Engineered AAV capsids with enhanced targeting of the nervous system after intravenous administration

Type: Molecular / Cellular,

Type: Other,

Keywords: AAV, Viral Vector, CNS, PNS, Mouse, Non-Human Primate

Engineered AAV capsids with enhanced targeting of the nervous system after intravenous administration

A toolkit of engineered adeno-associated virus (AAV) capsids developed in the Gradinaru lab at Caltech with enhanced potency for the central or peripheral nervous system after intravenous administration. These capsids enable efficient and non-invasive gene delivery across a wide variety of model organisms from mouse to macaque. Systemic AAVs may be chosen for broad cell type targeting or restricted infection to a single cell class, e.g. neurons or vasculature, and readily combine with cell-type specific enhancer or miRNA target site gene regulatory elements.