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CASE STUDY

A Model for Better Gene Therapy

Explore how humanized liver chimeric mouse models smooth the transition between the laboratory and clinical trials.

 


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Researchers develop gene therapies for a variety of diseases by targeting the human liver and delivering therapeutics with the use of adeno-associated virus (AAV) vector delivery systems. While this treatment area is exploding in popularity, scientists face challenges when transitioning from animal models to human clinical trials, such as discrepancies in AAV delivery, efficacy, and toxicity. Humanized liver chimeric mouse models offer a promising alternative to traditional models because they better predict AAV gene therapy success in patients.

Download this case study from PhoenixBio to learn how to select more effective AAV vectors using humanized liver chimeric mouse models.