Table 1: List of reported hiPSC disease models.

Disease Target cell Potential to be disease modelDrug test Reference
Successful differentiated into target cell type Neuronal pathology

Early-onset neurological disorders

Fragile X syndromeNDNDLoss of FMR1 expressionND[44]
Prader-Willi syndromeNeuronsYesImprint disorderND[48, 65]
Rett’s syndromeNeuronsYesLoss of synapses, reduced spine density, smaller soma sizeYes[31]
Familial dysautonomiaNeural crest cellsYesLoss of neural crest cellsYes[54]
Friedreich’s ataxaiaMotor neuronYesFXN gene repressionND[61]
Angelman’s syndromeNeuronsYesImprint disorderND[65]
Down’s syndromeNeuronNDNDND[69]
Spinal muscular atrophyMotor neuronsYesLoss of neuron formation, loss of SMN gene expressionYes[34]

Late-onset neurological disorders

Amyotrophic lateral sclerosis (ALS)Motor neuronsNDNot shownND[38]
Huntington’s disease (HD)Striatal neuronsYesNot shownND[37]
Parkinson’s disease (PD)Dopaminergic neuronsYesNot shownND[35, 36]
Alzheimer’s disease (AD)Cholinergic neuronsYesIncrease ratio of Aβ42 to Aβ40Yes[87]

*ND: not determined.