从3型脊髓小脑共济失调/马查多-约瑟夫病患者的成纤维细胞中生成人诱导多能干细胞系IBCHi002-A。
Generation of human iPS cell line IBCHi002-A from spinocerebellar ataxia type 3/Machado-Joseph disease patient's fibroblasts.
作者信息
Ciolak Agata, Krzyzosiak Wlodzimierz J, Kozlowska Emilia, Fiszer Agnieszka
机构信息
Department of Molecular Biomedicine, Institute of Bioorganic Chemistry, Polish Academy of Sciences, Noskowskiego 12/14 Str., 61-704 Poznan, Poland.
Department of Molecular Biomedicine, Institute of Bioorganic Chemistry, Polish Academy of Sciences, Noskowskiego 12/14 Str., 61-704 Poznan, Poland.
出版信息
Stem Cell Res. 2020 May;45:101796. doi: 10.1016/j.scr.2020.101796. Epub 2020 Apr 20.
Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease (MJD), is autosomal-dominant neurodegenerative disease caused by an expansion of polyglutamine-encoding CAG repeats in the ATXN3 gene. Here we established IBCHi002-A induced pluripotent stem cells (iPSCs) line generated from SCA3 patient fibroblasts by using non-integrative Sendai-virus delivery system of four reprogramming factors. This cellular model provides a valid platform for study SCA3 pathogenesis and potential therapies for this so far incurable disease.
3型脊髓小脑共济失调（SCA3），也称为马查多-约瑟夫病（MJD），是一种常染色体显性神经退行性疾病，由ATXN3基因中编码聚谷氨酰胺的CAG重复序列扩增引起。在此，我们利用四种重编程因子的非整合仙台病毒递送系统，建立了由SCA3患者成纤维细胞产生的IBCHi002-A诱导多能干细胞（iPSC）系。该细胞模型为研究SCA3发病机制以及针对这种迄今为止无法治愈的疾病的潜在疗法提供了一个有效的平台。
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