Genome editing-based therapies typically aim to treat disease by correcting underlying genetic mutations in patient’s cells. However, most genetic disorders are caused by dozens or even thousands of ...

To our immune system, a potentially lifesaving gene therapy can look a lot like a dangerous infection. That's because most genetic medicine uses viruses or double-stranded DNA to deliver genetic ...

The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and clinical developments that are driving today's explosion of gene ...

A protein long studied for its role in amyotrophic lateral sclerosis and frontotemporal dementia now appears to serve a ...