Delivery of Recombinant Adeno-Associated Virus into the Rodent Brain
Marselina Levis Rabi, Natalia Hurst-Calle, Jonathan Willman, Matthew Willman, Casey Cook, Aurelie Joly-Amado 1 and Kevin R. Nash
Published November 1, 2025
https://doi.org/10.65856/HFZM4247
KEYWORDS: Recombinant adeno-associated virus (rAAV), Central nervous system, Gene Therapy, Stereotaxy, Intracerebroventricular, Intravenous, Retroorbital, Rodent.
ABSTRACT: Recombinant adeno-associated virus (rAAV) has emerged as a powerful and versatile tool for manipulating gene expression in the central nervous system of experimental animals. Its utility stems from a favorable safety profile, capacity for long-term transgene expression, and ability to target specific cell types depending on serotype and promoter selection. Given the recent FDA approval of AAV for clinical use, its application in preclinical models is now even more relevant and translational, reinforcing its value in bridging experimental research and therapeutic development. In this article, we present a comprehensive overview of methodologies for rAAV-mediated gene delivery to the rodent brain. We detail protocols for intracranial, intra-cisterna magna and intravenous administration.