AAV9-Retro mediates efficient transduction with axon terminal absorption and blood-brain barrier transportation.

ABSTRACT: Recombinant adeno-associated viruses (rAAVs), particularly those that permit efficient gene transfer to neurons from axonal terminals or across the blood-brain barrier, are useful vehicles for structural and functional studies of the neural circuit and for the treatment of many gene-deficient brain diseases that need to compensate for the correct genes in every cell in the whole brain. However, AAVs with these two advantages have not been reported. Here, we describe a new capsid engineering method, which exploits the combination of different capsids and aims to yield a capsid that can provide more alternative routes of administration that are more suitable for the wide-scale transduction of the central nervous system (CNS). A new AAV variant, AAV9-Retro, was developed by inserting the 10-m

SUBMITTER: Lin K 

PROVIDER: S-EPMC7556953 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature