Modeling Neurological Disorders by Human Induced Pluripotent Stem Cells

Table 1

List of reported hiPSC disease models.

Disease	Target cell	Potential to be disease model		Drug test	Reference
		Successful differentiated into target cell type	Neuronal pathology
Early-onset neurological disorders
Fragile X syndrome	ND	ND	Loss of FMR1 expression	ND	[44]
Prader-Willi syndrome	Neurons	Yes	Imprint disorder	ND	[48, 65]
Rett’s syndrome	Neurons	Yes	Loss of synapses, reduced spine density, smaller soma size	Yes	[31]
Familial dysautonomia	Neural crest cells	Yes	Loss of neural crest cells	Yes	[54]
Friedreich’s ataxaia	Motor neuron	Yes	FXN gene repression	ND	[61]
Angelman’s syndrome	Neurons	Yes	Imprint disorder	ND	[65]
Down’s syndrome	Neuron	ND	ND	ND	[69]
Spinal muscular atrophy	Motor neurons	Yes	Loss of neuron formation, loss of SMN gene expression	Yes	[34]
Late-onset neurological disorders
Amyotrophic lateral sclerosis (ALS)	Motor neurons	ND	Not shown	ND	[38]
Huntington’s disease (HD)	Striatal neurons	Yes	Not shown	ND	[37]
Parkinson’s disease (PD)	Dopaminergic neurons	Yes	Not shown	ND	[35, 36]
Alzheimer’s disease (AD)	Cholinergic neurons	Yes	Increase ratio of Aβ42 to Aβ40	Yes	[87]

*ND: not determined.