In vivo generated hematopoietic stem cells from genome edited induced pluripotent stem cells are functional in platelet-targeted gene therapy of murine hemophilia A

被引：10

作者：

Wang, Dawei ^{[1
,2
]}

Zhang, Guowei ^{[1
,3
]}

Gu, Junjie ^{[4
,5
]}

Shao, Xiaohu ^{[1
]}

Dai, Yuting ^{[1
]}

Li, Jianfeng ^{[1
]}

Pan, Xiaohong ^{[1
]}

Yao, Shuxian ^{[1
]}

Xu, Aining ^{[1
]}

Jin, Ying ^{[4
,5
]}

Huang, Jinyan ^{[1
]}

Shi, Qizhen ^{[6
]}

Zhu, Jiang ^{[1
]}

Xi, Xiaodong ^{[1
]}

Chen, Zhu ^{[1
,2
]}

Chen, Saijuan ^{[1
,2
]}

机构：

[1] Shanghai Jiao Tong Univ, Sch Med, Ruijin Hosp, State Key Lab Med Genom,Shanghai Inst Hematol, Shanghai, Peoples R China

[2] Shanghai Jiao Tong Univ, Sch Med, Natl Res Ctr Translat Med, Shanghai, Peoples R China

[3] Hangzhou Normal Univ, Sch Med, Key Lab Aging & Canc Biol Zhejiang Prov, Hangzhou, Peoples R China

[4] Chinese Acad Sci, Shanghai Inst Biol Sci, Ctr Excellence Mol & Cell Sci, Key Lab Stem Cell Biol,Inst Hlth Sci, Shanghai, Peoples R China

[5] Shanghai Jiao Tong Univ, Sch Med, Shanghai, Peoples R China

[6] Med Coll Wisconsin, Dept Pediat, 8701 Watertown Plank Rd, Milwaukee, WI 53226 USA

来源：

HAEMATOLOGICA | 2020年 / 105卷 / 04期

关键词：

PROGENITOR CELLS;

D O I：

10.3324/haematol.2019.219089

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页数：5

共 15 条

[1] In vivo generation of transplantable human hematopoietic cells from induced pluripotent stem cells [J].

Amabile, Giovanni ;

Welner, Robert S. ;

Nombela-Arrieta, Cesar ;

D'Alise, Anna Morena ;

Di Ruscio, Annalisa ;

Ebralidze, Alexander K. ;

Kraytsberg, Yevgenya ;

Ye, Min ;

Kocher, Olivier ;

Neuberg, Donna S. ;

Khrapko, Konstantin ;

Silberstein, Leslie E. ;

Tenen, Daniel G. .

BLOOD, 2013, 121 (08) :1255-1264

[2] Stem Cells in the Treatment of Disease [J].

Blau, Helen M. ;

Daley, George Q. .

NEW ENGLAND JOURNAL OF MEDICINE, 2019, 380 (18) :1748-1760

[3] Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting [J].

Chang, Chia-Wei ;

Lai, Yi-Shin ;

Westin, Erik ;

Khodadadi-Jamayran, Alireza ;

Pawlik, Kevin M. ;

Lamb, Lawrence S., Jr. ;

Goldman, Frederick D. ;

Townes, Tim M. .

CELL REPORTS, 2015, 12 (10) :1668-1677

[4] Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study [J].

Gouw, Samantha C. ;

van der Bom, Johanna G. ;

van den Berg, H. Marijke .

BLOOD, 2007, 109 (11) :4648-4654

[5] Induced Pluripotent Stem Cells Meet Genome Editing [J].

Hockemeyer, Dirk ;

Jaenisch, Rudolf .

CELL STEM CELL, 2016, 18 (05) :573-586

[6] Development and Applications of CRISPR-Cas9 for Genome Engineering [J].

Hsu, Patrick D. ;

Lander, Eric S. ;

Zhang, Feng .

CELL, 2014, 157 (06) :1262-1278

[7] Parallel use of by-passing agents in haemophilia with inhibitors: a critical review [J].

Ingerslev, Jorgen ;

Sorensen, Benny .

BRITISH JOURNAL OF HAEMATOLOGY, 2011, 155 (02) :256-262

[8] Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA [J].

Long, Chengzu ;

McAnally, John R. ;

Shelton, John M. ;

Mireault, Alex A. ;

Bassel-Duby, Rhonda ;

Olson, Eric N. .

SCIENCE, 2014, 345 (6201) :1184-1188

[9] Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies [J].

Shi, Qizhen ;

Wilcox, David A. ;

Fahs, Scot A. ;

Weiler, Hartmut ;

Wells, Clive W. ;

Cooley, Brian C. ;

Desai, Drashti ;

Morateck, Patricia A. ;

Gorski, Jack ;

Montgomery, Robert R. .

JOURNAL OF CLINICAL INVESTIGATION, 2006, 116 (07) :1974-1982

[10] Haematopoietic stem and progenitor cells from human pluripotent stem cells [J].

Sugimura, Ryohichi ;

Jha, Deepak Kumar ;

Han, Areum ;

Soria-Valles, Clara ;

da Rocha, Edroaldo Lummertz ;

Lu, Yi-Fen ;

Goettel, Jeremy A. ;

Serrao, Erik ;

Rowe, R. Grant ;

Alleshaiah, Mohan M. ;

Wong, Irene ;

Sousa, Patricia ;

Zhu, Ted N. ;

Ditadi, Andrea ;

Keller, Gordon ;

Engelman, Alan N. ;

Snapper, Scott B. ;

Doulatov, Sergei ;

Daley, George Q. .

NATURE, 2017, 545 (7655) :432-+

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