AUTHORS
Josephine Jüttner, Arnold Szabo, Brigitte Gross-Scherf, Rei K Morikawa, Santiago B Rompani, Peter Hantz, Tamas Szikra, Federico Esposti, Cameron S Cowan, Arjun Bharioke, Claudia P Patino-Alvarez, Özkan Keles, Akos Kusnyerik, Thierry Azoulay, Dominik Hartl, Arnaud R Krebs, Dirk Schübeler, Rozina I Hajdu, Akos Lukats, Janos Nemeth, Zoltan Z Nagy, Kun-Chao Wu, Rong-Han Wu, Lue Xiang, Xiao-Long Fang, Zi-Bing Jin, David Goldblum, Pascal W Hasler, Hendrik P N Scholl, Jacek Krol, Botond Roska
Nat Neurosci. 2019 Aug;22(8):1345-1356. doi: 10.1038/s41593-019-0431-2. Epub 2019 Jul 8.
ABSTRACT
Targeting genes to specific neuronal or glial cell types is valuable for both understanding and repairing brain circuits. Adeno-associated viruses (AAVs) are frequently used for gene delivery, but targeting expression to specific cell types is an unsolved problem. We created a library of 230 AAVs, each with a different synthetic promoter designed using four independent strategies. We show that a number of these AAVs specifically target expression to neuronal and glial cell types in the mouse and non-human primate retina in vivo and in the human retina in vitro. We demonstrate applications for recording and stimulation, 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.
PMID:31285614 | DOI:10.1038/s41593-019-0431-2
