Fig. 3: Corrected SCA3-iPSCs (SCA3-C3-iPSCs) maintained pluripotent characteristics and normal karyotype.

a Flow cytometry of pluripotent markers, including nuclear staining for NANOG, OCT4, and cell surface staining for SSEA4. b Immunofluorescence staining of pluripotent markers, including nuclear staining for NANOG, SOX2, and cell surface staining for SSEA4. Scale bar: 100 μm. c Total RNA was isolated from iPSCs and analyzed with RT-qPCR. Primers used for NANOG, OCT4, and SOX2 specifically detect the transcripts from the endogenous genes. d Karyotyping and G-band analysis showed corrected SCA3-iPSCs have a normal 46, XX karyotype. e Teratomas test comprised derivatives of the 3 germ layers. Scale bar: 100 µm. SCA3-iPSCs: SCA3 patient-derived iPSCs. Ctrl-iPSCs: healthy control iPSCs. SCA3-C3-iPSCs and SCA3-C12-iPSCs: corrected SCA3-iPSCs.