AAV-SEND

AAV-SEND is a hybrid gene therapy platform that combines adeno-associated virus (AAV) vectors with the Selective Endogenous Encapsidation for Cellular Delivery (SEND) system. Developed by researchers at Virginia Commonwealth University, the platform utilizes a blood-brain barrier (BBB)-crossing AAV (such as AAV-PhP.eB) to transduce astrocytes, which then serve as long-lived producer cells. These astrocytes express and package cargo RNA into endogenous retrovirus-like particles (ERVLPs) using the PEG10 protein and an envelope protein like Syncytin A (SynA). These ERVLPs are subsequently released to deliver the RNA payload to neighboring, hard-to-transduce central nervous system (CNS) cells, including microglia and oligodendrocytes. This relay approach expands the therapeutic reach of gene therapy beyond the direct tropism of the initial AAV vector, offering a modular path for delivering various RNA-based cargos or genome editors to difficult CNS targets.

Other names

AAV-enabled SEND relay platform