Mitochondrial diseases affect approximately 1 in 5,000 people worldwide, causing debilitating symptoms ranging from muscle weakness to stroke-like episodes.

Researchers have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI) cancers. The results show ...

Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

The nonprofit says its pipeline offers more affordable and accessible therapeutics, including point-of-care CAR-T cell therapy for leukemia and lymphoma.

Scientists from the Keck School of Medicine at University of Southern California have developed a spectral flow ...

Just a few weeks after conception, stem cells are already orchestrating the future structure of the human brain. A new ...

A team of researchers from the Keck School of Medicine of USC has developed an advanced tool for analyzing chimeric antigen ...

London hospital gene therapy breakthrough gives boy with ‘death sentence’ disease a normal life - Eisa Hussein took part in a ...

Boy with ‘death sentence’ disease living a normal life after breakthrough gene therapy - Four-year-old Eisa can now play ...

A boy with an ultra-rare immune disease which can carry a “death sentence” is living a normal life after taking part in a groundbreaking gene therapy trial. Four-year-old Eisa Hussain now enjoys ...