Recent trends for novel options in experimental biological therapy of β-thalassemia

Safe mobilization of CD34+ cells in adults with  -thalassemia and validation of effective globin gene transfer for clinical investigation

(2014) F. Boulad et al.   BLOOD

Mutations in Kruppel-like factor 1 cause transfusion-dependent hemolytic anemia and persistence of embryonic globin gene expression

(2014) V. Viprakasit et al.   BLOOD

Modulation of gamma globin genes expression by histone deacetylase inhibitors: anin vitrostudy

(2014) Luisa Ronzoni et al.   BRITISH JOURNAL OF HAEMATOLOGY

Gene Therapy for Hemoglobinopathies

(2014) Shanmuganathan Chandrakasan et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Targeted Fetal Hemoglobin Induction for Treatment of Beta Hemoglobinopathies

(2014) Susan P. Perrine et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

HBS1L-MYB intergenic variants modulate fetal hemoglobin via long-range MYB enhancers

(2014) Ralph Stadhouders et al.   JOURNAL OF CLINICAL INVESTIGATION

Engineered zinc-finger transcription factors activate OCT4 (POU5F1), SOX2, KLF4, c-MYC (MYC) and miR302/367

(2014) Qingzhou Ji et al.   NUCLEIC ACIDS RESEARCH

Dissecting the function of the adult β-globin downstream promoter region using an artificial zinc finger DNA-binding domain

(2014) Joeva J. Barrow et al.   NUCLEIC ACIDS RESEARCH

Direct activation of human and mouse Oct4 genes using engineered TALE and Cas9 transcription factors

(2014) Jiabiao Hu et al.   NUCLEIC ACIDS RESEARCH

A do-it-yourself protocol for simple transcription activator-like effector assembly

(2013) Claudia Uhde-Stone et al.   BIOLOGICAL PROCEDURES ONLINE

Seamless correction of the sickle cell disease mutation of theHBBgene in human induced pluripotent stem cells using TALENs

(2013) Ning Sun et al.   BIOTECHNOLOGY AND BIOENGINEERING

Mi2 -mediated silencing of the fetal  -globin gene in adult erythroid cells

(2013) M. Amaya et al.   BLOOD

LIN28B-mediated expression of fetal hemoglobin and production of fetal-like erythrocytes from adult human erythroblasts ex vivo

(2013) Y. T. Lee et al.   BLOOD

Enhanced erythroid cell differentiation in hypoxic condition is in part contributed by miR-210

(2013) Orawan Sarakul et al.   BLOOD CELLS MOLECULES AND DISEASES

UtroUp is a novel six zinc finger artificial transcription factor that recognises 18 base pairs of the utrophin promoter and efficiently drives utrophin upregulation

(2013) Annalisa Onori et al.   BMC MOLECULAR BIOLOGY

Quantification ofHBGmRNA in primary erythroid cultures: prediction of the response to hydroxyurea in sickle cell and beta-thalassemia

(2013) Alice Pecoraro et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

Transcriptional regulators Myb and BCL11A interplay with DNA methyltransferase 1 in developmental silencing of embryonic and fetal β-like globin genes

(2013) Mark Roosjen et al.   FASEB JOURNAL

Resveratrol accelerates erythroid maturation by activation of FoxO3 and ameliorates anemia in beta-thalassemic mice

(2013) S. S. Franco et al.   HAEMATOLOGICA

Transcription Activator-like Effector Nuclease (TALEN)-mediated Gene Correction in Integration-free β-Thalassemia Induced Pluripotent Stem Cells

(2013) Ning Ma et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Emodin can induce K562 cells to erythroid differentiation and improve the expression of globin genes

(2013) Yan-Ni Ma et al.   MOLECULAR AND CELLULAR BIOCHEMISTRY

A Feedback Loop Consisting of MicroRNA 23a/27a and the  -Like Globin Suppressors KLF3 and SP1 Regulates Globin Gene Expression

(2013) Y. Ma et al.   MOLECULAR AND CELLULAR BIOLOGY

Generation of an HIV Resistant T-cell Line by Targeted “Stacking” of Restriction Factors

(2013) Richard A Voit et al.   MOLECULAR THERAPY

Nuclease-mediated gene editing by homologous recombination of the human globin locus

(2013) Richard A. Voit et al.   NUCLEIC ACIDS RESEARCH

Updates of the HbVar database of human hemoglobin variants and thalassemia mutations

(2013) Belinda Giardine et al.   NUCLEIC ACIDS RESEARCH

MicroRNA-486-3p Regulates γ-Globin Expression in Human Erythroid Cells by Directly Modulating BCL11A

(2013) Valentina Lulli et al.   PLoS One

An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level

(2013) D. E. Bauer et al.   SCIENCE

Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient

(2013) Alisa Tubsuwan et al.   STEM CELLS

Gene therapy for hemoglobinopathies: progress and challenges

(2013) Alisa Dong et al.   Translational Research

Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia

(2013) Laura Breda et al.   Expert Review of Hematology

Management of the Thalassemias

(2013) N. F. Olivieri et al.   Cold Spring Harbor Perspectives in Medicine

A combined approach for β-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction

(2012) Cristina Zuccato et al.   ANNALS OF HEMATOLOGY

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

Primitive Erythropoiesis Is Regulated by miR-126 via Nonhematopoietic Vcam-1+ Cells

(2012) Christopher M. Sturgeon et al.   DEVELOPMENTAL CELL

Alternative options for DNA-based experimental therapy of β-thalassemia

(2012) Roberto Gambari   EXPERT OPINION ON BIOLOGICAL THERAPY

FLASH assembly of TALENs for high-throughput genome editing

(2012) Deepak Reyon et al.   NATURE BIOTECHNOLOGY

KLF10gene expression is associated with high fetal hemoglobin levels and with response to hydroxyurea treatment in β-hemoglobinopathy patients

(2012) Joseph Borg et al.   PHARMACOGENOMICS

Involvement of miRNA in erythroid differentiation

(2012) Nicoletta Bianchi et al.   Epigenomics

Correction of murine  -thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

(2011) O. Negre et al.   BLOOD

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

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

(2011) Nicoletta Bianchi et al.   BMB Reports

Modulation of the Biological Activity of microRNA-210 with Peptide Nucleic Acids (PNAs)

(2011) Enrica Fabbri et al.   ChemMedChem

Artificial Restriction DNA Cutters to Promote Homologous Recombination in Human Cells

(2011) Hitoshi Katada et al.   CURRENT GENE THERAPY

Thalassaemia

(2011) Douglas R Higgs et al.   LANCET

Nuclear Receptors TR2 and TR4 Recruit Multiple Epigenetic Transcriptional Corepressors That Associate Specifically with the Embryonic  -Type Globin Promoters in Differentiated Adult Erythroid Cells

(2011) S. Cui et al.   MOLECULAR AND CELLULAR BIOLOGY

Hematopoietic Stem Cell Mobilization for Gene Therapy of Adult Patients With Severe β-Thalassemia: Results of Clinical Trials Using G-CSF or Plerixafor in Splenectomized and Nonsplenectomized Subjects

(2011) Evangelia Yannaki et al.   MOLECULAR THERAPY

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Progress in Understanding the Hemoglobin Switch

(2011) Bernard G. Forget   NEW ENGLAND JOURNAL OF MEDICINE

A Functional Element Necessary for Fetal Hemoglobin Silencing

(2011) Vijay G. Sankaran et al.   NEW ENGLAND JOURNAL OF MEDICINE

A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity

(2011) Claudio Mussolino et al.   NUCLEIC ACIDS RESEARCH

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

(2011) Imane Azzouzi et al.   PLoS One

Forced TR2/TR4 expression in sickle cell disease mice confers enhanced fetal hemoglobin synthesis and alleviated disease phenotypes

(2011) A. D. Campbell et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

Targeted Therapeutic Strategies for Fetal Hemoglobin Induction

(2011) V. G. Sankaran   Hematology-American Society of Hematology Education Program

Expression of microRNA-451 in normal and thalassemic erythropoiesis

(2010) Saovaros Svasti et al.   ANNALS OF HEMATOLOGY

The challenge of obtaining therapeutic levels of genetically modified hematopoietic stem cells in β-thalassemia patients

(2010) Derek A. Persons   Annals of the New York Academy of Sciences

Transcriptional silencing of fetal hemoglobin by BCL11A

(2010) Vijay G. Sankaran et al.   Annals of the New York Academy of Sciences

A zinc-finger transcriptional activator designed to interact with the  -globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts

(2010) A. Wilber et al.   BLOOD

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

(2010) A. Wilber et al.   BLOOD

Gene therapy for β-thalassaemia: the continuing challenge

(2010) Evangelia Yannaki et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

Mechanism of human Hb switching: a possible role of the kit receptor/miR 221-222 complex

(2010) M. Gabbianelli et al.   HAEMATOLOGICA

Hemoglobin Gene Therapy for β-Thalassemia

(2010) Arthur Bank   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

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

The widespread regulation of microRNA biogenesis, function and decay

(2010) Jacek Krol et al.   NATURE REVIEWS GENETICS

Beta-thalassemia

(2010) Renzo Galanello et al.   Orphanet Journal of Rare Diseases

Global burden, distribution and prevention of β-thalassemias and hemoglobin E disorders

(2010) Roshan Colah et al.   Expert Review of Hematology

Discovering the genetics underlying foetal haemoglobin production in adults

(2009) Swee Lay Thein et al.   BRITISH JOURNAL OF HAEMATOLOGY

An Electronic Infrastructure for Research and Treatment of the Thalassemias and Other Hemoglobinopathies: The Euro-Mediterranean Ithanet Project

(2009) Carsten W. Lederer et al.   HEMOGLOBIN

Gene Therapy for Homozygous β -thalassemia. Is it a Reality?

(2009) Farid Boulad et al.   HEMOGLOBIN

Induction of endogenous γ-globin gene expression with decoy oligonucleotide targeting Oct-1 transcription factor consensus sequence

(2009) Xiaoxin S Xu et al.   Journal of Hematology & Oncology

Let-7 microRNAs are developmentally regulated in circulating human erythroid cells

(2009) Seung-Jae Noh et al.   Journal of Translational Medicine

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Therapeutic Applications of DNA and RNA Aptamers

(2009) Kristina W. Thiel et al.   OLIGONUCLEOTIDES

Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases

(2009) L. Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

 -Thalassemia Treatment Succeeds, With a Caveat

(2009) J. Kaiser   SCIENCE

Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies

(2008) Ugo Testa   ANNALS OF HEMATOLOGY

Mir-144 selectively regulates embryonic  -hemoglobin synthesis during primitive erythropoiesis

(2008) Y.-F. Fu et al.   BLOOD

The role of iron in patients after bone marrow transplantation

(2008) Theo de Witte   BLOOD REVIEWS

Recent Advances in Bone Marrow Transplantation in Hemoglobinopathies

(2008) Jennifer Michlitsch et al.   CURRENT MOLECULAR MEDICINE

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

MicroRNA-223 reversibly regulates erythroid and megakaryocytic differentiation of K562 cells

(2008) Jin-Yun Yuan et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

(2008) Elena E Perez et al.   NATURE BIOTECHNOLOGY

Update on Thalassemia: Clinical Care and Complications

(2008) Melody J. Cunningham   PEDIATRIC CLINICS OF NORTH AMERICA

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

(2008) Vijay G. Sankaran et al.   SCIENCE

Fetal Hemoglobin Inducers from the Natural World: A Novel Approach for Identification of Drugs for the Treatment of β-Thalassemia and Sickle-Cell Anemia

(2007) Nicoletta Bianchi et al.   Evidence-based Complementary and Alternative Medicine