Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates and humans

Abstract

Summary Targeting genes to specific neuronal or glial cell types is valuable both for understanding and for repairing brain circuits. Adeno-associated viral vectors (AAVs) are frequently used for gene delivery, but targeting expression to specific cell types is a challenge. We created a library of 230 AAVs, each with a different synthetic promoter designed using four independent strategies. We show that ~11% of these AAVs specifically target expression to neuronal and glial cell types in the mouse retina, mouse brain, non-human primate retina in vivo , and in the human retina in vitro . We demonstrate applications for recording, stimulation, and molecular characterization, as well as the intersectional and combinatorial labeling of cell types. These resources and approaches allow economic, fast, and efficient cell-type targeting in a variety of species, both for fundamental science and for gene therapy.

Abstract of "Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates and humans" by Josephine Jüttner, Arnold Szabó, Brigitte Gross Scherf et al., https://doi.org/10.1038/s41593-019-0431-2, used under its Creative Commons Attribution license (CC-BY); markup removed, otherwise unmodified.