Table 3.

Pluripotent stem cell-based genome-editing Parkinson’s disease models

Gene mutation	Editing system	Cell line	Phenotype demonstrated	Application	Ref.
SNCA A30P	CRISPR/CAS 9	hiPSC	Not demonstrated	Locus mutation	[48]
LRRK2 G2019S	ZFN	hiPSC	Not demonstrated	Gene correction	[95]
LRRK2 G2019S	CRISPR/CAS 9	hiPSC	Synaptic defect, fraction of TH+/S129P-αS+ neurons was significantly reduced	Locus mutation	[48]
SNCA E46K	ZFN	hESC	Not demonstrated	Locus mutation	[53]
SNCA A53T	ZFN	hiPSC	Not demonstrated	Gene correction	[52]
SNCA A30P/A53T	CRISPR/CAS 9	hiPSC	Not demonstrated	Locus mutation	[98]
SNCA (rs356165 A/G)	CRISPR/CAS 9	hiPSC	Not demonstrated	Locus mutation	[49]
LRRK2 G2019S	ZFN	hiPSC	Basic phenotypes: autophagy defects, synaptic defects, increased apoptosis, accumulation of τ and α-synuclein. Phenotypes were alleviated after genetic correction	Gene correction	[94]

hiPSC: Human induced pluripotent stem cell.