Understanding α-globin gene regulation and implications for the treatment of β-thalassemia

 -Globin as a molecular target in the treatment of  -thalassemia

(2015) S. Mettananda et al.   BLOOD

Phase 1/2 trial of vorinostat in patients with sickle cell disease who have not benefitted from hydroxyurea

(2015) M. M. Okam et al.   BLOOD

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

The genome editing toolbox: a spectrum of approaches for targeted modification

(2014) Joseph K Cheng et al.   CURRENT OPINION IN BIOTECHNOLOGY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

Uncovering Enhancer Functions Using the α-Globin Locus

(2014) Douglas Vernimmen   PLoS Genetics

α-Globin Gene Quadruplication and Heterozygous β-Thalassemia: A Not So Rare Cause of Thalassemia Intermedia

(2013) Raffaella Origa et al.   ACTA HAEMATOLOGICA

Occupying Chromatin: Polycomb Mechanisms for Getting to Genomic Targets, Stopping Transcriptional Traffic, and Staying Put

(2013) Jeffrey A. Simon et al.   MOLECULAR CELL

Chromatin proteins and modifications as drug targets

(2013) Kristian Helin et al.   NATURE

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Ineffective Erythropoiesis inβ-Thalassemia

(2013) Jean-Antoine Ribeil et al.   TheScientificWorldJOURNAL

The Molecular Basis of  -Thalassemia

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

Alpha Thalassaemia and extended alpha globin genes in Sri Lanka

(2012) Sasikala Suresh et al.   BLOOD CELLS MOLECULES AND DISEASES

Chromatin loop formation in the β-globin locus and its role in globin gene transcription

(2012) AeRi Kim et al.   MOLECULES AND CELLS

Epigenetic Modifications and Chromosome Conformations of the Beta Globin Locus throughout Development

(2012) Kai-Hsin Chang et al.   Stem Cell Reviews and Reports

An interspecies analysis reveals a key role for unmethylated CpG dinucleotides in vertebrate Polycomb complex recruitment

(2011) Magnus D Lynch et al.   EMBO JOURNAL

Polycomb eviction as a new distant enhancer function

(2011) D. Vernimmen et al.   GENES & DEVELOPMENT

Variants in genetic modifiers of  -thalassemia can help to predict the major or intermedia type of the disease

(2011) C. Badens et al.   HAEMATOLOGICA

Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors

(2011) Shu-Yang Xie et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Generation of bivalent chromatin domains during cell fate decisions

(2011) Marco De Gobbi et al.   Epigenetics & Chromatin

Novel large deletions in the human α-globin gene cluster: Clarifying the HS-40 long-range regulatory role in the native chromosome environment

(2010) Andreia Coelho et al.   BLOOD CELLS MOLECULES AND DISEASES

The CRISPR/Cas bacterial immune system cleaves bacteriophage and plasmid DNA

(2010) Josiane E. Garneau et al.   NATURE

α-thalassaemia

(2010) Cornelis L Harteveld et al.   Orphanet Journal of Rare Diseases

Effect of α-gene numbers on phenotype of HbE/β thalassemia patients

(2009) Vineeta Sharma et al.   ANNALS OF HEMATOLOGY

Chromosome looping at the human  -globin locus is mediated via the major upstream regulatory element (HS -40)

(2009) D. Vernimmen et al.   BLOOD

Association of   globin gene quadruplication and heterozygous   thalassemia in patients with thalassemia intermedia

(2009) M. C. Sollaino et al.   HAEMATOLOGICA

The role of the polycomb complex in silencing  -globin gene expression in nonerythroid cells

(2008) D. Garrick et al.   BLOOD

Long-range regulation of ?? globin gene expression during erythropoiesis

(2008) Douglas R Higgs et al.   CURRENT OPINION IN HEMATOLOGY

siRNA-mediated reduction of  -globin results in phenotypic improvements in  -thalassemic cells

(2008) H. P. J. Voon et al.   HAEMATOLOGICA

Controlling  -globin: a review of  -globin expression and its impact on  -thalassemia

(2008) H. P. J. Voon et al.   HAEMATOLOGICA