TY - JOUR
T1 - The Promise and Perils of Compound Discovery Screening with Inducible Pluripotent Cell-Derived Neurons
AU - Sharlow, Elizabeth R.
AU - Sharlow, Elizabeth R.
AU - Koseoglu, Mehmet Murat
AU - Bloom, George S.
AU - Lazo, John S.
N1 - Funding Information:
The authors greatly appreciate the generous financial support of the Cure Alzheimer’s Fund (E.R.S., G.S.B., and J.S.L.), the Owens Family Foundation (G.S.B. and J.S.L.), National Institutes of Health (AG051085 to G.S.B., AG063400 to E.R.S., and CA044579 and OD021723 to J.S.L.), Alzheimer’s Asso- ciation (Grants 4079 and ZEN-16-363266 to G.S.B.), The Virginia Chapter of the Ladies Auxiliary of the Fraternal Order of Eagles (G.S.B.), The University of Virginia President’s Fund for Excellence (G.S.B.), and the Fiske Drug Discovery Fund ( J.S.L. and E.R.S.).
Publisher Copyright:
© Copyright 2020, Mary ann Liebert, Inc., publishers 2020.
PY - 2020/2/1
Y1 - 2020/2/1
N2 - Neurological diseases comprise more than a thousand ailments that adversely affect the brain and nervous system. When grouped together, these neurological conditions impact an estimated 100 million individuals in the United States and up to a billion people worldwide, making drug discovery efforts imperative. However, recent research and development efforts for these neurological diseases, including alzheimer's disease and amyotrophic lateral sclerosis, have been exceedingly disappointing and typify the challenges associated with translating in vitro and cell-based discoveries to successful preclinical models and subsequent human clinical trials. Our viewpoint is that neuronal progenitor cells and neurons derived from inducible pluripotent stem cells afford an innovative translational bridge, with higher pathological relevancy than previous cellular models. We outline some of the opportunities and challenges associated with their evolving usage in drug discovery and development.
AB - Neurological diseases comprise more than a thousand ailments that adversely affect the brain and nervous system. When grouped together, these neurological conditions impact an estimated 100 million individuals in the United States and up to a billion people worldwide, making drug discovery efforts imperative. However, recent research and development efforts for these neurological diseases, including alzheimer's disease and amyotrophic lateral sclerosis, have been exceedingly disappointing and typify the challenges associated with translating in vitro and cell-based discoveries to successful preclinical models and subsequent human clinical trials. Our viewpoint is that neuronal progenitor cells and neurons derived from inducible pluripotent stem cells afford an innovative translational bridge, with higher pathological relevancy than previous cellular models. We outline some of the opportunities and challenges associated with their evolving usage in drug discovery and development.
KW - NPC
KW - inducible
KW - neuron
KW - pluripotent
UR - http://www.scopus.com/inward/record.url?scp=85079823476&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85079823476&partnerID=8YFLogxK
U2 - 10.1089/adt.2019.914
DO - 10.1089/adt.2019.914
M3 - Review article
C2 - 31095406
AN - SCOPUS:85079823476
SN - 1540-658X
VL - 18
SP - 97
EP - 103
JO - Assay and Drug Development Technologies
JF - Assay and Drug Development Technologies
IS - 2
ER -
