Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective

Pharmacologic induction of PGC ‐1α stimulates fetal haemoglobin gene expression

(2022) Yanan Sun et al.   BRITISH JOURNAL OF HAEMATOLOGY

Investigating the Efficacy and Safety of Thalidomide for Treating Patients With ß-Thalassemia: A Meta-Analysis

(2022) Yanfei Lu et al.   Frontiers in Pharmacology

Salubrinal induces fetal hemoglobin expression via the stress-signaling pathway in human sickle erythroid progenitors and sickle cell disease mice

(2022) Nicole H. Lopez et al.   PLoS One

Induction of Fetal Hemoglobin by Gene Therapy

(2021) Mark C. Walters   NEW ENGLAND JOURNAL OF MEDICINE

Delayed-Release Dimethyl Fumarate Safety and Efficacy in Pediatric Patients With Relapsing-Remitting Multiple Sclerosis

(2021) Raed Alroughani et al.   Frontiers in Neurology

Benserazide racemate and enantiomers induce fetal globin gene expression in vivo: Studies to guide clinical development for beta thalassemia and sickle cell disease

(2021) Betty S. Pace et al.   BLOOD CELLS MOLECULES AND DISEASES

β-Thalassemias

(2021) Ali T. Taher et al.   NEW ENGLAND JOURNAL OF MEDICINE

A comprehensive review of hydroxyurea for β-haemoglobinopathies: the role revisited during COVID-19 pandemic

(2021) Nirmani Yasara et al.   Orphanet Journal of Rare Diseases

Acyclovir induces fetal hemoglobin via down regulation of γ-globin repressors, BCL11A and SOX6 trans-acting factors

(2021) Hamad Ali et al.   BIOCHEMICAL PHARMACOLOGY

Comparison of efficacy and safety of thalidomide vs hydroxyurea in patients with Hb E-β thalassemia - a pilot study from a tertiary care Centre of India

(2021) Manisha Jain et al.   BLOOD CELLS MOLECULES AND DISEASES

Moving somatic gene editing to the clinic: routes to market access and reimbursement in Europe

(2021) Tessel Rigter et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Synthesis and pharmacological evaluation of pomalidomide derivatives useful for sickle cell disease treatment

(2021) Thais Regina Ferreira de Melo et al.   BIOORGANIC CHEMISTRY

Efficacy and Safety of Thalidomide in Patients With Transfusion-Dependent Thalassemia

(2021) Jagdish Chandra et al.   INDIAN PEDIATRICS

Efficacy of Thalidomide Treatment in Children With Transfusion Dependent β-Thalassemia: A Retrospective Clinical Study

(2021) Xinyu Li et al.   Frontiers in Pharmacology

Cilostazol-mediated reversion of γ-globin silencing is associated with a high level of HbF production: A potential therapeutic candidate for β-globin disorders

(2021) Hamad Ali et al.   BIOMEDICINE & PHARMACOTHERAPY

Ftx-6058 Induces Fetal Hemoglobin Production and Ameliorates Disease Pathology in Sickle Cell Mice

(2021) David Matson et al.   BLOOD

Treatment with IMR-687, a Highly Selective PDE9 Inhibitor, Increases HbF and Reduces VOCs in Adults with Sickle Cell Disease in a Long-Term, Phase 2a, Open-Label Extension Study

(2021) Biree Andemariam et al.   BLOOD

Revisiting fetal hemoglobin inducers in beta-hemoglobinopathies: a review of natural products, conventional and combinatorial therapies

(2021) Mandrita Mukherjee et al.   MOLECULAR BIOLOGY REPORTS

Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations

(2021) Clare Samuelson et al.   Molecular Therapy-Methods & Clinical Development

Novel histone deacetylase inhibitor CT-101 induces γ-globin gene expression in sickle erythroid progenitors with targeted epigenetic effects

(2021) Louis H. Junker et al.   BLOOD CELLS MOLECULES AND DISEASES

Thalidomide in Patients with Transfusion-Dependent E-Beta Thalassemia Refractory to Hydroxyurea: A Single-Center Experience

(2020) Arijit Nag et al.   INDIAN JOURNAL OF HEMATOLOGY AND BLOOD TRANSFUSION

High‐level induction of fetal haemoglobin by pomalidomide in β‐thalassaemia/HbE erythroid progenitor cells

(2020) Pinyaphat Khamphikham et al.   BRITISH JOURNAL OF HAEMATOLOGY

THALIDOMIDE FOR PATIENTS WITH THALASSEMIA INTERMEDIA: A RETROSPECTIVE MULTICENTER CLINICAL STUDY

(2020) Kun Yang et al.   Mediterranean Journal of Hematology and Infectious Diseases

Fetal hemoglobin quantification per red blood cell allows to determine protective thresholds in sickle cell disease

(2020) Nicolas Hebert et al.   AMERICAN JOURNAL OF HEMATOLOGY

UNC0638 induces high levels of fetal hemoglobin expression in β-thalassemia/HbE erythroid progenitor cells

(2020) Tiwaporn Nualkaew et al.   ANNALS OF HEMATOLOGY

In vitro and in vivo induction of fetal hemoglobin with a reversible and selective DNMT1 inhibitor

(2020) Aidan G. Gilmartin et al.   HAEMATOLOGICA

Thalidomide for the Treatment of Thrombocytopenia and Hypersplenism in Patients With Cirrhosis or Thalassemia

(2020) Yaocheng Chen et al.   Frontiers in Pharmacology

Fetal Hemoglobin in Sickle Cell Anemia

(2020) Martin H Steinberg   BLOOD

Changing the Clinical Paradigm of Hydroxyurea Treatment for Sickle Cell Anemia Through Precision Medicine

(2020) Min Dong et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Potent and orally active purine-based fetal hemoglobin inducers for treating β-thalassemia and sickle cell disease

(2020) Zheng-Sheng Lai et al.   EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Tenofovir disoproxil fumarate induces fetal hemoglobin production in K562 cells and β-YAC transgenic mice: A therapeutic approach for γ-globin induction

(2020) Faisal Khan et al.   EXPERIMENTAL CELL RESEARCH

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Fetal Hemoglobin in Sickle Hemoglobinopathies: High HbF Genotypes and Phenotypes

(2020) Martin H. Steinberg   Journal of Clinical Medicine

Benserazide as a potential novel fetal hemoglobin inducer: an observational study in non-carriers of hemoglobin disorders

(2020) Marina Erê Hummel Pimenta Santos et al.   BLOOD CELLS MOLECULES AND DISEASES

NEW THERAPEUTIC OPTIONS FOR THE TREATMENT OF SICKLE CELL DISEASE

(2019) Alessandro Matte et al.   Mediterranean Journal of Hematology and Infectious Diseases

Salubrinal, a novel inhibitor of eIF‐2α dephosphorylation, promotes erythropoiesis at early stage targeted by ufmylation pathway

(2019) Fanghui Chen et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Robust Clinical and Laboratory Response to Hydroxyurea Using Pharmacokinetically Guided Dosing for Young Children with Sickle Cell Anemia

(2019) Patrick T. McGann et al.   AMERICAN JOURNAL OF HEMATOLOGY

A novel, highly potent and selective phosphodiesterase-9 inhibitor for the treatment of sickle cell disease

(2019) James G. McArthur et al.   HAEMATOLOGICA

Prospective longitudinal follow‐up of children with sickle cell disease treated with hydroxyurea since infancy

(2019) Ronay Thomas et al.   PEDIATRIC BLOOD & CANCER

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

(2019) Elisa Magrin et al.   BLOOD

Promising Response to Thalidomide in Symptomatic β-Thalassemia

(2019) Ahmed K. Yassin   INDIAN JOURNAL OF HEMATOLOGY AND BLOOD TRANSFUSION

Synergistic silencing of α-globin and induction of γ-globin by histone deacetylase inhibitor, vorinostat as a potential therapy for β-thalassaemia

(2019) Sachith Mettananda et al.   Scientific Reports

Emerging drugs in randomized controlled trials for sickle cell disease: are we on the brink of a new era in research and treatment?

(2019) Alessandro Matte et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Clinical trial on the effects of thalidomide on hemoglobin synthesis in patients with moderate thalassemia intermedia

(2018) Quan Ren et al.   ANNALS OF HEMATOLOGY

Metformin induces FOXO3-dependent fetal hemoglobin production in human primary erythroid cells

(2018) Yankai Zhang et al.   BLOOD

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

(2018) Nan Liu et al.   CELL

Chronic Administration of Hydroxyurea (HU) Benefits Caucasian Patients with Sickle-Beta Thalassemia

(2018) Rosario Di Maggio et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Thalassaemia

(2018) Ali T Taher et al.   LANCET

Fetal Hemoglobin Induction by Epigenetic Drugs

(2018) Donald Lavelle et al.   SEMINARS IN HEMATOLOGY

Use of metformin in patients with sickle cell disease

(2018) Jin Han et al.   AMERICAN JOURNAL OF HEMATOLOGY

Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?

(2018) Martina C. Cornel et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Thalidomide induces haematologic responses in patients with β-thalassaemia

(2017) Jiangming Chen et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

Thalidomide has a significant effect in patients with thalassemia intermedia

(2017) YunShuan Li et al.   Hematology

Histone Deacetylase Inhibitors as Anticancer Drugs

(2017) Tomas Eckschlager et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Sickle cell disease

(2017) Russell E Ware et al.   LANCET

Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study

(2017) Robert Molokie et al.   PLOS MEDICINE

Effects of hydroxyurea on F-cells in sickle cell disease and potential impact of a second fetal globin inducer

(2016) Yan Dai et al.   AMERICAN JOURNAL OF HEMATOLOGY

Effects of hydroxyurea treatment for patients with hemoglobin SC disease

(2016) Lori Luchtman-Jones et al.   AMERICAN JOURNAL OF HEMATOLOGY

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers

(2016) L. Breda et al.   BLOOD

Therapeutic fetal-globin inducers reduce transcriptional repression in hemoglobinopathy erythroid progenitors through distinct mechanisms

(2016) Yan Dai et al.   BLOOD CELLS MOLECULES AND DISEASES

Decreased fetal hemoglobin over time among youth with sickle cell disease on hydroxyurea is associated with higher urgent hospital use

(2016) Nancy S. Green et al.   PEDIATRIC BLOOD & CANCER

The Interplay Between Peroxiredoxin-2 and Nuclear Factor-Erythroid 2 Is Important in Limiting Oxidative Mediated Dysfunction in β-Thalassemic Erythropoiesis

(2015) Alessandro Matte et al.   ANTIOXIDANTS & REDOX SIGNALING

The LSD1 inhibitor RN-1 induces fetal hemoglobin synthesis and reduces disease pathology in sickle cell mice

(2015) S. Cui et al.   BLOOD

Inhibition of G9a methyltransferase stimulates fetal hemoglobin production by facilitating LCR/ -globin looping

(2015) I. Krivega et al.   BLOOD

EHMT1 and EHMT2 inhibition induces fetal hemoglobin expression

(2015) A. Renneville et al.   BLOOD

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

(2015) B. M. Dulmovits et al.   BLOOD

RN-1, a potent and selective lysine-specific demethylase 1 inhibitor, increases γ-globin expression, F reticulocytes, and F cells in a sickle cell disease mouse model

(2015) Angela Rivers et al.   EXPERIMENTAL HEMATOLOGY

Anemia: progress in molecular mechanisms and therapies

(2015) Vijay G Sankaran et al.   NATURE MEDICINE

Amelioration of inflammation and tissue damage in sickle cell model mice by Nrf2 activation

(2015) Nadine Keleku-Lukwete et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Novel Inducers of Fetal Globin Identified through High Throughput Screening (HTS) Are Active In Vivo in Anemic Baboons and Transgenic Mice

(2015) Michael S. Boosalis et al.   PLoS One

"Packaging" of fetal hemoglobin in sickle cell anemia

(2014) G. R. Buchanan   BLOOD

Hydroxyurea-inducible SAR1 gene acts through the Gi /JNK/Jun pathway to regulate  -globin expression

(2014) J. Zhu et al.   BLOOD

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

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

Reactivation of Developmentally Silenced Globin Genes by Forced Chromatin Looping

(2014) Wulan Deng et al.   CELL

Reprogramming ovarian and breast cancer cells into non-cancerous cells by low-dose metformin or SN-38 through FOXO3 activation

(2014) Theodore Hu et al.   Scientific Reports

Efficacy of delayed-release dimethyl fumarate in relapsing-remitting multiple sclerosis: integrated analysis of the phase 3 trials

(2014) Vissia Viglietta et al.   Annals of Clinical and Translational Neurology

Stressing HbF synthesis: role of translation?

(2013) J.-J. Chen et al.   BLOOD

Eukaryotic initiation factor 2  phosphorylation mediates fetal hemoglobin induction through a post-transcriptional mechanism

(2013) C. K. Hahn et al.   BLOOD

Fetal hemoglobin in sickle cell anemia: a glass half full?

(2013) M. H. Steinberg et al.   BLOOD

Clinical experience with fetal hemoglobin induction therapy in patients with  -thalassemia

(2013) K. M. Musallam et al.   BLOOD

Activation of AMPK inhibits cervical cancer cell growth through AKT/FOXO3a/FOXM1 signaling cascade

(2013) Mingo Ming Ho Yung et al.   BMC CANCER

Interpreting elevated fetal hemoglobin in pathology and health at the basic laboratory level: new and known γ- gene mutations associated with hereditary persistence of fetal hemoglobin

(2013) A. Amato et al.   International Journal of Laboratory Hematology

Metformin Suppresses Expression of the Selenoprotein P Gene via an AMP-activated Kinase (AMPK)/FoxO3a Pathway in H4IIEC3 Hepatocytes

(2013) Hiroaki Takayama et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Discovery of an in Vivo Chemical Probe of the Lysine Methyltransferases G9a and GLP

(2013) Feng Liu et al.   JOURNAL OF MEDICINAL CHEMISTRY

Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin induction

(2013) Lihong Shi et al.   NATURE MEDICINE

Oxidative Stress andβ-Thalassemic Erythroid Cells behind the Molecular Defect

(2013) Lucia De Franceschi et al.   Oxidative Medicine and Cellular Longevity

Corepressor-dependent silencing of fetal hemoglobin expression by BCL11A

(2013) J. Xu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The Switch from Fetal to Adult Hemoglobin

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

G9a/GLP-dependent histone H3K9me2 patterning during human hematopoietic stem cell lineage commitment

(2012) X. Chen et al.   GENES & DEVELOPMENT

Brain-Penetrant LSD1 Inhibitors Can Block Memory Consolidation

(2011) Ramesh Neelamegam et al.   ACS Chemical Neuroscience

Pomalidomide augments fetal hemoglobin production without the myelosuppressive effects of hydroxyurea in transgenic sickle cell mice

(2011) S. E. Meiler et al.   BLOOD

A pilot study of subcutaneous decitabine in  -thalassemia intermedia

(2011) N. F. Olivieri et al.   BLOOD

Identification and Characterization of Small-Molecule Inducers of Fetal Hemoglobin

(2011) Tae-gyu Nam et al.   ChemMedChem

Pregnancy outcomes in women with elevated levels of fetal hemoglobin

(2011) Ally Murji et al.   Journal of Maternal-Fetal & Neonatal Medicine

Thrombosis and Sickle Cell Disease

(2011) Lucia Franceschi et al.   SEMINARS IN THROMBOSIS AND HEMOSTASIS

The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up

(2010) Martin H. Steinberg et al.   AMERICAN JOURNAL OF HEMATOLOGY

The HDAC inhibitors trichostatin A and suberoylanilide hydroxamic acid exhibit multiple modalities of benefit for the vascular pathobiology of sickle transgenic mice

(2010) R. P. Hebbel et al.   BLOOD

Desensitization to hydroxycarbamide following long-term treatment of thalassaemia intermedia as observed in vivo and in primary erythroid cultures from treated patients

(2010) Paolo Rigano et al.   BRITISH JOURNAL OF HAEMATOLOGY

Early Detection of Response to Hydroxyurea Therapy in Patients with Sickle Cell Anemia

(2010) Samir K. Ballas et al.   HEMOGLOBIN

Chemical genetic strategy identifies histone deacetylase 1 (HDAC1) and HDAC2 as therapeutic targets in sickle cell disease

(2010) James E. Bradner et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The effect of prolonged administration of hydroxyurea on morbidity and mortality in adult patients with sickle cell syndromes: results of a 17-year, single-center trial (LaSHS)

(2009) E. Voskaridou et al.   BLOOD

Response to hydroxyurea in β thalassemia major and intermedia: Experience in western India

(2009) Khushnooma Y. Italia et al.   CLINICA CHIMICA ACTA

Hydroxycarbamide-induced changes in E/beta thalassemia red blood cells

(2008) Sylvia T. Singer et al.   AMERICAN JOURNAL OF HEMATOLOGY

Thalidomide therapy in a patient with thalassemia major

(2008) Lilia Beatriz Aguilar-Lopez et al.   BLOOD CELLS MOLECULES AND DISEASES

Clinical effectiveness of decitabine in severe sickle cell disease

(2008) Yogen Saunthararajah et al.   BRITISH JOURNAL OF HAEMATOLOGY

Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human CD34+ cells

(2008) Laure A. Moutouh-de Parseval et al.   JOURNAL OF CLINICAL INVESTIGATION

Hydroxyurea for the Treatment of Sickle Cell Anemia

(2008) Orah S. Platt   NEW ENGLAND JOURNAL OF MEDICINE