Stem Cell Research (Jul 2020)
Generation of corrected-hiPSC (USTCi001-A-1) from epilepsy patient iPSCs using TALEN-mediated editing of the SCN1A gene
- Huifang Zhao,
- Lang He,
- Shuai Li,
- Hualin Huang,
- Feng Tang,
- Xiaobo Han,
- Zuoxian Lin,
- Chao Tian,
- Rongqi Huang,
- Peng Zhou,
- Jufang Huang,
- Sihao Deng,
- Zhiyuan Li
Affiliations
- Huifang Zhao
- School of Life Sciences, University of Science and Technology of China, Hefei, China; CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Lang He
- Department of Neurology, Xiangya Hospital, Central South University, Changsha, Hunan, China
- Shuai Li
- CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; University of Chinese Academy of Sciences, Beijing 100049, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Hualin Huang
- CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; University of Chinese Academy of Sciences, Beijing 100049, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Feng Tang
- CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; University of Chinese Academy of Sciences, Beijing 100049, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Xiaobo Han
- CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; University of Chinese Academy of Sciences, Beijing 100049, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Zuoxian Lin
- CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Chao Tian
- School of Life Sciences, University of Science and Technology of China, Hefei, China; CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Rongqi Huang
- CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; University of Chinese Academy of Sciences, Beijing 100049, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China
- Peng Zhou
- Department of Anatomy and Neurobiology, Xiangya School of Medicine, Central South University, Changsha, Hunan, China
- Jufang Huang
- Department of Anatomy and Neurobiology, Xiangya School of Medicine, Central South University, Changsha, Hunan, China
- Sihao Deng
- Department of Anatomy and Neurobiology, Xiangya School of Medicine, Central South University, Changsha, Hunan, China
- Zhiyuan Li
- School of Life Sciences, University of Science and Technology of China, Hefei, China; CAS Key Laboratory of Regenerative Biology, Guangdong Provincial Key Laboratory of Stem Cell and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou 510530, China; GZMU-GIBH Joint School of Life Sciences, Guangzhou Medical University, China; Department of Anatomy and Neurobiology, Xiangya School of Medicine, Central South University, Changsha, Hunan, China; University of Chinese Academy of Sciences, Beijing 100049, China; Guangzhou Regenerative Medicine and Health Guangdong Laboratory, 510005 Guangzhou, China; Corresponding author.
- Journal volume & issue
-
Vol. 46
p. 101864
Abstract
Dravet syndrome is a neurological disorder characterized by treatment-resistant polymorphic seizures, primarily caused by loss-of-function in the SCN1A gene. To develop an in vitro model of this disease, in a previously study we generated an induced pluripotent stem cell line from a 10-year-old boy carrying the NM_001165963.1:c.5768A to G (Q1923R) mutation in SCN1A. Using TALEN-mediated genome editing, we have now generated an isogenic control line in which the disease-causing mutation found in the epilepsy patient iPSCs was corrected, in order to eliminate the interference of different genetic backgrounds in future analyses.
