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Updated: Sep 26, 2025

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Adeno-Associated Virus Toolkit to Target Diverse Brain Cells.

Rosemary C Challis1, Sripriya Ravindra Kumar1, Xinhong Chen1

  • 1Division of Biology and Biological Engineering, California Institute of Technology, Pasadena, California, USA;

Annual Review of Neuroscience
|April 20, 2022
PubMed
Summary

Engineered adeno-associated viruses (AAVs) offer advanced tools for neuroscience. Modified capsids and cargo enable precise genetic targeting of specific brain cells and circuits for detailed research.

Keywords:
AAVadeno-associated virussystemic deliverytargeted brain deliveryviral capsid engineeringviral cargo

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Area of Science:

  • Neuroscience
  • Molecular Biology
  • Genetics

Background:

  • Recombinant adeno-associated viruses (AAVs) are crucial gene delivery vectors in neuroscience research.
  • AAVs possess engineerable capsids and cargo, allowing for tailored tropism and transgene expression.
  • Existing AAV variants show enhanced central nervous system transduction, cell-specific targeting, and neuronal retrograde transport.

Purpose of the Study:

  • To review recent advancements in engineering AAVs for neuroscience research.
  • To highlight the development of a comprehensive AAV toolkit for precise genetic access to defined brain cell types.

Main Methods:

  • Modification of AAV capsid proteins to alter tissue and cell tropism.
  • Engineering of AAV viral genomes (cargo) for controlled transgene expression.
  • Integration of advanced gene regulatory elements, reporters, sensors, and effectors within AAVs.

Main Results:

  • Identification of engineered AAV capsids with enhanced tropisms, including improved CNS transduction and neuronal retrograde transport.
  • Development of AAV systems enabling highly specific transgene expression for detailed brain cell and circuit analysis.
  • Creation of a comprehensive AAV toolkit combining capsid and cargo engineering.

Conclusions:

  • Engineered AAVs provide powerful tools for precise genetic manipulation in neuroscience.
  • The combination of advanced capsid and cargo engineering allows for unprecedented specificity in targeting molecularly defined brain cell types.
  • These advancements facilitate sophisticated anatomical and functional studies of neural circuits.