Simple and Highly Specific Targeting of Resident Microglia with Adeno-Associated Virus

Carolina Serrano; Sergio Cananzi; Tianjin Shen; Lei-Lei Wang; Chun-Li Zhang

doi:10.1101/2023.12.12.571321

Simple and Highly Specific Targeting of Resident Microglia with Adeno-Associated Virus

bioRxiv [Preprint]. 2023 Dec 13:2023.12.12.571321. doi: 10.1101/2023.12.12.571321.

Authors

Carolina Serrano^{1

2}, Sergio Cananzi^{1

2}, Tianjin Shen^{1

2}, Lei-Lei Wang^{1

2}, Chun-Li Zhang^{1

2}

Affiliations

¹ Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
² Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

Abstract

Microglia, as the immune cells of the central nervous system (CNS), play dynamic roles in both health and diseased conditions. The ability to genetically target microglia using viruses is crucial for understanding their functions and advancing microglia-based treatments. We here show that resident microglia can be simply and specifically targeted using adeno-associated virus (AAV) vectors containing a 466-bp DNA fragment from the human IBA1 (hIBA1) promoter. This targeting approach is applicable to both resting and reactive microglia. When combining the short hIBA1 promoter with the target sequence of miR124, up to 95% of transduced cells are identified as microglia. Such a simple and highly specific microglia-targeting strategy may be further optimized for research and therapeutics.

Keywords: AAV; adeno-associated virus; human IBA1; lentivirus; microglia; virus transduction.

Publication types

Preprint

Abstract

Publication types

Grants and funding