4.7 Review

Emerging Preclinical Applications of Humanized Mouse Models in the Discovery and Validation of Novel Immunotherapeutics and Their Mechanisms of Action for Improved Cancer Treatment

Journal

PHARMACEUTICS
Volume 15, Issue 6, Pages -

Publisher

MDPI
DOI: 10.3390/pharmaceutics15061600

Keywords

humanized mouse model; cancer; immunotherapy; drug development; drug repurposing; drug discovery; disease mechanisms; resistance mechanisms

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Cancer therapeutics have been extensively studied in the past decade, with a focus on targeted modalities and immunotherapy. The adverse side effects of immune checkpoint inhibitors have underlined the need for clinically relevant animal models. Humanized mouse models have emerged as valuable tools for pre-clinical research, enabling the evaluation of efficacy and safety of immunotherapy, as well as the investigation of novel disease mechanisms.
Cancer therapeutics have undergone immense research over the past decade. While chemotherapies remain the mainstay treatments for many cancers, the advent of new molecular techniques has opened doors for more targeted modalities towards cancer cells. Although immune checkpoint inhibitors (ICIs) have demonstrated therapeutic efficacy in treating cancer, adverse side effects related to excessive inflammation are often reported. There is a lack of clinically relevant animal models to probe the human immune response towards ICI-based interventions. Humanized mouse models have emerged as valuable tools for pre-clinical research to evaluate the efficacy and safety of immunotherapy. This review focuses on the establishment of humanized mouse models, highlighting the challenges and recent advances in these models for targeted drug discovery and the validation of therapeutic strategies in cancer treatment. Furthermore, the potential of these models in the process of uncovering novel disease mechanisms is discussed.

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