Fuse Vectors has raised $5.2 million of funding as it moves closer to commercialising its innovative new approach to ...

An experimental trial of gene therapy has helped four toddlers - born with one of the most severe forms of childhood ...

RNA-based therapeutics have rapidly emerged over the past decade, offering a new class of medicines that differ significantly from conventional drugs. These therapies can be programmed to target or ...

A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic ...

A Connecticut boy is among four children all born with severe childhood blindness who gained “life-changing improvements” to ...

Researchers developed a new gene-editing strategy that dramatically boosts the effectiveness of gene therapies in the liver, a breakthrough that could lead to new treatments for about 700 genetic ...

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic ...