一种用于研究罕见和难治性疾病的疾病特异性iPS细胞资源。

IF 5 3区 医学 Q2 IMMUNOLOGY
Megumu K Saito, Mitsujiro Osawa, Nao Tsuchida, Kotaro Shiraishi, Akira Niwa, Knut Woltjen, Isao Asaka, Katsuhisa Ogata, Suminobu Ito, Shuzo Kobayashi, Shinya Yamanaka
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引用次数: 0

摘要

背景:疾病特异性诱导多能干细胞(iPSCs)是罕见病病理分析和诊断的有用工具。鉴于现有资源有限,储存这种源自疾病的多能干细胞并促进其广泛使用将是解开罕见疾病之谜的一种有希望的方法。在此,我们全面构建了来自日本指定顽固性疾病患者的iPSC,并评估了它们的性质,以丰富罕见病iPSC资源。方法:招募指定顽固性疾病患者,经患者或其监护人书面知情同意后采集血样。从获得的样品中,使用episomal方法建立iPSCs。建立的iPSCs储存在细胞库中。结果:我们从139种指定顽固性疾病的259例患者中建立了1532个iPSC克隆。iPSC的建立效率不受年龄和性别的影响。大多数iPSC克隆来源于非t和非b造血细胞。所有iPSC克隆均表达关键转录因子OCT3/4(范围0.27-1.51;平均0.79)和NANOG(范围0.15-3.03;平均1.00),相对于参考201B7 iPSC克隆。结论:这些新建立的多能干细胞对研究人员来说是很容易获得的,可以证明是罕见难治性疾病研究的有用资源。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

A disease-specific iPS cell resource for studying rare and intractable diseases.

A disease-specific iPS cell resource for studying rare and intractable diseases.

A disease-specific iPS cell resource for studying rare and intractable diseases.

A disease-specific iPS cell resource for studying rare and intractable diseases.

Background: Disease-specific induced pluripotent stem cells (iPSCs) are useful tools for pathological analysis and diagnosis of rare diseases. Given the limited available resources, banking such disease-derived iPSCs and promoting their widespread use would be a promising approach for untangling the mysteries of rare diseases. Herein, we comprehensively established iPSCs from patients with designated intractable diseases in Japan and evaluated their properties to enrich rare disease iPSC resources.

Methods: Patients with designated intractable diseases were recruited for the study and blood samples were collected after written informed consent was obtained from the patients or their guardians. From the obtained samples, iPSCs were established using the episomal method. The established iPSCs were deposited in a cell bank.

Results: We established 1,532 iPSC clones from 259 patients with 139 designated intractable diseases. The efficiency of iPSC establishment did not vary based on age and sex. Most iPSC clones originated from non-T and non-B hematopoietic cells. All iPSC clones expressed key transcription factors, OCT3/4 (range 0.27-1.51; mean 0.79) and NANOG (range 0.15-3.03; mean 1.00), relative to the reference 201B7 iPSC clone.

Conclusions: These newly established iPSCs are readily available to the researchers and can prove to be a useful resource for research on rare intractable diseases.

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来源期刊
CiteScore
11.10
自引率
1.20%
发文量
45
审稿时长
11 weeks
期刊介绍: Inflammation and Regeneration is the official journal of the Japanese Society of Inflammation and Regeneration (JSIR). This journal provides an open access forum which covers a wide range of scientific topics in the basic and clinical researches on inflammation and regenerative medicine. It also covers investigations of infectious diseases, including COVID-19 and other emerging infectious diseases, which involve the inflammatory responses. Inflammation and Regeneration publishes papers in the following categories: research article, note, rapid communication, case report, review and clinical drug evaluation.
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