Harnessing human genetics and stem cells for precision cardiovascular medicine

Caudal, Arianne; Snyder, Michael P.; Wu, Joseph C.

doi:10.1016/j.xgen.2023.100445

Cited by 3 publications

(1 citation statement)

References 126 publications

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“…These systems are revolutionizing our ability to investigate disease mechanisms and promote the discovery of novel therapeutics while sidestepping the pitfalls of conventional animal-based research that often fails to translate to the clinic ( Zushin et al, 2023 ). For example, PSC-derived cardiomyocytes have been successfully leveraged to elucidate patient-specific disease mechanisms, making them a valuable platform to investigate targeted therapeutics based on the patient's very own genetic background ( Wang et al, 2023 ; Caudal et al, 2024 ). Moreover, biobanking is increasingly playing a crucial role in this process by significantly improving the accessibility of PSCs to researchers and industry, helping to create a powerful combined approach for conducting precise disease modeling and drug screening at scale, known as the ‘clinical trial in a dish’ ( Sayed et al, 2020 ).…”

Section: Introductionmentioning

confidence: 99%

The role of the International Society for Stem Cell Research (ISSCR) guidelines in disease modeling

Juguilon,

2024

Disease Models &Amp; Mechanisms

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Human stem cell-based modeling systems are valuable tools that can greatly improve the clinical translation of basic research. Importantly, the successful application of human stem cell-based models to biomedical research depends on the widespread adoption of ethical principles and practical standards. To achieve this outcome, the International Society for Stem Cell Research (ISSCR) provides a comprehensive set of recommendations that aim to promote the ethical usage of human stem cells and to ensure rigor and reproducibility within the field. Understanding and implementing these recommendations should be a top priority for investigators around the world.

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Section: Introductionmentioning

confidence: 99%

The role of the International Society for Stem Cell Research (ISSCR) guidelines in disease modeling

Juguilon,

2024

Disease Models &Amp; Mechanisms

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Generation of two induced pluripotent stem cell lines from patients with Li-Fraumeni Syndrome carrying TP53 mutation

Sun,

Ren,

Canel Rivero

et al. 2024

Stem Cell Research

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Clinical trials in-a-dish for cardiovascular medicine

Wu,

Swanson,

Yildirim

et al. 2024

European Heart Journal

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Cardiovascular diseases persist as a global health challenge that requires methodological innovation for effective drug development. Conventional pipelines relying on animal models suffer from high failure rates due to significant interspecies variation between humans and animal models. In response, the recently enacted Food and Drug Administration Modernization Act 2.0 encourages alternative approaches including induced pluripotent stem cells (iPSCs). Human iPSCs provide a patient-specific, precise, and screenable platform for drug testing, paving the way for cardiovascular precision medicine. This review discusses milestones in iPSC differentiation and their applications from disease modelling to drug discovery in cardiovascular medicine. It then explores challenges and emerging opportunities for the implementation of ‘clinical trials in-a-dish’. Concluding, this review proposes a framework for future clinical trial design with strategic incorporations of iPSC technology, microphysiological systems, clinical pan-omics, and artificial intelligence to improve success rates and advance cardiovascular healthcare.

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Harnessing human genetics and stem cells for precision cardiovascular medicine

Cited by 3 publications

References 126 publications

The role of the International Society for Stem Cell Research (ISSCR) guidelines in disease modeling

The role of the International Society for Stem Cell Research (ISSCR) guidelines in disease modeling

Generation of two induced pluripotent stem cell lines from patients with Li-Fraumeni Syndrome carrying TP53 mutation

Clinical trials in-a-dish for cardiovascular medicine

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