Homologous recombination-mediated gene correction in iPS cells o Ornithine transcarbamylase deficiency

iPS 细胞中同源重组介导的基因校正 o 鸟氨酸转氨甲酰酶缺陷

• 批准号: 23659511
• 负责人: TSUCHIYA Shigeru
• 金额: $ 2.33万
• 依托单位: Tohoku University
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Challenging Exploratory Research
• 财政年份: 2011
• 资助国家: 日本
• 起止时间: 2011 至 2012
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/grant/KAKENHI-PROJECT-23659511/
• 关键词: 遺伝子修復; iPS 細胞; OTC 欠損症; iPS細胞; OTC欠損症

iPS cells was derived from fibroblasts of OTCD. While the cells were transduced with AAV targeting vector for the homologous recombination-mediated gene correction, corrected clones were not obtained. For making iPS cells, we developed the strategies of culturing keratinocytes from human hair. These cells can be passaged and cultured for a long time. The establishment of iPS cells using these cells is in progress.

iPS 细胞源自 OTCD 的成纤维细胞。虽然用 AAV 靶向载体转导细胞进行同源重组介导的基因校正，但未获得校正的克隆。为了制造 iPS 细胞，我们开发了从人类头发中培养角质形成细胞的策略。这些细胞可以长期传代和培养。使用这些细胞建立 iPS 细胞的工作正在进行中。

项目成果

Octa-arginine mediated delivery of wild-type Lnk protein inhibits TPO-induced M-MOK megakaryoblastic leukemic cell growth by promoting apoptosis.

• DOI: 10.1371/journal.pone.0023640
• 发表时间: 2011
• 期刊: PloS one
• 影响因子: 3.7
• 作者: Looi CY;Imanishi M;Takaki S;Sato M;Chiba N;Sasahara Y;Futaki S;Tsuchiya S;Kumaki S
• 通讯作者: Kumaki S

Functional roles of WIP in Wiskott-Aldrich syndrome

WIP 在 Wiskott-Aldrich 综合征中的功能作用

• 发表时间: 2012
• 作者: Yoji Sasahara;Yuko Watanabe;Looi Chung Yeng;Shigeru Tsuchiya;Shigeo Kure.
• 通讯作者: Shigeo Kure.

A female case of West syndrome with remission of spams following multiple cerebral hemorrhages

多发性脑出血后垃圾邮件缓解的 West 综合征女性病例

• DOI: 10.1016/j.braindev.2010.10.010
• 发表时间: 2011
• 期刊: Brain Dev
• 作者: Hino-Fukuyo N;Haginoya K;Uematsu M;Tsuchiya S
• 通讯作者: Tsuchiya S

Effect of a blackout in pediatric patients with home medical devices during the 2011 eastern Japan earthquake.

2011 年日本东部地震期间，家庭医疗设备停电对儿童患者的影响。

• DOI: 10.1016/j.braindev.2013.02.001
• 发表时间: 2013
• 期刊: Brain Dev.
• 作者: Niihori T;Ihara K;他22名;Tojo Nakayama
• 通讯作者: Tojo Nakayama

RISTを施行した当科年長児および成人期WAS/XLT3症例の臨床的検討

我科接受 RIST 的年龄较大儿童和成人 WAS/XLT3 病例的临床回顾

• 发表时间: 2012
• 作者: 笹原洋二;鈴木信;大内芽里;渡辺祐子;小沼正栄;入江正寛;内山徹;力石健;峯岸正好
• 通讯作者: 峯岸正好