“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

A 3-year-old girl who underwent a breakthrough gene therapy treatment to treat profound hearing loss can hear on her own, two ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the ...

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has ...

Cell and gene therapies (CGTs) are moving rapidly from specialist trials to routine care: more than thirty products are ...

Wellness influencers can spread misinformation to the masses at the click of a button, but what is being done about it?

New research found that gene therapy for children with a form of severe combined immunodeficiency was successful in 95% of ...

A human cell is a Rube Goldberg machine like no other, full of biological chain reactions that make the difference between ...