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 ...

An “inverse vaccine” that delivers deactivated allergens to the liver was able to reduce reactions in mice with allergic asthma for at least a year after treatment. Should the approach ...

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 ...

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.

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 ...

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 ...

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 ...