Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing
about
Customizing the genome as therapy for the β-hemoglobinopathiesProgress and promise in understanding the genetic basis of common diseasesGenomic approaches to identifying targets for treating β hemoglobinopathiesFetal globin gene repressors as drug targets for molecular therapies to treat the β-globinopathiesPluripotent stem cells in research and treatment of hemoglobinopathiesSickle cell disease in childrenSickle cell disease and H3Africa: enhancing genomic research on cardiovascular diseases in African patientsAnemia: progress in molecular mechanisms and therapiesCtip1 Regulates the Balance between Specification of Distinct Projection Neuron Subtypes in Deep Cortical Layers.Animal models of β-hemoglobinopathies: utility and limitationsβ-thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapiesTherapeutic genome editing: prospects and challengesAdvances in sickle cell therapies in the hydroxyurea eraErythro-megakaryocytic transcription factors associated with hereditary anemiaHuman genetics in rheumatoid arthritis guides a high-throughput drug screen of the CD40 signaling pathwayCorepressor Rcor1 is essential for murine erythropoiesisBCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesisPerspectives in Genetics and Sickle Cell Disease Prevention in Africa: Beyond the Preliminary Data from CameroonHemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin.Treating sickle cell disease by targeting HbS polymerizationGeneration of a genomic reporter assay system for analysis of γ- and β-globin gene regulation.Family cord blood banking for sickle cell disease: a twenty-year experience in two dedicated public cord blood banks.Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin inductionHydroxyurea-inducible SAR1 gene acts through the Giα/JNK/Jun pathway to regulate γ-globin expressionLIN28A expression reduces sickling of cultured human erythrocytes.Characterization of transcription factor networks involved in umbilical cord blood CD34+ stem cells-derived erythropoiesis.Gene therapy for hemoglobinopathies: the state of the field and the future.Genetics of human cardiovascular diseaseHydroxyurea treatment in β-thalassemia patients: to respond or not to respond?Controlling gene networks and cell fate with precision-targeted DNA-binding proteins and small-molecule-based genome readers.Induction of adult levels of β-globin in human erythroid cells that intrinsically express embryonic or fetal globin by transduction with KLF1 and BCL11A-XLMicroRNA-486-3p regulates γ-globin expression in human erythroid cells by directly modulating BCL11A.Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9Comparison of DNA methylation profiles in human fetal and adult red blood cell progenitors.Nuclease-mediated gene editing by homologous recombination of the human globin locus.BCL11A enhancer haplotypes and fetal hemoglobin in sickle cell anemia.Association of variants at BCL11A and HBS1L-MYB with hemoglobin F and hospitalization rates among sickle cell patients in Cameroon.An integrated cell purification and genomics strategy reveals multiple regulators of pancreas development.Pharmacological Induction of Human Fetal Globin Gene in Hydroxyurea-Resistant Primary Adult Erythroid Cells2p15-p16.1 microdeletions encompassing and proximal to BCL11A are associated with elevated HbF in addition to neurologic impairment
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Correction of sickle cell disease in adult mice by interference with fetal hemoglobin silencing
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
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scientific article published on 13 October 2011
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
Correction of sickle cell dise ...... ith fetal hemoglobin silencing
@en
Correction of sickle cell dise ...... th fetal hemoglobin silencing.
@nl
type
label
Correction of sickle cell dise ...... ith fetal hemoglobin silencing
@en
Correction of sickle cell dise ...... th fetal hemoglobin silencing.
@nl
prefLabel
Correction of sickle cell dise ...... ith fetal hemoglobin silencing
@en
Correction of sickle cell dise ...... th fetal hemoglobin silencing.
@nl
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P2860
P356
P1433
P1476
Correction of sickle cell dise ...... ith fetal hemoglobin silencing
@en
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Benjamin L Ebert
Bryan G Chong
Erica B Esrick
Philip W Tucker
Stuart H Orkin
Vijay G Sankaran
Yuko Fujiwara
P2860
P304
P356
10.1126/SCIENCE.1211053
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P577
2011-10-13T00:00:00Z