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从一名七岁截断型 NOVA2 变异(c.625del)患者的 PBMC 中诱导多能干细胞系(FDCHi014-A)的建立。

Derivation of an induced pluripotent stem cell line (FDCHi014-A) from PBMCs of a seven-year-old patient with a truncating NOVA2 variant (c.625del).

机构信息

Center for Molecular Medicine, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai 201102, China.

Department of Rehabilitation, Children's Hospital of Fudan University, Shanghai 201102, China.

出版信息

Stem Cell Res. 2024 Apr;76:103369. doi: 10.1016/j.scr.2024.103369. Epub 2024 Feb 28.

Abstract

Neurodevelopmental disorder with or without autistic features and/or structural brain abnormalities (NEDASB) is a rare autosomal dominant disorder caused by a heterozygous mutation in the NOVA2 gene on chromosome 19q13. Here, we describe the generation and characterization of an iPSC line derived from the peripheral blood of a 7-year-old patient carrying a novel heterozygous mutation in NOVA2 (c.625 del). The iPSCs with the confirmed patient-specific mutation were demonstrated by pluripotency markers, a normal karyotype, and the ability to differentiate into three germ layers. This NOVA2-mutant iPSC line could facilitate disease modeling and therapy development studies for NEDASB.

摘要

伴有或不伴有自闭症特征和/或结构性脑异常的神经发育障碍(NEDASB)是一种罕见的常染色体显性遗传疾病,由 19q13 染色体上的 NOVA2 基因的杂合突变引起。在这里,我们描述了从携带 NOV2 基因(c.625del)新型杂合突变的 7 岁患者外周血中衍生的 iPSC 系的产生和特征。通过多能性标记物、正常核型和分化为三个胚层的能力证实了具有患者特异性突变的 iPSCs。该 NOV2 突变的 iPSC 系可促进 NEDASB 的疾病建模和治疗开发研究。

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