TY - JOUR
T1 - 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
AU - Camões dos Santos, João
AU - Cazaux Mateus, Francisca
AU - Arez, Maria
AU - Bekman, Evguenia P.
AU - da Rocha, Simão T.
N1 - Publisher Copyright:
© 2025 The Authors
PY - 2025/8
Y1 - 2025/8
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=105005950449&partnerID=8YFLogxK
U2 - 10.1016/j.scr.2025.103741
DO - 10.1016/j.scr.2025.103741
M3 - Article
AN - SCOPUS:105005950449
SN - 1873-5061
VL - 86
JO - Stem Cell Research
JF - Stem Cell Research
M1 - 103741
ER -