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. 2022 Apr 12;13:870799. doi: 10.3389/fneur.2022.870799

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Copyright © 2022 Marino and Holt.

This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

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rAAV vectors for systemic delivery of mAbs. (Left) AAV capsid structure. The AAV capsid is composed of 60 protein monomers (VP1, VP2, and VP3 subunits with a stoichiometry of 1:1:10) arranged in icosahedral symmetry. (Top right) Representation of the single stranded (ss) DNA genome found in naturally occurring AAVs. Two open reading frames (ORFs) express genes necessary for replication (rep) and capsid structure (cap). An alternate reading frame overlapping the cap gene encodes the assembly-activating protein (AAP), promoting capsid formation and stability. (Bottom right) In recombinant AAV-based vectors (rAAV), the viral genes are substituted by a synthetic transgene cassette, in which a promoter drives expression of a transgene of interest, followed by a polyA tail.