Supplementary Fig.1 The overall workflow

0-1 1 3 5 13 15 20 40 60Day

Cell plating

7 4~5 days

CHIRActivinA

BMP4 XavMGCHIR

Induced differentiation

PMOEndo-Porter

Exon skipping

RT-PCR, Dystrophin WB, Immunocytochemistry

Ca imaging

E8 RPMI/B27-ins

Single cell replating

RT-PCR

RPMI/B27

Control Patient

OCT4

SOX2

NANOG

β-actin

A B

CDAPI NANOG SSEA-4/DAPIOCT3/4

Supplementary Fig.2 Characterization of established DMD-iPSCs

Neuroepithelium Cartilage Intestine

D

+ S.C. + PMO 10 μM

Δ46-55 + S.C. + PMO 5 μM + PMO 10 μM

500

Supplementary Fig.3 Skipping efficiency of PMO in short and long term culture

A

B

500

1000

1000

690 bp

514 bp

44 45 56 5743

44 56 5743

690 bp

514 bp

44 45 56 5743

44 56 5743

BD FACSDiva 8.0

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Supplementary Fig.4. Flow cytometry for cardiomyocyte differentiation of human iPSCs

BD FACSDiva 8.0

Global Sheet1 Printed on: 7 9, 2018 06:25:36 JSTPage 1 of 1

Cel

l cou

nt

cTnT-PE

Cel

l cou

nt

Isotype IgG-PE