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1 results

  • 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
  • Edited by Jeffrey Cummings, University of Nevada, Las Vegas, Jefferson Kinney, University of Nevada, Las Vegas, Howard Fillit
  • Book:
    Alzheimer's Disease Drug Development
    Published online:
    03 March 2022
    Print publication:
    31 March 2022, pp 95-105

  • 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.