Generation of two pairs of induced pluripotent stem cells from Angelman syndrome patients with class I 15q11.2-q13.1 deletions and their unaffected familial controls

João Camões dos Santos, Francisca Cazaux Mateus, Maria Arez, Evguenia P. Bekman, Simão T. da Rocha

Resultado de pesquisa: ???type-name??????researchoutput.researchoutputtypes.contributiontojournal.article???revisão de pares

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 artigo103741
RevistaStem Cell Research
Volume86
DOIs
Estado da publicação???researchoutput.status.published??? - ago. 2025

Impressão digital

Mergulhe nos tópicos de investigação de “Generation of two pairs of induced pluripotent stem cells from Angelman syndrome patients with class I 15q11.2-q13.1 deletions and their unaffected familial controls“. Em conjunto formam uma impressão digital única.

Citar isto