Efficacy Evaluation of AAV-Delivered Liver-Specific Promoters

Webinar Abstract

Adeno-associated virus (AAV)-derived vectors have emerged as a promising gene delivery vehicle for a broad range of clinical indications. The liver in particular is an interesting target as it is easily transduced with AAV vectors and allows a persistent expression of the transgene. However, off-target gene transduction remains a significant challenge in the development of successful gene therapeutics; thus, it is crucial to develop efficient organ-specific promoters for gene therapy constructs. While simple in vitro 2D cell culture models are useful for initial higher throughput screening, their limited complexity often results in responses that do not translate to animal models or human patients. Organ-on-a-Chip technology is helping to bridge this gap by enabling researchers to assess gene therapy constructs in a more physiologically relevant microenvironment. 

In this webinar, Dr. Rui Sun, Research Scientist at Bayer AG, presented on how he applied the Emulate Liver-Chip to evaluate the transduction efficiency and selectivity of liver-specific AAV promoters.

Learn how the Liver-Chip can be used to:

  • Improve the evaluation of AAV vector transduction efficiency 
  • Rank order various candidate promoter constructs 
  • Quantify transgene expression efficiency and specificity 
  • Investigate transduction efficiency by cell type
  • Evaluate promoter efficacy in a liver disease model


About gene therapy + Organ-on-a-Chip Technology

Gene therapy is a cutting-edge area of research that is generating a tremendous amount of excitement, as it could bring cures to some of the world’s most devastating diseases, including cancer, muscular atrophy, and blindness. Download our free eBook to learn what gene therapy is, how it works, and why Organ-on-a-Chip technology could help gene therapy reach widespread use.