Genome editing-based therapies typically aim to treat disease by correcting underlying genetic mutations in patient's cells.

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

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 ...

A protein long studied for its role in amyotrophic lateral sclerosis and frontotemporal dementia now appears to serve a second, equally critical function, safeguarding the integrity of human DNA.

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 ...

INSTALL results showed successful and safe non-viral insertion of large genetic payloads in the livers of mice when delivered by lipid nanoparticles (LNPs). In contrast, mice experienced fatal immune ...

Genome editing-based therapies typically aim to treat disease by correcting underlying genetic mutations in patient's cells. However, most 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 ...