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

Comparison of different methods for erythroid differentiation in the K562 cell line

(2016) Laleh Shariati et al.   BIOTECHNOLOGY LETTERS

Editing the genome to introduce a beneficial naturally occurring mutation associated with increased fetal globin

(2015) Beeke Wienert et al.   Nature Communications

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Three fingers on the switch

(2013) Michael R. Tallack et al.   CURRENT OPINION IN HEMATOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

In vivo cleavage of transgene donors promotes nuclease-mediated targeted integration

(2012) Sandra Cristea et al.   BIOTECHNOLOGY AND BIOENGINEERING

Genetic correction of β-thalassemia patient-specific iPS cells and its use in improving hemoglobin production in irradiated SCID mice

(2012) Yixuan Wang et al.   CELL RESEARCH

The HDAC inhibitor Givinostat modulates the hematopoietic transcription factors NFE2 and C-MYB in JAK2V617F myeloproliferative neoplasm cells

(2012) Ariel Amaru Calzada et al.   EXPERIMENTAL HEMATOLOGY

Obligate Ligation-Gated Recombination (ObLiGaRe): Custom-designed nuclease-mediated targeted integration through nonhomologous end joining

(2012) M. Maresca et al.   GENOME RESEARCH

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Transfusion-related biologic effects and free hemoglobin, heme, and iron

(2012) Caroline P. Ozment et al.   TRANSFUSION

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

Expression of miR-210 during erythroid differentiation and induction of γ-globin gene expression

(2011) Nicoletta Bianchi et al.   BMB Reports

MicroRNA-96 Directly Inhibits γ-Globin Expression in Human Erythropoiesis

(2011) Imane Azzouzi et al.   PLoS One

MicroRNA-15a and -16-1 act via MYB to elevate fetal hemoglobin expression in human trisomy 13

(2011) V. G. Sankaran et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transcriptional silencing of  -globin by BCL11A involves long-range interactions and cooperation with SOX6

(2010) J. Xu et al.   GENES & DEVELOPMENT

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Thalassemia: An Overview of 50 Years of Clinical Research

(2010) Vijay G. Sankaran et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Haploinsufficiency for the erythroid transcription factor KLF1 causes hereditary persistence of fetal hemoglobin

(2010) Joseph Borg et al.   NATURE GENETICS

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

(2010) Dewang Zhou et al.   NATURE GENETICS

Reversing the Hemoglobin Switch

(2010) Vijay G. Sankaran et al.   NEW ENGLAND JOURNAL OF MEDICINE

Amelioration of Sardinian  0 thalassemia by genetic modifiers

(2009) R. Galanello et al.   BLOOD

A genome-wide association identified the common genetic variants influence disease severity in β0-thalassemia/hemoglobin E

(2009) Manit Nuinoon et al.   HUMAN GENETICS

Developmental and species-divergent globin switching are driven by BCL11A

(2009) Vijay G. Sankaran et al.   NATURE

Recent Advances in Bone Marrow Transplantation in Hemoglobinopathies

(2008) Jennifer Michlitsch et al.   CURRENT MOLECULAR MEDICINE

Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific RepressorBCL11A

(2008) Vijay G. Sankaran et al.   SCIENCE