Identify human outcomes of gene therapies in a chimeric mouse model with a humanized-liver.
Gene therapies have vast potential in treating human diseases. However, research pipelines frequently experience setbacks because of translational issues when progressing from animal models to clinical trials. For example, studies often find discrepancies between animal models and humans when testing the biodistribution and efficacy of gene therapies delivered via adeno-associated viruses (AAVs). This ultimately inhibits the ability of life-changing therapies to reach patients.
Our PXB-mouse® model with a humanized liver helps you to overcome the challenges of moving from the lab to the clinic. By utilizing a chimeric mouse model with a humanized liver, you can evaluate the transduction and transfection of your gene therapies in human hepatocytes, observe human-specific hepatotoxicity, and measure the efficacy of your therapeutics on human genes.
PXB-mice offer unrivalled translational capabilities in predicting human outcomes compared to conventional mouse models due to their high and stable expression of human genes and proteins.
With over 15 years of characterization of our model and proven customer use cases, we provide you with the technical support, research data and expert knowledge you need to confidently plan your next gene therapy project using our humanized liver chimeric mouse model. Increase the confidence of your projects moving towards clinical trials.
PXB-mice offer a logistically simple way to enhance late-stage studies when smaller numbers of specialized mice with humanized livers are required to test the delivery, safety, and efficacy of your lead gene therapy research therapeutics.
Our study services with PXB-mice are ideal for selecting gene therapy candidates for clinical development. These services include:
Due to their highly humanized livers, PXB-mice have been shown to better predict transduction efficiency and toxicity of AAVs in patients.
Read this case study
This case study covers four separate projects from organizations that utilized the PXB-mouse® model in their studies. Their data highlights how PXB-mice enabled effective AAV selection with greater insights into human physiology. These studies found: