Literature DB >> 31296582

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

Dawei Wang1,2, Guowei Zhang3,4, Junjie Gu5, Xiaohu Shao1, Yuting Dai1, Jianfeng Li1, Xiaohong Pan1, Shuxian Yao1, Aining Xu1, Ying Jin5, Jinyan Huang1, Qizhen Shi6, Jiang Zhu1, Xiaodong Xi1, Zhu Chen1,2, Saijuan Chen7,2.   

Abstract

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Year:  2019        PMID： 31296582      PMCID： PMC7109733          DOI： 10.3324/haematol.2019.219089

Source DB:  PubMed          Journal:  Haematologica        ISSN： 0390-6078            Impact factor:   9.941


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  15 in total

Review 1.  Stem Cells in the Treatment of Disease.

Authors:  Helen M Blau; George Q Daley
Journal:  N Engl J Med       Date:  2019-05-02       Impact factor: 91.245

2.  Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.

Authors:  Chengzu Long; John R McAnally; John M Shelton; Alex A Mireault; Rhonda Bassel-Duby; Eric N Olson
Journal:  Science       Date:  2014-08-14       Impact factor: 47.728

Review 3.  Induced Pluripotent Stem Cells Meet Genome Editing.

Authors:  Dirk Hockemeyer; Rudolf Jaenisch
Journal:  Cell Stem Cell       Date:  2016-05-05       Impact factor: 24.633

4.  Respecifying human iPSC-derived blood cells into highly engraftable hematopoietic stem and progenitor cells with a single factor.

Authors:  Yu-Ting Tan; Lin Ye; Fei Xie; Ashley I Beyer; Marcus O Muench; Jiaming Wang; Zhu Chen; Han Liu; Sai-Juan Chen; Yuet Wai Kan
Journal:  Proc Natl Acad Sci U S A       Date:  2018-01-31       Impact factor: 11.205

5.  Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.

Authors:  Qizhen Shi; David A Wilcox; Scot A Fahs; Hartmut Weiler; Clive W Wells; Brian C Cooley; Drashti Desai; Patricia A Morateck; Jack Gorski; Robert R Montgomery
Journal:  J Clin Invest       Date:  2006-07       Impact factor: 14.808

6.  Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells.

Authors:  D A Wilcox; J C Olsen; L Ishizawa; M Griffith; G C White
Journal:  Proc Natl Acad Sci U S A       Date:  1999-08-17       Impact factor: 11.205

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

Authors:  Jørgen Ingerslev; Benny Sørensen
Journal:  Br J Haematol       Date:  2011-09-07       Impact factor: 6.998

8.  Generation of engraftable hematopoietic stem cells from induced pluripotent stem cells by way of teratoma formation.

Authors:  Nao Suzuki; Satoshi Yamazaki; Tomoyuki Yamaguchi; Motohito Okabe; Hideki Masaki; Satoshi Takaki; Makoto Otsu; Hiromitsu Nakauchi
Journal:  Mol Ther       Date:  2013-05-14       Impact factor: 11.454

9.  Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting.

Authors:  Chia-Wei Chang; Yi-Shin Lai; Erik Westin; Alireza Khodadadi-Jamayran; Kevin M Pawlik; Lawrence S Lamb; Frederick D Goldman; Tim M Townes
Journal:  Cell Rep       Date:  2015-08-28       Impact factor: 9.423

10.  In vivo generation of transplantable human hematopoietic cells from induced pluripotent stem cells.

Authors:  Giovanni Amabile; Robert S Welner; Cesar Nombela-Arrieta; Anna Morena D'Alise; Annalisa Di Ruscio; Alexander K Ebralidze; Yevgenya Kraytsberg; Min Ye; Olivier Kocher; Donna S Neuberg; Konstantin Khrapko; Leslie E Silberstein; Daniel G Tenen
Journal:  Blood       Date:  2012-12-04       Impact factor: 22.113
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  4 in total

Review 1.  Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes.

Authors:  Sergey A Sinenko; Sergey V Ponomartsev; Alexey N Tomilin
Journal:  Cell Mol Life Sci       Date:  2020-10-03       Impact factor: 9.261

2.  Long-term correction of hemorrhagic diathesis in hemophilia A mice by an AAV-delivered hybrid FVIII composed of the human heavy chain and the rat light chain.

Authors:  Jianhua Mao; Yun Wang; Wei Zhang; Yan Shen; Guowei Zhang; Wenda Xi; Qiang Wang; Zheng Ruan; Jin Wang; Xiaodong Xi
Journal:  Front Med       Date:  2022-01-17       Impact factor: 9.927

3.  Activated factor X targeted stored in platelets as an effective gene therapy strategy for both hemophilia A and B.

Authors:  Dawei Wang; Xiaohu Shao; Qiang Wang; Xiaohong Pan; Yujun Dai; Shuxian Yao; Tong Yin; Zhugang Wang; Jiang Zhu; Xiaodong Xi; Zhu Chen; Saijuan Chen; Guowei Zhang
Journal:  Clin Transl Med       Date:  2021-03

Review 4.  An overview of development in gene therapeutics in China.

Authors:  Dawei Wang; Kang Wang; Yujia Cai
Journal:  Gene Ther       Date:  2020-06-11       Impact factor: 5.250
  4 in total

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