A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

A team at CNIC has developed an innovative gene therapy to treat arrhythmogenic right ventricular cardiomyopathy type 5 ...

Gene therapy for cystic fibrosis is advancing fast. Explore key biotech players, clinical progress, and its potential to ...

In its first full year on the U.S. market, bladder cancer treatment Adstiladrin earned about $77 million. Elsewhere, Lilly ...

Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations. The system can be formulated completely as ...

A recent study led by the UAB demonstrates the potential of gene therapy to restore motor capacity in an ultra-rare disease, ...

Gene therapy is an area of intensive research and development in the retina space, with promising prospects and significant challenges. The benefits and pitfalls of gene therapy were discussed by two ...

Giulio Cossu, MD, University of Manchester, discusses barriers to gene therapy research and development. While standards for research are important to ensure safe and accurate testing for new gene ...

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

Inherited retinal degeneration (IRD) encompasses various disorders characterized by progressive loss of retinal photoreceptor cells, ultimately leading to vision loss and blindness.

Vision is one of the most crucial human senses, yet more than 300 million people worldwide are at risk of vision loss due to ...

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative gene-therapy strategy ...