Autor: |
Challis RC; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Ravindra Kumar S; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Chen X; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Goertsen D; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Coughlin GM; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Hori AM; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Chuapoco MR; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Otis TS; Sainsbury Wellcome Centre, University College London, London, United Kingdom., Miles TF; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu., Gradinaru V; Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA; email: viviana@caltech.edu. |
Abstrakt: |
Recombinant adeno-associated viruses (AAVs) are commonly used gene delivery vehicles for neuroscience research. They have two engineerable features: the capsid (outer protein shell) and cargo (encapsulated genome). These features can be modified to enhance cell type or tissue tropism and control transgene expression, respectively. Several engineered AAV capsids with unique tropisms have been identified, including variants with enhanced central nervous system transduction, cell type specificity, and retrograde transport in neurons. Pairing these AAVs with modern gene regulatory elements and state-of-the-art reporter, sensor, and effector cargo enables highly specific transgene expression for anatomical and functional analyses of brain cells and circuits. Here, we discuss recent advances that provide a comprehensive (capsid and cargo) AAV toolkit for genetic access to molecularly defined brain cell types. |