedside genetic engineering, according to a proof-of-principle study from bioengineers at Fred Hutch Cancer Center. In the ...

A first-of-its-kind inhalable gene therapy for lung cancer that genetically modifies people’s lung cells has been fast-tracked towards potential approval after promising clinical trial results. “Very ...

Advocates stress that while gene therapy is causing a buzz in wealthy countries, African governments must prioritise prevention, screening and accessible treatment NEWS ANALYSIS | JOHN MUSENZE | As ...

Researchers have used gene therapy to partially reprogram the brain cells that store memories in aging mice, reversing signs of cellular decline and restoring learning ability. The technique, ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Retiree Chip Hailey, who has a severe form of hemophilia B, participated in a groundbreaking gene therapy trial. Hailey experienced a life-threatening delay in treatment after a car accident, ...