Adeno-associated viruses as liver-directed gene delivery vehicles: focus on lipoprotein metabolism

Adeno-associated viral vectors have proven to be excellent gene delivery vehicles for somatic overexpression. These viral vectors can efficiently and selectively target the liver, which plays a central role in lipoprotein metabolism. Both liver-expressed as well as non-hepatic secreted proteins can be easily examined in different mouse models using this approach. The dosability of adeno-associated viral (AAV) vectors, as well as their potential for long-term expression, makes them an excellent choice for assessing gene function in vivo. This section will cover the use of AAV to study lipoprotein metabolism-including vector design, virus production and purification, and viral delivery, as well as monitoring of transgene expression and resulting phenotypic changes. Practical information is provided to assist the investigator in designing, interpreting, and troubleshooting experiments.