Liver-directed AAV-based gene therapy offers promise for people living with hemophilia B or other genetic disorders. 6,7,11–18 However, the long-term safety and efficacy of this approach remain ...

Blocking the activity of the protein turns formerly “cold,” or immune-resistant, liver tumors in mice into “hot” tumors teeming with cancer-fighting immune cells. When combined with an ...

such as the dorsal root ganglia or the liver, which may help avoid toxicities commonly associated with AAV delivery. Using TRACER, Voyager is also identifying capsids with the capacity for strong ...

Removing EPO signaling in mouse liver tumors leads to tumor regression along with sensitivity to immunotherapy.

Of particular concern is the virus’s liking for the liver. “If you look at ... administered them to mice intravenously, and compared delivery efficiencies of the GFP molecules across the brain.

AAV4 also mostly avoids the liver, a major target organ for most ... Walkey et al, A comprehensive atlas of AAV tropism in the mouse, Molecular Therapy (2025). DOI: 10.1016/j.ymthe.

AAV4 also mostly avoids the liver, a major target ... gene therapy studies in mice more efficient and reproducible, given that a lot of the homework on the best AAV for a given cell type has ...

This finding indicates that the IRF8/BMAL1/ PPARγ axis may play an important role in liver lipid metabolism. Furthermore, adeno-associated virus-mediated IRF8 knockdown in mouse liver markedly ...

Their preclinical studies found that blocking activity of EPO converts formerly “cold” – or immune-resistant – liver tumors in mice into “hot” tumors teeming with cancer-fighting ...

'Cold' tumors are resistant to common immunotherapies. Researchers have uncovered a master regulator that can be manipulated to prevent tumor growth in mice. A protein identified nearly 40 years ...