Literature DB >> 22473006

Reprogramming based gene therapy for inherited red blood cell disorders.

Xiuling Xu1, Jing Qu, Keiichiro Suzuki, Mo Li, Weizhou Zhang, Guang-Hui Liu, Juan Carlos Izpisua Belmonte.   

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Year:  2012        PMID： 22473006      PMCID： PMC3367524          DOI： 10.1038/cr.2012.54

Source DB:  PubMed          Journal:  Cell Res        ISSN： 1001-0602            Impact factor:   25.617


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  18 in total

1.  Globin phenotype of erythroid cells derived from human induced pluripotent stem cells.

Authors:  Kai-Hsin Chang; Andy Huang; Roli K Hirata; Pei-Rong Wang; David W Russell; Thalia Papayannopoulou
Journal:  Blood       Date:  2010-03-25       Impact factor: 22.113

2.  Generation of induced pluripotent stem cells from human beta-thalassemia fibroblast cells.

Authors:  Yixuan Wang; Yonghua Jiang; Sheng Liu; Xiaofang Sun; Shaorong Gao
Journal:  Cell Res       Date:  2009-08-18       Impact factor: 25.617

3.  Butyrate greatly enhances derivation of human induced pluripotent stem cells by promoting epigenetic remodeling and the expression of pluripotency-associated genes.

Authors:  Prashant Mali; Bin-Kuan Chou; Jonathan Yen; Zhaohui Ye; Jizhong Zou; Sarah Dowey; Robert A Brodsky; Joyce E Ohm; Wayne Yu; Stephen B Baylin; Kosuke Yusa; Allan Bradley; David J Meyers; Chandrani Mukherjee; Philip A Cole; Linzhao Cheng
Journal:  Stem Cells       Date:  2010-04       Impact factor: 6.277

4.  Overcoming reprogramming resistance of Fanconi anemia cells.

Authors:  Lars U W Müller; Michael D Milsom; Chad E Harris; Rutesh Vyas; Kristina M Brumme; Kalindi Parmar; Lisa A Moreau; Axel Schambach; In-Hyun Park; Wendy B London; Kelly Strait; Thorsten Schlaeger; Alexander L Devine; Elke Grassman; Alan D'Andrea; George Q Daley; David A Williams
Journal:  Blood       Date:  2012-02-27       Impact factor: 22.113

5.  Direct conversion of human fibroblasts to multilineage blood progenitors.

Authors:  Eva Szabo; Shravanti Rampalli; Ruth M Risueño; Angelique Schnerch; Ryan Mitchell; Aline Fiebig-Comyn; Marilyne Levadoux-Martin; Mickie Bhatia
Journal:  Nature       Date:  2010-11-07       Impact factor: 49.962

6.  Definitive-like erythroid cells derived from human embryonic stem cells coexpress high levels of embryonic and fetal globins with little or no adult globin.

Authors:  Kai-Hsin Chang; Angelique M Nelson; Hua Cao; Linlin Wang; Betty Nakamoto; Carol B Ware; Thalia Papayannopoulou
Journal:  Blood       Date:  2006-04-27       Impact factor: 22.113

7.  A protocol describing the genetic correction of somatic human cells and subsequent generation of iPS cells.

Authors:  Angel Raya; Ignasi Rodríguez-Pizà; Susana Navarro; Yvonne Richaud-Patin; Guillermo Guenechea; Adriana Sánchez-Danés; Antonella Consiglio; Juan Bueren; Juan Carlos Izpisúa Belmonte
Journal:  Nat Protoc       Date:  2010-03-11       Impact factor: 13.491

8.  Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells.

Authors:  Eirini P Papapetrou; Gabsang Lee; Nirav Malani; Manu Setty; Isabelle Riviere; Laxmi M S Tirunagari; Kyuichi Kadota; Shoshannah L Roth; Patricia Giardina; Agnes Viale; Christina Leslie; Frederic D Bushman; Lorenz Studer; Michel Sadelain
Journal:  Nat Biotechnol       Date:  2010-12-12       Impact factor: 54.908

9.  Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.

Authors:  Angel Raya; Ignasi Rodríguez-Pizà; Guillermo Guenechea; Rita Vassena; Susana Navarro; María José Barrero; Antonella Consiglio; Maria Castellà; Paula Río; Eduard Sleep; Federico González; Gustavo Tiscornia; Elena Garreta; Trond Aasen; Anna Veiga; Inder M Verma; Jordi Surrallés; Juan Bueren; Juan Carlos Izpisúa Belmonte
Journal:  Nature       Date:  2009-05-31       Impact factor: 49.962

10.  Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases.

Authors:  Lin Ye; Judy C Chang; Chin Lin; Xiaofang Sun; Jingwei Yu; Yuet Wai Kan
Journal:  Proc Natl Acad Sci U S A       Date:  2009-05-29       Impact factor: 11.205
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  5 in total

Review 1.  Pluripotent stem cells and gene therapy.

Authors:  Pavel Simara; Jason A Motl; Dan S Kaufman
Journal:  Transl Res       Date:  2013-01-23       Impact factor: 7.012

2.  Eliminate mitochondrial diseases by gene editing in germ-line cells and embryos.

Authors:  Si Wang; Fei Yi; Jing Qu
Journal:  Protein Cell       Date:  2015-07       Impact factor: 14.870

3.  The potential role of cell penetrating peptides in the intracellular delivery of proteins for therapy of erythroid related disorders.

Authors:  Lefkothea C Papadopoulou; Asterios S Tsiftsoglou
Journal:  Pharmaceuticals (Basel)       Date:  2013-01-07

4.  Concurrent progress of reprogramming and gene correction to overcome therapeutic limitation of mutant ALK2-iPSC.

Authors:  Bu-Yeo Kim; SangKyun Jeong; Seo-Young Lee; So Min Lee; Eun Jeong Gweon; Hyunjun Ahn; Janghwan Kim; Sun-Ku Chung
Journal:  Exp Mol Med       Date:  2016-06-03       Impact factor: 8.718

Review 5.  Treating primary immunodeficiencies with defects in NK cells: from stem cell therapy to gene editing.

Authors:  C Eguizabal; L Herrera; M Inglés-Ferrándiz; J C Izpisua Belmonte
Journal:  Stem Cell Res Ther       Date:  2020-10-27       Impact factor: 6.832
  5 in total

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