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8 - Use of Induced Pluripotent Stem Cell-Derived Neuronal Disease Models from Patients with Familial Early-Onset Alzheimer’s Disease in Drug Discovery

from Section 2 - Non-clinical Assessment of Alzheimer’s Disease Candidate Drugs

Published online by Cambridge University Press:  03 March 2022

By
Brenda Hug ,
Rudolph E. Tanzi  and
Steven L. Wagner
Edited by
Jeffrey Cummings ,
Jefferson Kinney  and
Howard Fillit
Jeffrey Cummings
Affiliation:
University of Nevada, Las Vegas
Jefferson Kinney
Affiliation:
University of Nevada, Las Vegas
Howard Fillit
Affiliation:
Alzheimer’s Drug Discovery Foundation
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Summary

Incorporation of familial early-onset Alzheimer’s disease (EOAD) patient-based induced pluripotent stem cell (iPSC)-derived neuronal cell models into the AD drug discovery and preclinical development processes, provides for a tremendous technological advance, with implications extending from enabling a far more thorough preclinical pharmacological evaluation, using human patient-derived cellular model systems to assess efficacy against established, clinically relevant disease-associated biomarkers, including the evaluation of the effects on disease-associated endotypes, to unveiling previously unknown, pathologically-relevant pathways and identifying novel and potentially druggable therapeutic targets. This chapter discusses the status of promising disease-modifying therapeutics for AD, including the discovery and preclinical development of a clinically relevant series of small molecules and how familial EOAD patient-based iPSC-derived neuronal cell models have been critically utilized to dramatically improve this arduous yet necessary process.

Keywords

Alzheimer’s diseaseEarly Onset Familial Alzheimer’s DiseaseiPS cellsdrug discoveryneuronal cell modelsnovel targets
Type
Chapter
Information
Alzheimer's Disease Drug Development
Research and Development Ecosystem
 , pp. 95 - 105
Publisher: Cambridge University Press
Print publication year: 2022

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