A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons.

ABSTRACT: Efficient retrograde access to projection neurons for the delivery of sensors and effectors constitutes an important and enabling capability for neural circuit dissection. Such an approach would also be useful for gene therapy, including the treatment of neurodegenerative disorders characterized by pathological spread through functionally connected and highly distributed networks. Viral vectors, in particular, are powerful gene delivery vehicles for the nervous system, but all available tools suffer from inefficient retrograde transport or limited clinical potential. To address this need, we applied in vivo directed evolution to engineer potent retrograde functionality into the capsid of adeno-associated virus (AAV), a vector that has shown promise in neuroscience research and the clinic.

SUBMITTER: Tervo DG 

PROVIDER: S-EPMC5872824 | biostudies-literature | 2016 Oct

REPOSITORIES: biostudies-literature