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Ling Xu et al.
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Silvia Fluhr et al.
EPIGENETICS (2017)
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Jessica Gasparello et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2017)
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Kai-Hsin Chang et al.
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The multidimensional mechanisms of long noncoding RNA function
Francesco P. Marchese et al.
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KLF1 drives the expression of fetal hemoglobin in British HPFH
Beeke Wienert et al.
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Pomalidomide reverses γ-globin silencing through the transcriptional reprogramming of adult hematopoietic progenitors
Brian M. Dulmovits et al.
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Customizing the genome as therapy for the β-hemoglobinopathies
Matthew C. Canver et al.
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Strict in vivo specificity of the Bcl11a erythroid enhancer
Elenoe C. Smith et al.
BLOOD (2016)
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Olivier Negre et al.
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Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype
Christian Brendel et al.
JOURNAL OF CLINICAL INVESTIGATION (2016)
Genetic disruption of the KLF1 gene to overexpress the -globin gene using the CRISPR/Cas9 system
Laleh Shariati et al.
JOURNAL OF GENE MEDICINE (2016)
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
Daniel P. Dever et al.
NATURE (2016)
Open-source guideseq software for analysis of GUIDE-seq data
Shengdar Q. Tsai et al.
NATURE BIOTECHNOLOGY (2016)
A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition
Elizabeth A. Traxler et al.
NATURE MEDICINE (2016)
Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia
Lin Ye et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)
Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin
Takeshi Masuda et al.
SCIENCE (2016)
2p15-p16.1 microdeletions encompassing and proximal to BCL11A are associated with elevated HbF in addition to neurologic impairment
Alister P. W. Funnell et al.
BLOOD (2015)
BCL11A deletions result in fetal hemoglobin persistence and neurodevelopmental alterations
Anindita Basak et al.
JOURNAL OF CLINICAL INVESTIGATION (2015)
BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
Matthew C. Canver et al.
NATURE (2015)
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
Shengdar Q. Tsai et al.
NATURE BIOTECHNOLOGY (2015)
Recent trends in the gene therapy of β-thalassemia
Alessia Finotti et al.
JOURNAL OF BLOOD MEDICINE (2015)
IthaGenes: An Interactive Database for Haemoglobin Variations and Epidemiology
Petros Kountouris et al.
PLOS ONE (2014)
STAR: ultrafast universal RNA-seq aligner
Alexander Dobin et al.
BIOINFORMATICS (2013)
Bcl11a Controls Flt3 Expression in Early Hematopoietic Progenitors and Is Required for pDC Development In Vivo
Xiaodi Wu et al.
PLOS ONE (2013)
The Switch from Fetal to Adult Hemoglobin
Vijay G. Sankaran et al.
COLD SPRING HARBOR PERSPECTIVES IN MEDICINE (2013)
The Molecular Basis of β-Thalassemia
Swee Lay Thein
COLD SPRING HARBOR PERSPECTIVES IN MEDICINE (2013)
Reawakening fetal hemoglobin: prospects for new therapies for the β-globin disorders
Daniel E. Bauer et al.
BLOOD (2012)
Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34+ cells after lentiviral vector-mediated gene transfer
Andrew Wilber et al.
BLOOD (2011)
Glanzmann thrombasthenia: a review of ITGA2B and ITGB3 defects with emphasis on variants, phenotypic variability, and mouse models
Alan T. Nurden et al.
BLOOD (2011)
Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities
Andrew Wilber et al.
BLOOD (2011)
The multifunctional role of EKLF/KLF1 during erythropoiesis
Miroslawa Siatecka et al.
BLOOD (2011)
Erythroid phenotypes associated with KLF1 mutations
Joseph Borg et al.
HAEMATOLOGICA-THE HEMATOLOGY JOURNAL (2011)
Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia
Keshet Ronen et al.
MOLECULAR THERAPY (2011)
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Lionel Arnaud et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2010)
BEDTools: a flexible suite of utilities for comparing genomic features
Aaron R. Quinlan et al.
BIOINFORMATICS (2010)
Advances in the understanding of haemoglobin switching
Vijay G. Sankaran et al.
BRITISH JOURNAL OF HAEMATOLOGY (2010)
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana-Calvo et al.
NATURE (2010)
KLF1 regulates BCL11A expression and γ- to β-globin gene switching
Dewang Zhou et al.
NATURE GENETICS (2010)
In vivo functions of the patched protein: Requirement of the C terminus for target gene inactivation but not hedgehog sequestration
RL Johnson et al.
MOLECULAR CELL (2000)
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