ESC
iPSC — Renal

iPSC-Derived Kidney Organoids

I differentiate human iPSCs into self-organizing kidney organoids containing nephron-like structures with proximal tubules, podocyte-bearing glomeruli, distal tubules, and collecting-duct elements. Built on the Takasato/Morizane intermediate-mesoderm protocols, these organoids enable modeling of polycystic kidney disease, congenital tubulopathies, acute kidney injury, and nephrotoxic drug screening on relevant human tissue.

IM → Nephron Tubular & Glomerular PKD · AKI Nephrotoxicity RNA-seq
01

Intermediate Mesoderm to Self-Organizing Nephrons

The protocol commits iPSCs to posterior primitive streak with high-dose CHIR (Wnt activation), then patterns intermediate mesoderm (IM) by FGF9 + activin A, splits IM into metanephric mesenchyme and ureteric epithelium with a brief CHIR pulse, and finally allows self-organization into nephron-like structures via 3D Matrigel droplet or air-liquid interface culture. Mature organoids contain LTL+ proximal tubules, NPHS1+ podocytes, ECAD+ distal tubules, and GATA3+ collecting-duct epithelium.

Differentiation Timeline
iPSC → Posterior Streak → Intermediate Mesoderm → Metanephric Mesenchyme → Nephron Organoid
Day 0–4
Posterior Streak
T · MIXL1 · CDX2
High-dose CHIR99021 8 µM for 4 days in TeSR-E6 (Wnt activation).
Day 4–7
Intermediate Mesoderm
PAX2 · LHX1 · OSR1
FGF9 (200 ng/mL) + Heparin in TeSR-E6.

High-dose CHIR99021 8 µM in TeSR-E6 for 4 days. Wnt activation drives posterior primitive streak (T+/MIXL1+/CDX2+).

Day 7–9
CHIR Pulse
SIX2 · WT1
Brief 1-h pulse of CHIR99021 5 µM to commit metanephric mesenchyme; aggregate to 3D.
Day 9–12
Renal Vesicle
JAG1 · PAX8
Continue FGF9 in 3D Matrigel droplets or air-liquid interface culture on Transwells.

Brief 1-h pulse of CHIR99021 5 µM to commit to metanephric mesenchyme (SIX2+/WT1+). Then dissociate and aggregate ~5×10⁵ cells per organoid.

Day 12–25+
Nephron Organoid
LTL · ECAD · NPHS1 · PODXL
Withdraw growth factors; allow self-organization. Glomerular and tubular segments visible by D18–25.

Withdraw growth factors. Self-organizing nephron segments by D18–25: LTL+ proximal tubules, NPHS1+/PODXL+ podocytes, ECAD+ distal/collecting epithelium.

  1. iPSC seeding

    Plate iPSCs at low density (10–15k cells/cm²) on Matrigel in mTeSR1 + ROCK-i.

  2. Posterior primitive streak (Day 0–4)

    Switch to TeSR-E6 + CHIR99021 8 µM for 4 days. Daily medium changes.

  3. Intermediate mesoderm (Day 4–7)

    Replace with TeSR-E6 + FGF9 (200 ng/mL) + Heparin (1 µg/mL) for 3 days.

  4. Metanephric induction (Day 7)

    1-h pulse of CHIR99021 5 µM, then dissociate to single cells and aggregate ~5×10⁵ cells per organoid in low-attachment plates.

  5. 3D culture (Day 7–25)

    Place aggregates on Transwell membranes (air-liquid interface) or embed in Matrigel droplets. Maintain in TeSR-E6 + FGF9 for 5 days, then withdraw growth factors.

  6. Maturation & assay

    Organoids develop nephron segments by D18–25. Validate by IF (LTL, ECAD, NPHS1) and bulk/single-cell RNA-seq.

D18–25
nephron segments visible
4 segments
prox. tubule, podocyte, distal, CD
96-well
organoid arrays for screens
PKD1/2
isogenic CRISPR cyst lines
02

Tubular & Glomerular Validation

Whole-mount and section IF map the major nephron segments: Lotus tetragonolobus (LTL) lectin marks proximal tubules; cadherin (ECAD) marks distal tubules and collecting duct; nephrin (NPHS1) and podocin (NPHS2) mark glomerular podocytes; GATA3 marks ureteric epithelium. Functional uptake of fluorescent dextran or albumin and methotrexate transport demonstrate proximal-tubule activity.

SIX2 WT1 LTL NPHS1 PODXL ECAD GATA3 PAX2
IF Quantification — % Cells Positive at Day 25
Mean ± SEM, n = 3 differentiations
Representative values from typical batches.
RT-qPCR — Kidney Organoid D25 vs iPSC (log₂ FC)
ΔΔCt method · housekeeping: GAPDH, ACTB · n = 3
Bars above zero = up-regulated; bars below zero = down-regulated relative to undifferentiated iPSC.
03

PKD, AKI & Tubulopathies

PKD1/PKD2 isogenic KO organoids develop spontaneous tubular cysts that expand with cAMP agonists, providing a tractable readout for cyst-modifying compounds. AKI is modeled with cisplatin, gentamicin, or hypoxia/reoxygenation. Patient lines (e.g. NPHS1-mutant for steroid-resistant nephrotic syndrome, COL4A5 for Alport) capture monogenic glomerulopathies.

04

Drug-Induced Nephrotoxicity Screening

Dose–response of nephrotoxic compounds (cisplatin, gentamicin, polymyxin B, vancomycin) is read out as KIM-1 induction, NGAL release into supernatant, ATP-based viability, and high-content imaging of tubular injury. Bulk RNA-seq across compound classes maps shared and pathway-specific nephrotoxic signatures.