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- W4205461074 abstract "The systemic amyloidoses represent a devastating class of multisystem protein folding disorders whose etiologies involve complex interactions between multiple tissue types (e.g., aberrant protein producing effector and downstream damaged target organs). Problematically, this hallmark multiorgan involvement presents unique challenges for developing robust in vitro and in vivo preclinical models of these diseases, ultimately slowing the discovery of novel therapeutics. To these ends, induced pluripotent stem cells (iPSCs), capable of differentiating into any cell type of the body while at the same time maintaining the entire genetic context of the individual from which the line was derived, have the potential to allow for novel insights into systemic amyloid diseases such as transthyretin amyloidosis (ATTR amyloidosis). In this chapter, we will discuss the contribution of iPSCs to the study of this class of complex, devastating disorders, with a special emphasis on ATTR amyloidosis. In the process, we will highlight how patient-specific iPSC-based models of amyloid disease can lend novel insight into disease progression, revolutionize the drug discovery pipeline, and improve clinical management of disease through development of novel diagnostic biomarkers." @default.
- W4205461074 created "2022-01-26" @default.
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- W4205461074 date "2022-01-01" @default.
- W4205461074 modified "2023-10-16" @default.
- W4205461074 title "Patient-specific induced pluripotent stem cells for understanding and assessing novel therapeutics for multisystem transthyretin amyloid disease" @default.
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- W4205461074 doi "https://doi.org/10.1016/b978-0-12-823882-0.00011-4" @default.
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