CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs

被引：89

作者：

Wang, Lixia ^{[1
,2
,3
]}

Yi, Fei ^{[4
]}

Fu, Lina ^{[1
,3
]}

Yang, Jiping ^{[1
,3
]}

Wang, Si ^{[1
,3
]}

Wang, Zhaoxia ^{[5
]}

Suzuki, Keiichiro ^{[6
,7
]}

Sun, Liang ^{[9
]}

Xu, Xiuling ^{[1
]}

Yu, Yang ^{[8
]}

Qiao, Jie ^{[8
]}

Belmonte, Juan Carlos Izpisua ^{[6
]}

Yang, Ze ^{[9
]}

Yuan, Yun ^{[5
]}

Qu, Jing ^{[2
,3
]}

Liu, Guang-Hui ^{[1
,3
,10
,11
]}

机构：

[1] Chinese Acad Sci, CAS Ctr Excellence Biomacromol, Inst Biophys, Natl Lab Biomacromol, Beijing 100101, Peoples R China

[2] Chinese Acad Sci, Inst Zool, State Key Lab Stem Cell & Reprod Biol, Beijing 100101, Peoples R China

[3] Univ Chinese Acad Sci, Beijing 100049, Peoples R China

[4] Stanford Univ, Sch Med, Dept Mol & Cellular Physiol, Stanford, CA 94305 USA

[5] Peking Univ, Hosp 1, Dept Neurol, Beijing 100034, Peoples R China

[6] Salk Inst Biol Studies, Gene Express Lab, 10010 North Torrey Pines Rd, La Jolla, CA 92037 USA

[7] Univ Catolica San Antonio Murcia UCAM, Campus Jeronimos,N 135 Guadalupe, Murcia 30107, Spain

[8] Peking Univ, Hosp 3, Dept Gynecol & Obstet, Beijing 100191, Peoples R China

[9] Minist Hlth, Beijing Hosp, Beijing 100730, Peoples R China

[10] Jinan Univ, Inst Aging & Regenerat Med, Key Lab Regenerat Med, Minist Educ, Guangzhou 510632, Guangdong, Peoples R China

[11] Capital Med Univ, Beijing Inst Brain Disorders, Beijing 100069, Peoples R China

来源：

PROTEIN & CELL | 2017年 / 8卷 / 05期

基金：

国家高技术研究发展计划(863计划); 中国国家自然科学基金; 北京市自然科学基金;

关键词：

ALS; CRISPR/Cas9; gene correction; iPSC disease modeling; PLURIPOTENT STEM-CELLS; MOTOR-NEURON DEGENERATION; ER STRESS; WILD-TYPE; ALS; MUTATIONS; PROTEIN; FUS; SOD1; DISEASE;

D O I：

10.1007/s13238-017-0397-3

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease with cellular and molecular mechanisms yet to be fully described. Mutations in a number of genes including SOD1 and FUS are associated with familial ALS. Here we report the generation of induced pluripotent stem cells (iPSCs) from fibroblasts of familial ALS patients bearing SOD1(+/A272C) and FUS+/G1566A mutations, respectively. We further generated gene corrected ALS iPSCs using CRISPR/Cas9 system. Genome-wide RNA sequencing (RNA-seq) analysis of motor neurons derived from SOD1(+/A272C) and corrected iPSCs revealed 899 aberrant transcripts. Our work may shed light on discovery of early biomarkers and pathways dysregulated in ALS, as well as provide a basis for novel therapeutic strategies to treat ALS.

引用

页码：365 / 378

页数：14

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