Resumo
Angelman syndrome (AS) is a rare neurodevelopmental disorder characterized by severe developmental delay, speech impairment, ataxia and happy demeanor. AS is caused by loss-of-function of maternal UBE3A in neurons due to (epi)genetic abnormalities. Here, we report two new induced pluripotent stem cell (iPSC) lines from male and female patients carrying ∼ 6 Mb deletions in chr15q11.2-q13.1, together with familial control iPSC lines. All lines express pluripotent stem cell markers, demonstrate trilineage differentiation, and maintain genetic and epigenetic integrity at the locus of interest. These iPSCs provide a platform to model class I deletions, the most severe AS cause, and accelerate therapy development.
| Idioma original | ???core.languages.en_GB??? |
|---|---|
| Número do artigo | 103741 |
| Revista | Stem Cell Research |
| Volume | 86 |
| DOIs | |
| Estado da publicação | ???researchoutput.status.published??? - ago. 2025 |
Impressão digital
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