The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to ...

A new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond ...

The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Oliver Chu was born with a rare genetic condition called Hunter syndrome that impacts physical and mental development. The ...

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

A new wave of gene therapy treatments offer a new lease on life for people with inherited diseases such as haemophilia. But ...

Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...