Silva Isabelle Leticia Zaboroski, Gomes-Júnior Rubens, da Silva Evelin Brandão, Vaz Isadora May, Jamur Valderez Ravaglio, de Freitas Souza Bruno Solano, Shigunov Patrícia
Stem Cell Basic Biology Laboratory, Instituto Carlos Chagas, Fiocruz PR, Curitiba, PR, 81310-020, Brazil.
Core for Cell Technology, School of Medicine, Pontifícia Universidade Católica do Paraná, Curitiba, PR, 80215-901, Brazil.
Hum Cell. 2023 Nov;36(6):2237-2246. doi: 10.1007/s13577-023-00978-4. Epub 2023 Aug 30.
Induced pluripotent stem cells (iPSCs) opened the possibility to use patient cells as a model for several diseases. iPSCs can be reprogrammed from somatic cells collected in a non-invasive way, and then differentiated into any other cell type, while maintaining the donor´s genetic background. CYFIP2 variants were associated with the onset of an early form of epileptic encephalopathy. Studies with patients showed that the R87C variant seems to be one of the variants that causes more severe disease, however, to date there are no studies with a human cell model that allows investigation of the neuronal phenotype of the R87C variant. Here, we generated an iPSC line from a patient with epileptic encephalopathy caused by the CYFIP2 R87C variant. We obtained iPSC clones by reprogramming urinary progenitor cells from a female patient. The generated iPSC line presented a pluripotent stem cell morphology, normal karyotype, expressed pluripotency markers and could be differentiated into the three germ layers. In further studies, this cell line could be used as model for epileptic encephalopathy disease and drug screening studies.
诱导多能干细胞(iPSC)开启了将患者细胞用作多种疾病模型的可能性。iPSC可以从以非侵入性方式收集的体细胞重编程而来,然后分化为任何其他细胞类型,同时保持供体的遗传背景。CYFIP2变体与早期癫痫性脑病的发病有关。对患者的研究表明,R87C变体似乎是导致更严重疾病的变体之一,然而,迄今为止,尚无使用人类细胞模型研究R87C变体神经元表型的研究。在此,我们从一名由CYFIP2 R87C变体引起的癫痫性脑病患者中生成了一条iPSC系。我们通过重编程一名女性患者的尿祖细胞获得了iPSC克隆。所生成的iPSC系呈现出多能干细胞形态,核型正常,表达多能性标志物,并且可以分化为三个胚层。在进一步的研究中,该细胞系可作为癫痫性脑病疾病模型和药物筛选研究的模型。