Direct promoter repression by BCL11A controls the fetal to adult hemoglobin switch

Liu, Nan, Hargreaves, Victoria V., Zhu, Qian, Kurland, Jesse V., Hong, Jiyoung, Kim, Woojin, Sher, Falak, Macias-Trevino, Claudio +8 more·2018·Cell

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  10. Elevating fetal hemoglobin: recently discovered regulators and mechanisms
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  11. Epigenetic inactivation of ERF reactivates γ-globin expression in β-thalassemia
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  12. Potential new approaches to the management of the Hb Bart’s hydrops fetalis syndrome: the most severe form of α-thalassemia
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  13. Molecular Medicine: Found in Translation
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  14. A unified model of human hemoglobin switching through single-cell genome editing
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  15. Identification of chromatin states during zebrafish gastrulation using CUT & RUN and CUT &Tag
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  16. Single-cell transcriptomic landscape of nucleated cells in umbilical cord blood
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  17. MicroRNA expression patterns in HbE/β-thalassemia patients: The passwords to unlock fetal hemoglobin expression in β-hemoglobinopathies
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  18. Collateral damage: benchmarking off-target effects in genome editing
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  19. Embryonic and fetal human hemoglobins: structures, oxygen binding, and physiological roles
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  20. CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia
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  25. The (unusual) heuristic value of Hox gene clusters; a matter of time?
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  26. Embryonic erythropoiesis and hemoglobin switching require transcriptional repressor ETO2 to modulate chromatin organization
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  27. Heterochromatin rewiring and domain disruption-mediated chromatin compaction during erythropoiesis
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  30. Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy
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  31. Biology in balance: human diploid genome integrity, gene dosage, and genomic medicine
    J. Lupski · 2022 · Trends in Genetics
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  33. Promise of gene therapy to treat sickle cell disease
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  34. Loss of BRD4 induces cell senescence in HSC/HPCs by deregulating histone H3 clipping
    Hui Yang, Pinpin Sui, Ying Guo, Chen Shi, Marie E. Maloof, Guo Ge, Francine Nihozeko, Caroline R. Delma, Ganqian Zhu, Peng Zhang et al. · 2023 · EMBO Reports
  35. CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells
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  36. Research in Sickle Cell Disease: From Bedside to Bench to Bedside
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  37. Integrating quantitative proteomics with accurate genome profiling of transcription factors by greenCUT&RUN
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  38. A ChIP-exo screen of 887 Protein Capture Reagents Program transcription factor antibodies in human cells
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  41. Pathogenic BCL11A variants provide insights into the mechanisms of human fetal hemoglobin silencing
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  42. Multidimensional profiling reveals GATA1-modulated stage-specific chromatin states and functional associations during human erythropoiesis
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  43. Transcription factor-mediated intestinal metaplasia and the role of a shadow enhancer
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  44. An evolutionarily ancient mechanism for regulation of hemoglobin expression in vertebrate red cells
    Masato Miyata, Nynke Gillemans, Dorit Hockman, Jeroen Demmers, Jan‐Fang Cheng, Jun Hou, Matti Salminen, Christopher A. Fisher, Stephen Taylor, Richard J. Gibbons et al. · 2020 · Blood
  45. Fetal Hemoglobin Regulation in Beta-Thalassemia
    Henry Y. Lu, Stuart H. Orkin, Vijay G. Sankaran · 2023 · Hematology/Oncology Clinics of North America
  46. BCL11A intellectual developmental disorder: defining the clinical spectrum and genotype-phenotype correlations
    Angela Peron, Felice D’Arco, Kimberly A. Aldinger, Constance L. Smith‐Hicks, Christiane Zweier, Gyri Aasland Gradek, Kimberley Bradbury, Andrea Accogli, Erica Andersen, Ping Yee Billie Au et al. · 2024 · European Journal of Human Genetics
  47. Gene-centric functional dissection of human genetic variation uncovers regulators of hematopoiesis
    Satish Kumar Nandakumar, Sean McFarland, Laura Marlene Mateyka, Caleb A. Lareau, Jacob C. Ulirsch, Leif S. Ludwig, Gaurav Agarwal, J Engreitz, Bartlomiej P. Przychodzen, Marie Ellen McConkey et al. · 2019 · eLife
  48. Translational regulation and deregulation in erythropoiesis
    Alexandra Vatikioti, Elena Karkoulia, Marina Ioannou, John Strouboulis · 2019 · Experimental Hematology
  49. A modified CUT&RUN protocol and analysis pipeline to identify transcription factor binding sites in human cell lines
    Nikki R. Kong, Li Chai, Daniel Geoffrey Tenen, Mahmoud Adel Bassal · 2021 · STAR Protocols
  50. Identification and characterization of RBM12 as a novel regulator of fetal hemoglobin expression
    Aoi Wakabayashi, Maryanne Kihiu, Malini Sharma, A. Josephine Thrasher, Megan S. Saari, Mathieu Quesnel-Vallières, Osheiza Y. Abdulmalik, Scott A. Peslak, Eugene Khandros, Cheryl A. Keller et al. · 2022 · Blood Advances
  51. Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations
    Kaitly J. Woodard, Phillip A. Doerfler, Kalin D. Mayberry, Akshay Sharma, Rachel M. Levine, Jonathan S. Yen, Virginia A. Valentine, Lance E. Palmer, Marc B. Valentine, Mitchell J. Weiss · 2022 · Disease Models & Mechanisms
  52. Current landscape of gene‐editing technology in biomedicine: Applications, advantages, challenges, and perspectives
    Weilin Zhou, Jinrong Yang, Yalan Zhang, Xiaoyi Hu, Wei Wang · 2022 · MedComm
  53. Epigenetic Regulation of β-Globin Genes and the Potential to Treat Hemoglobinopathies through Epigenome Editing
    Letizia Fontana, Zoe Alahouzou, Annarita Miccio, Panagiotis Antoniou · 2023 · Genes
  54. Oncogenic YAP mediates changes in chromatin accessibility and activity that drive cell cycle gene expression and cell migration
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  55. Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies
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  56. Co-Regulated Genes and Gene Clusters
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  57. High-throughput methods for the analysis of transcription factors and chromatin modifications: Low input, single cell and spatial genomic technologies
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  58. CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression
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  59. Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies
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  60. 3′HS1 CTCF binding site in human β-globin locus regulates fetal hemoglobin expression
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  61. Developmental cell death of cortical projection neurons is controlled by a Bcl11a/Bcl6‐dependent pathway
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  70. Transcriptional silencing of fetal hemoglobin expression by NonO
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  72. A tetramer of BCL11A is required for stable protein production and fetal hemoglobin silencing
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  74. Exploring epigenetic and microRNA approaches for γ-globin gene regulation
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  81. Logic and lineage impacts on functional transcription factor deployment for T-cell fate commitment
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  82. Single Nucleotide Polymorphisms in XMN1-HBG2, HBS1L-MYB, and BCL11A and Their Relation to High Fetal Hemoglobin Levels That Alleviate Anemia
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  84. Small molecule therapeutics to treat the β-globinopathies
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  85. Molecular simulation studies on B-cell lymphoma/leukaemia 11A (BCL11A).
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  86. Induction of therapeutic levels of HbF in genome‐edited primary β039‐thalassaemia haematopoietic stem and progenitor cells
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  90. Recent advancements in gene therapy for sickle cell disease and β-thalassemia
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  91. An evolving understanding of multiple causal variants underlying genetic association signals
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  92. Episomal vectors based on S/MAR and the β-globin Replicator, encoding a synthetic transcriptional activator, mediate efficient γ-globin activation in haematopoietic cells
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  95. Molecular analysis of the erythroid phenotype of a patient with BCL11A haploinsufficiency
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  97. Targeting fetal hemoglobin expression to treat β hemoglobinopathies
    Martin H. Steinberg · 2022 · Expert Opinion on Therapeutic Targets
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