Related Experiment Video
Updated: Jul 27, 2025
09:59
AAV Deployment of Enhancer-Based Expression Constructs In Vivo in Mouse Brain
Published on: March 31, 2022
2.8K
Hardwiring tissue-specific AAV transduction in mice through engineered receptor expression.
James Zengel1, Yu Xin Wang2,3, Jai Woong Seo4
1Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Nature Methods
|June 8, 2023
Summary
A new method, SELECTIV, uses adeno-associated virus (AAV) vectors and AAV receptor (AAVR) overexpression to efficiently and specifically express genes in mouse models. This advances gene delivery and the creation of novel transgenic mouse systems.
Area of Science:
- Molecular Biology
- Genetics
- Virology
Background:
- Transgenic mouse models are crucial for biological and disease research.
- Generating these models is often inefficient and resource-intensive.
- Adeno-associated virus (AAV) vectors are widely used for gene delivery but face limitations in efficiency and specificity.
Purpose of the Study:
- To develop a novel system for efficient and specific transgene expression in vivo.
- To overcome limitations of current AAV-mediated gene delivery methods.
- To create advanced transgenic mouse models for research.
Main Methods:
- Development of the SELective Expression and Controlled Transduction In Vivo (SELECTIV) system.
- Coupling AAV vectors with Cre-inducible overexpression of the AAV receptor (AAVR).
- Utilizing whole-body knockout of endogenous Aavr for enhanced specificity.
Main Results:
- SELECTIV significantly increased AAV transduction efficiency in diverse cell types, including muscle stem cells.
- Demonstrated superior specificity in cardiomyocytes, hepatocytes, and cholinergic neurons.
- Showcased broad utility for creating new mouse models and expanding AAV gene delivery applications.
Conclusions:
- The SELECTIV system offers enhanced efficacy and exquisite specificity for AAV-mediated gene delivery.
- This approach facilitates the development of novel transgenic mouse models.
- SELECTIV broadens the application of AAV vectors for in vivo gene delivery research.
