Efficacy Evaluation Of AAV-Delivered Liver-Specific Promoters

Adeno-associated virus (AAV)-derived vectors have emerged as a promising vehicle for gene delivery across a wide range of clinical applications. The liver stands out as a compelling target as it is easily transduced with AAV vectors and allows a sustained expression of the transgene. However, off-target gene transduction remains a significant hurdle in the development of effective gene therapeutics. Therefore, there is a pressing need to design organ-specific promoters that enhance the specificity of gene therapy constructs. While conventional 2D cell culture models are valuable for initial higher throughput screening, their limited biological complexity often leads to results that do not translate to animal models or human patients. Organ-on-a-Chip technology is emerging as a solution to this challenge, offering a more physiologically relevant microenvironment for evaluating gene therapy constructs.

In this webinar, Dr. Rui Sun, a Research Scientist at Bayer AG, discusses his work using the Emulate Liver-Chip to assess the transduction efficiency and selectivity of liver-specific AAV promoters.