Of particular concern is the virus’s liking for the liver. “If you look at the number of ... and compared delivery efficiencies of the GFP molecules across the brain. AAV-PHP.B showed at least 40-fold ...

Each AAV variant carried a transgene encoding green fluorescent protein (GFP) under the control of the cytomegalovirus (CMV) promoter. This enabled the fluorescent expression to be assessed ...

Ferriero et al. show that liver fibrosis reduces the efficiency of AAV-based gene therapy by limiting liver uptake and altering vector distribution to other organs. The impact of fibrosis varies ...

Using this model, the researchers generated proteins with mutations that were predicted to lead to improved versions of GFP ...

A gene therapy to correct a mutation that causes maple syrup urine disease prevented newborn death and normalized growth in a calf as well as in mice.