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ArticlesPeer ReviewedFull Text
Highly efficient therapeutic gene editing of human hematopoietic stem cells
Author / Creator:Wu, Yuxuan ; Zeng, Jing ; Roscoe, Benjamin P. ; Liu, Pengpeng ; Yao, Qiuming ; Lazzarotto, Cicera R. ; Clement, Kendell ; Cole, Mitchel A. ; Luk, Kevin ; Baricordi, Cristina ; Shen, Anne H. ; Ren, Chunyan ; Esrick, Erica B. ; Manis, John P. ; Dorfman, David M. ; Williams, David A. ; Biffi, Alessandra ; Brugnara, Carlo ; Biasco, Luca ; Brendel, Christian ; Pinello, Luca ; Tsai, Shengdar Q. ; Wolfe, Scot A. ; Bauer, Daniel E.
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2019-05, Vol.25 (5), p.776-783
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ArticlesPeer ReviewedFull Text
Activation of γ-globin expression by hypoxia-inducible factor 1α
Author / Creator:Feng, Ruopeng ; Mayuranathan, Thiyagaraj ; Huang, Peng ; Doerfler, Phillip A. ; Li, Yichao ; Yao, Yu ; Zhang, Jingjing ; Palmer, Lance E. ; Mayberry, Kalin ; Christakopoulos, Georgios E. ; Xu, Peng ; Li, Chunliang ; Cheng, Yong ; Blobel, Gerd A. ; Simon, M. Celeste ; Weiss, Mitchell J.
Publisher:London: Nature Publishing Group UK
Journal title:Nature (London), 2022-10, Vol.610 (7933), p.783-790
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ArticlesPeer ReviewedFull Text
In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia
Author / Creator:Wang, Hongjie ; Georgakopoulou, Aphrodite ; Psatha, Nikoletta ; Li, Chang ; Capsali, Chrysi ; Samal, Himanshu Bhusan ; Anagnostopoulos, Achilles ; Ehrhardt, Anja ; Izsvák, Zsuzsanna ; Papayannopoulou, Thalia ; Yannaki, Evangelia ; Lieber, André
Publisher:United States: American Society for Clinical Investigation
Journal title:The Journal of clinical investigation, 2019-02, Vol.129 (2), p.598-615
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ArticlesPeer ReviewedFull Text
miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction
Author / Creator:Guda, Swaroopa ; Brendel, Christian ; Renella, Raffaele ; Du, Peng ; Bauer, Daniel E ; Canver, Matthew C ; Grenier, Jennifer K ; Grimson, Andrew W ; Kamran, Sophia C ; Thornton, James ; de Boer, Helen ; Root, David E ; Milsom, Michael D ; Orkin, Stuart H ; Gregory, Richard I ; Williams, David A
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2015-09, Vol.23 (9), p.1465-1474
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ArticlesPeer ReviewedFull Text
Systematic identification and validation of ceRNA-driven regulatory mechanisms in pediatric β-Thalassemia major
Author / Creator:Wu, Tao ; Ren, Zhenmin ; Liu, Xiaorong ; Xing, Zhihao ; Fu, Xiaoying ; Li, Wujiao ; Chen, Moxian ; Li, Defa ; Chen, Yunsheng
Publisher:Berlin/Heidelberg: Springer Berlin Heidelberg
Journal title:Annals of hematology, 2025-02, Vol.104 (2), p.963-972
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ArticlesPeer ReviewedFull Text
Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin induction
Author / Creator:Shi, Lihong ; Cui, Shuaiying ; Engel, James D ; Tanabe, Osamu
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2013-03, Vol.19 (3), p.291-294
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ArticlesPeer ReviewedFull Text
Synergistic silencing of α-globin and induction of γ-globin by histone deacetylase inhibitor, vorinostat as a potential therapy for β-thalassaemia
Author / Creator:Mettananda, Sachith ; Yasara, Nirmani ; Fisher, Christopher A. ; Taylor, Stephen ; Gibbons, Richard ; Higgs, Doug
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2019-08, Vol.9 (1), p.11649-8, Article 11649
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ArticlesPeer ReviewedFull Text
Increase of microRNA-210, Decrease of Raptor Gene Expression and Alteration of Mammalian Target of Rapamycin Regulated Proteins following Mithramycin Treatment of Human Erythroid Cells
Author / Creator:Bianchi, Nicoletta ; Finotti, Alessia ; Ferracin, Manuela ; Lampronti, Ilaria ; Zuccato, Cristina ; Breveglieri, Giulia ; Brognara, Eleonora ; Fabbri, Enrica ; Borgatti, Monica ; Negrini, Massimo ; Gambari, Roberto
Publisher:United States: Public Library of Science
Journal title:PloS one, 2015-04, Vol.10 (4), p.e0121567
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ArticlesPeer ReviewedFull Text
Nitric Oxide-cGMP Signaling Stimulates Erythropoiesis through Multiple Lineage-Specific Transcription Factors: Clinical Implications and a Novel Target for Erythropoiesis
Author / Creator:Ikuta, Tohru ; Sellak, Hassan ; Odo, Nadine ; Adekile, Adekunle D. ; Gaensler, Karin M. L.
Publisher:United States: Public Library of Science
Journal title:PloS one, 2016-01, Vol.11 (1), p.e0144561
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ArticlesPeer ReviewedFull Text
A Cell-Based High-Throughput Screen for Novel Chemical Inducers of Fetal Hemoglobin for Treatment of Hemoglobinopathies
Author / Creator:Peterson, Kenneth R. ; Costa, Flávia C. ; Fedosyuk, Halyna ; Neades, Renee Y. ; Chazelle, Allen M. ; Zelenchuk, Lesya ; Fonteles, Andrea H. ; Dalal, Parmita ; Roy, Anuradha ; Chaguturu, Rathnam ; Li, Biaoru ; Pace, Betty S.
Publisher:United States: Public Library of Science
Journal title:PloS one, 2014-09, Vol.9 (9), p.e107006
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ArticlesPeer ReviewedFull Text
Characterization of Transcription Factor Networks Involved in Umbilical Cord Blood CD34+ Stem Cells-Derived Erythropoiesis
Author / Creator:Li, Biaoru ; Ding, Lianghao ; Yang, Chinrang ; Kang, Baolin ; Liu, Li ; Story, Michael D. ; Pace, Betty S.
Publisher:United States: Public Library of Science
Journal title:PloS one, 2014-09, Vol.9 (9), p.e107133-e107133
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ArticlesPeer ReviewedFull Text
A reduced curcuminoid analog as a novel inducer of fetal hemoglobin
Author / Creator:Chaneiam, Nattawara ; Changtam, Chatchawan ; Mungkongdee, Thongperm ; Suthatvoravut, Umaporn ; Winichagoon, Pranee ; Vadolas, Jim ; Suksamrarn, Apichart ; Fucharoen, Suthat ; Svasti, Saovaros
Publisher:Berlin/Heidelberg: Springer-Verlag
Journal title:Annals of hematology, 2013-03, Vol.92 (3), p.379-386
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ArticlesPeer ReviewedFull Text
cAMP response element-binding protein 1 is required for hydroxyurea-mediated induction of γ-globin expression in K562 cells
Author / Creator:Banan, Mehdi ; Esmaeilzadeh-Gharehdaghi, Elika ; Nezami, Majid ; Deilami, Zahra ; Farashi, Samaneh ; Philipsen, Sjaak ; Esteghamat, Fatemehsadat ; Pourfarzad, Farzin ; Ali Imam, AM ; Najmabadi, Hossein
Publisher:Australia: Blackwell Publishing Ltd
Journal title:Clinical and experimental pharmacology & physiology, 2012-06, Vol.39 (6), p.510-517
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Highly efficient therapeutic gene editing of human hematopoietic stem cells

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Activation of γ-globin expression by hypoxia-inducible factor 1α

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In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

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miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction

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Systematic identification and validation of ceRNA-driven regulatory mechanisms in pediatric β-Thalassemia major

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Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin induction

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Synergistic silencing of α-globin and induction of γ-globin by histone deacetylase inhibitor, vorinostat as a potential therapy for β-thalassaemia

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Increase of microRNA-210, Decrease of Raptor Gene Expression and Alteration of Mammalian Target of Rapamycin Regulated Proteins following Mithramycin Treatment of Human Erythroid Cells

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Nitric Oxide-cGMP Signaling Stimulates Erythropoiesis through Multiple Lineage-Specific Transcription Factors: Clinical Implications and a Novel Target for Erythropoiesis

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A Cell-Based High-Throughput Screen for Novel Chemical Inducers of Fetal Hemoglobin for Treatment of Hemoglobinopathies

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Characterization of Transcription Factor Networks Involved in Umbilical Cord Blood CD34+ Stem Cells-Derived Erythropoiesis

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A reduced curcuminoid analog as a novel inducer of fetal hemoglobin

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cAMP response element-binding protein 1 is required for hydroxyurea-mediated induction of γ-globin expression in K562 cells

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