Emerging hiPSC Models for Drug Discovery in Neurodegenerative Diseases.
Alzheimer’s disease
neurodegeneration
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
30 Jul 2021
30 Jul 2021
Historique:
received:
15
06
2021
revised:
26
07
2021
accepted:
27
07
2021
entrez:
7
8
2021
pubmed:
8
8
2021
medline:
9
9
2021
Statut:
epublish
Résumé
Neurodegenerative diseases affect millions of people worldwide and are characterized by the chronic and progressive deterioration of neural function. Neurodegenerative diseases, such as Alzheimer's disease (AD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS), and Huntington's disease (HD), represent a huge social and economic burden due to increasing prevalence in our aging society, severity of symptoms, and lack of effective disease-modifying therapies. This lack of effective treatments is partly due to a lack of reliable models. Modeling neurodegenerative diseases is difficult because of poor access to human samples (restricted in general to postmortem tissue) and limited knowledge of disease mechanisms in a human context. Animal models play an instrumental role in understanding these diseases but fail to comprehensively represent the full extent of disease due to critical differences between humans and other mammals. The advent of human-induced pluripotent stem cell (hiPSC) technology presents an advantageous system that complements animal models of neurodegenerative diseases. Coupled with advances in gene-editing technologies, hiPSC-derived neural cells from patients and healthy donors now allow disease modeling using human samples that can be used for drug discovery.
Identifiants
pubmed: 34360966
pii: ijms22158196
doi: 10.3390/ijms22158196
pmc: PMC8347370
pii:
doi:
Substances chimiques
Neuroprotective Agents
0
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : NIH HHS
ID : DA041722
Pays : United States
Organisme : NIH HHS
ID : NS086890
Pays : United States
Organisme : NIA NIH HHS
ID : R01 AG078756
Pays : United States
Organisme : California Institute of Regenerative Medicine
ID : DISC2-11070
Organisme : NIH HHS
ID : AG051129
Pays : United States
Organisme : NIA NIH HHS
ID : R01 AG056259
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS086890
Pays : United States
Organisme : NIH HHS
ID : DA048882
Pays : United States
Organisme : NIH HHS
ID : AG056259
Pays : United States
Organisme : NIH HHS
ID : AG057409
Pays : United States
Organisme : NIA NIH HHS
ID : R35 AG071734
Pays : United States
Organisme : NIDA NIH HHS
ID : R01 DA048882
Pays : United States
Organisme : NIA NIH HHS
ID : RF1 AG057409
Pays : United States
Organisme : Autism Speaks
ID : 11721
Pays : United States
Organisme : NIDA NIH HHS
ID : DP1 DA041722
Pays : United States
Organisme : NIA NIH HHS
ID : U24 AG051129
Pays : United States
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