In vivo gene therapy holds enormous promise to treat inherited and acquired genetic diseases affecting the liver, including lysosomal storage disorders, liver metabolic disorders, and hemophilia. Creating an effective transgene for these genetic disorders is only half the battle—it’s just as critical to create a safe and efficient vehicle to deliver the transgene to the target cells.
The development of these vehicles is where one of the biggest challenges lies and where progress has been hampered due to limitations of conventional research models. Animal studies are time consuming, costly, and tightly regulated, while 2D cell culture models lack the complexity to deliver physiological relevance. These challenges have led to a slow pace of gene therapy development for safe and effective therapeutics to become available for patients in need.
Instead of waiting months to get results from animal studies that may not translate to humans, it is now possible to get physiologically relevant data within weeks by using the Emulate Liver-Chip to assess gene therapy vectors. The adeno-associated virus (AAV) transduction application for the Emulate Liver-Chip enables gene therapy researchers to test the delivery efficiency and toxicity of AAV vectors in the most human-relevant in vitro model of the liver sinusoid, with proven validity in predicting drug toxicity. This AAV transduction application will allow rapid iteration on AAV design to optimize the delivery of gene therapies and accelerate therapeutic development.
In this webinar, you will learn how the Liver-Chip has been used to:
- Assess time- and concentration-dependent AAV transduction efficiency
- Discriminate between the transduction efficiency of different AAVs
- Evaluate the toxicity of AAV-based gene therapy vectors
- Study AAV transport from vasculature to target epithelial tissue in a proof-of-concept study