Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin

CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells

(2019) Jennifer E. Chung et al.   PLoS One

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

(2019) Olivier Humbert et al.   Science Translational Medicine

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

(2019) Elisa Magrin et al.   BLOOD

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

(2018) Chiara Antoniani et al.   BLOOD

Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch

(2018) Nan Liu et al.   CELL

Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding

(2018) Gabriella E. Martyn et al.   NATURE GENETICS

Epigenetic dysregulation of the erythropoietic transcription factor KLF1 and the β-like globin locus in juvenile myelomonocytic leukemia

(2017) Silvia Fluhr et al.   Epigenetics

BCL11A mRNA Targeting by miR-210: A Possible Network Regulating γ-Globin Gene Expression

(2017) Jessica Gasparello et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders

(2017) Neelam Lohani et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Lentiviral Transfer of γ-Globin with Fusion Gene NUP98-HOXA10HD Expands Hematopoietic Stem Cells and Ameliorates Murine β-Thalassemia

(2017) Hui Fen Zhao et al.   MOLECULAR THERAPY

Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells

(2017) Kai-Hsin Chang et al.   Molecular Therapy-Methods & Clinical Development

Customizing the genome as therapy for the β-hemoglobinopathies

(2016) Matthew C. Canver et al.   BLOOD

Strict in vivo specificity of the Bcl11a erythroid enhancer

(2016) E. C. Smith et al.   BLOOD

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene

(2016) Olivier Negre et al.   HUMAN GENE THERAPY

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

(2016) Christian Brendel et al.   JOURNAL OF CLINICAL INVESTIGATION

Genetic disruption of theKLF1gene to overexpress the γ-globin gene using the CRISPR/Cas9system

(2016) Laleh Shariati et al.   JOURNAL OF GENE MEDICINE

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

Open-source guideseq software for analysis of GUIDE-seq data

(2016) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

(2016) Elizabeth A Traxler et al.   NATURE MEDICINE

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia

(2016) Lin Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin

(2016) T. Masuda et al.   SCIENCE

2p15-p16.1 microdeletions encompassing and proximal to BCL11A are associated with elevated HbF in addition to neurologic impairment

(2015) A. P. W. Funnell et al.   BLOOD

Pomalidomide reverses  -globin silencing through the transcriptional reprogramming of adult hematopoietic progenitors

(2015) B. M. Dulmovits et al.   BLOOD

BCL11A deletions result in fetal hemoglobin persistence and neurodevelopmental alterations

(2015) Anindita Basak et al.   JOURNAL OF CLINICAL INVESTIGATION

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

IthaGenes: An Interactive Database for Haemoglobin Variations and Epidemiology

(2014) Petros Kountouris et al.   PLoS One

Bcl11a Controls Flt3 Expression in Early Hematopoietic Progenitors and Is Required for pDC Development In Vivo

(2013) Xiaodi Wu et al.   PLoS One

The Switch from Fetal to Adult Hemoglobin

(2013) V. G. Sankaran et al.   Cold Spring Harbor Perspectives in Medicine

The Molecular Basis of  -Thalassemia

(2013) S. L. Thein   Cold Spring Harbor Perspectives in Medicine

STAR: ultrafast universal RNA-seq aligner

(2012) Alexander Dobin et al.   BIOINFORMATICS

Reawakening fetal hemoglobin: prospects for new therapies for the  -globin disorders

(2012) D. E. Bauer et al.   BLOOD

Fetal hemoglobin in sickle cell anemia

(2011) I. Akinsheye et al.   BLOOD

Glanzmann thrombasthenia: a review of ITGA2B and ITGB3 defects with emphasis on variants, phenotypic variability, and mouse models

(2011) A. T. Nurden et al.   BLOOD

Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities

(2011) A. Wilber et al.   BLOOD

The multifunctional role of EKLF/KLF1 during erythropoiesis

(2011) M. Siatecka et al.   BLOOD

Erythroid phenotypes associated with KLF1 mutations

(2011) J. Borg et al.   HAEMATOLOGICA

Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia

(2011) Keshet Ronen et al.   MOLECULAR THERAPY

A Dominant Mutation in the Gene Encoding the Erythroid Transcription Factor KLF1 Causes a Congenital Dyserythropoietic Anemia

(2010) Lionel Arnaud et al.   AMERICAN JOURNAL OF HUMAN GENETICS

BEDTools: a flexible suite of utilities for comparing genomic features

(2010) Aaron R. Quinlan et al.   BIOINFORMATICS

Therapeutic levels of fetal hemoglobin in erythroid progeny of  -thalassemic CD34+ cells after lentiviral vector-mediated gene transfer

(2010) A. Wilber et al.   BLOOD

Advances in the understanding of haemoglobin switching

(2010) Vijay G. Sankaran et al.   BRITISH JOURNAL OF HAEMATOLOGY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

KLF1 regulates BCL11A expression and γ- to β-globin gene switching

(2010) Dewang Zhou et al.   NATURE GENETICS