Modeling Neurodevelopmental Disorders Using Human Neurons
Overview
Affiliations
The cellular and molecular mechanisms of neurodevelopmental conditions such as autism spectrum disorders have been studied intensively for decades. The unavailability of live patient neurons for research, however, has represented a major obstacle in the elucidation of the disease etiologies. Recently, the development of induced pluripotent stem cell (iPSC) technology allows for the generation of human neurons from somatic cells of patients. We review ongoing studies using iPSCs as an approach to model neurodevelopmental disorders, the promise and caveats of this technique and its potential for drug screening. The reproducible findings of relevant phenotypes in Rett syndrome iPSC-derived neurons suggest that iPSC technology offers a novel and unique opportunity for the understanding of and the development of therapeutics for other autism spectrum disorders.
FMRP and MOV10 regulate Dicer1 expression and dendrite development.
Lannom M, Nielsen J, Nawaz A, Shilikbay T, Ceman S PLoS One. 2021; 16(11):e0260005.
PMID: 34847178 PMC: 8631628. DOI: 10.1371/journal.pone.0260005.
All-Optical Electrophysiology in hiPSC-Derived Neurons With Synthetic Voltage Sensors.
Puppo F, Sadegh S, Trujillo C, Thunemann M, Campbell E, Vandenberghe M Front Cell Neurosci. 2021; 15:671549.
PMID: 34122014 PMC: 8193062. DOI: 10.3389/fncel.2021.671549.
Translating regenerative medicine techniques for the treatment of epilepsy.
Yasuhara T, Date I, Liska M, Kaneko Y, Vale F Brain Circ. 2018; 3(3):156-162.
PMID: 30276318 PMC: 6057691. DOI: 10.4103/bc.bc_21_17.
Rapamycin regulates autophagy and cell adhesion in induced pluripotent stem cells.
Sotthibundhu A, McDonagh K, von Kriegsheim A, Garcia-Munoz A, Klawiter A, Thompson K Stem Cell Res Ther. 2016; 7(1):166.
PMID: 27846905 PMC: 5109678. DOI: 10.1186/s13287-016-0425-x.
A human neurodevelopmental model for Williams syndrome.
Chailangkarn T, Trujillo C, Freitas B, Hrvoj-Mihic B, Herai R, Yu D Nature. 2016; 536(7616):338-43.
PMID: 27509850 PMC: 4995142. DOI: 10.1038/nature19067.