Stem cell researchOct 23, 2026

Creating Stem Cell Lines for Spinocerebellar Ataxia 27B and Matching Controls

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Oct 24, 2026

Abstract

Four induced pluripotent stem cell (iPSC) lines were generated, including isogenic gene-corrected controls for studying Spinocerebellar Ataxia 27B (SCA27B).

SCA27B is caused by a pathogenic GAA•TTC repeat expansion in the FGF14 gene.
The generated iPSC lines exhibited normal genetic structure and the ability to develop into various cell types.
These iPSC lines can serve as a platform for understanding the mechanisms behind SCA27B.
The lines may also be utilized for testing potential treatments for this neurodegenerative disorder.

Simplified

Spinocerebellar Ataxia 27B (SCA27B) is a late-onset neurodegenerative disorder caused by a heterozygous pathogenic GAA•TTC repeat expansion in intron 1 of FGF14. Despite being a common genetic cause of adult-onset cerebellar ataxia, the underlying molecular mechanisms remain unclear. Here, we performed simultaneous reprogramming and CRISPR-Cas9 genome editing to generate four induced pluripotent stem cell (iPSC) lines and their respective isogenic gene-corrected controls. All lines exhibited normal karyotypes, pluripotency, and trilineage differentiation capacity. These paired iPSC lines provide a valuable platform for investigating the pathomechanism(s) of SCA27B and can be used for screening of potential therapeutic treatments.

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Creating Stem Cell Lines for Spinocerebellar Ataxia 27B and Matching Controls

Abstract

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