Genetic vectors

“Lentiviral gene therapy to the liver establishes stable long-term normal to supra-normal coagulation factor VIII activity in mouse models of hemophilia A and in non-human primates, representing a potential new treatment option for people with hemophilia A.”.

Chemogenetic inactivation of the subthalamic nucleus in monkeys increases spike train variability in the pallidum and prolongs movement time, suggesting its role in stabilizing pallidal spike trains to achieve stable motor control.

Despite an emerging role for cerebrovascular endothelial cells in a range of neurological pathologies, AAV vector development to date has focused on tools designed to target neurons or astrocytes. Here, Krolak et al. describe a specific variant of AAV (AAV-BI30), with high specificity and efficacy for transduction of endothelial cells across the central nervous system.