Table 2.

iPSC-derived organoid or tubuloid models in pediatric genetic kidney diseases

Disease	Gene	Approaches	Applications	Reference
ARPKD	PKHD1	Patient iPSC-derived kidney organoids	Drug validation in a patient-specific manner	[78]
ARPKD	PKHD1	iPSC-derived organoid-on-a-chip model subjected to flow	Therapeutic target discovery using transcriptomics	[79]
ARPKD	PKHD1	iPSCs harboring loss-of-function mutations that recapitulate cystic phenotype	Disease modeling	[80]
NPH-RC	IFT140	Patient-derived iPSC kidney organoids, gene corrected	Disease modeling	[81]
Cystinosis	CTNS	Patient urine-derived tubuloid model	Personalized drug screening	[47]
Cystinosis	CTNS	iPSC-derived organoids	Drug testing	[39]
CAKUT	WT1, HNF1β, GLI3, COL4A3	Urine-derived patient-specific iPSCs and kidney organoids	Disease modeling	[82]
Nephrotic syndrome	NOS1AP	Human kidney organoids with knock-in of patient specific variant	Disease modeling	[83]
Nephrotic syndrome	NPHS1	Organoid-derived glomeruli and podocytes	Disease modeling	[84]
Nephrotic syndrome	NPHS1	Patient-derived iPSC organoids	Disease modeling	[40, 41]

ARPKD autosomal recessive polycystic kidney disease, NPH-RC nephronophthisis-related ciliopathy; CAKUT congenital anomalies of the kidney and urinary tract