Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges
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Title
Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges
Authors
Keywords
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Journal
Molecular Diagnosis & Therapy
Volume -, Issue -, Pages -
Publisher
Springer Nature
Online
2019-01-31
DOI
10.1007/s40291-019-00383-4
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Note: Only part of the references are listed.- Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice
- (2018) Karine Sii-Felice et al. EXPERIMENTAL HEMATOLOGY
- Hematopoietic Stem Cell Transplantation in Thalassemia
- (2018) Luisa Strocchio et al. HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA
- p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells
- (2018) Robert J. Ihry et al. NATURE MEDICINE
- Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
- (2018) Alexis A. Thompson et al. NEW ENGLAND JOURNAL OF MEDICINE
- Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population
- (2018) Vijaya L. Simhadri et al. Molecular Therapy-Methods & Clinical Development
- Cas9 immunity creates challenges for CRISPR gene editing therapies
- (2018) Julie M. Crudele et al. Nature Communications
- Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major
- (2018) Nikoletta Psatha et al. Molecular Therapy-Methods & Clinical Development
- Multiple integrated non-clinical studies predict safety of lentiviral mediated gene therapy for beta thalassemia
- (2018) Maria Rosa Lidonnici et al. Molecular Therapy-Methods & Clinical Development
- Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
- (2018) Valentina Poletti et al. Molecular Therapy-Methods & Clinical Development
- Gene Therapy Approaches to Hemoglobinopathies
- (2017) Giuliana Ferrari et al. HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA
- Peptides derived from evolutionarily conserved domains in Beclin-1 and Beclin-2 enhance the entry of lentiviral vectors into human cells
- (2017) Saliha Majdoul et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
- (2017) Nicole M. Gaudelli et al. NATURE
- Gene Therapy in a Patient with Sickle Cell Disease
- (2017) Jean-Antoine Ribeil et al. NEW ENGLAND JOURNAL OF MEDICINE
- RNA editing with CRISPR-Cas13
- (2017) David B. T. Cox et al. SCIENCE
- Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
- (2017) Sachith Mettananda et al. Nature Communications
- A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease
- (2017) Liuhong Cai et al. Stem Cells Translational Medicine
- Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6
- (2017) Rasmus O Bak et al. eLife
- Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy
- (2017) Erika Zonari et al. Stem Cell Reports
- 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
- Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers
- (2016) L. Breda et al. BLOOD
- No evidence for cell activation or brain vaso-occlusion with plerixafor mobilization in sickle cell mice
- (2016) Erika Choi et al. BLOOD CELLS MOLECULES AND DISEASES
- Transplantation for thalassemia major
- (2016) Franco Locatelli et al. CURRENT OPINION IN HEMATOLOGY
- Optimizing autologous cell grafts to improve stem cell gene therapy
- (2016) Nikoletta Psatha et al. EXPERIMENTAL HEMATOLOGY
- Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene
- (2016) Olivier Negre et al. HUMAN GENE THERAPY
- Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects
- (2016) Jorge Mansilla-Soto et al. HUMAN GENE THERAPY
- Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype
- (2016) Christian Brendel et al. JOURNAL OF CLINICAL INVESTIGATION
- Genome-Wide Screening of Retroviral Envelope Genes in the Nine-Banded Armadillo (Dasypus novemcinctus, Xenarthra) Reveals an Unfixed Chimeric Endogenous Betaretrovirus Using the ASCT2 Receptor
- (2016) Sébastien Malicorne et al. JOURNAL OF VIROLOGY
- CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells
- (2016) Megan D Hoban et al. MOLECULAR THERAPY
- CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
- (2016) Daniel P. Dever et al. NATURE
- Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin
- (2016) Rahul Palchaudhuri 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
- Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
- (2016) M. A. DeWitt et al. Science Translational Medicine
- Bcl11a Deficiency Leads to Hematopoietic Stem Cell Defects with an Aging-like Phenotype
- (2016) Sidinh Luc et al. Cell Reports
- Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases
- (2016) Carmen F Bjurström et al. Molecular Therapy-Nucleic Acids
- Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells
- (2015) M. D. Hoban et al. BLOOD
- Plerixafor+G-CSF-mobilized CD34+ cells represent an optimal graft source for thalassemia gene therapy
- (2015) G. Karponi et al. BLOOD
- Potentially therapeutic levels of anti-sickling globin gene expression following lentivirus-mediated gene transfer in sickle cell disease bone marrow CD34 + cells
- (2015) Fabrizia Urbinati et al. EXPERIMENTAL HEMATOLOGY
- Molecular Determinants of Vectofusin-1 and Its Derivatives for the Enhancement of Lentivirally Mediated Gene Transfer into Hematopoietic Stem/Progenitor Cells
- (2015) Saliha Majdoul et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
- (2015) Jianbin Wang et al. NATURE BIOTECHNOLOGY
- Peripheral Blood Progenitor Cell Mobilization for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation
- (2014) Hien K. Duong et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene transfer into early-cytokine-stimulated and resting HSCs
- (2014) A. Girard-Gagnepain et al. BLOOD
- Reactivation of Developmentally Silenced Globin Genes by Forced Chromatin Looping
- (2014) Wulan Deng et al. CELL
- Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
- (2014) Olivier Negre et al. CURRENT GENE THERAPY
- Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel
- (2014) E. Angelucci et al. HAEMATOLOGICA
- Genome-wide analysis reveals characteristics of off-target sites bound by the Cas9 endonuclease
- (2014) Cem Kuscu et al. NATURE BIOTECHNOLOGY
- Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
- (2014) Pablo Tebas et al. NEW ENGLAND JOURNAL OF MEDICINE
- CRISPR/Cas9 systems have off-target activity with insertions or deletions between target DNA and guide RNA sequences
- (2014) Y. Lin et al. NUCLEIC ACIDS RESEARCH
- Engraftment and Lineage Potential of Adult Hematopoietic Stem and Progenitor Cells Is Compromised Following Short-Term Culture in the Presence of an Aryl Hydrocarbon Receptor Antagonist
- (2014) Angel Gu et al. Human Gene Therapy Methods
- Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery
- (2014) Steven Lin et al. eLife
- β-globin gene transfer to human bone marrow for sickle cell disease
- (2013) Zulema Romero et al. JOURNAL OF CLINICAL INVESTIGATION
- Infectivity enhancement of different HIV-1-based lentiviral pseudotypes in presence of the cationic amphipathic peptide LAH4-L1
- (2013) David Fenard et al. JOURNAL OF VIROLOGICAL METHODS
- Nuclease-mediated gene editing by homologous recombination of the human globin locus
- (2013) Richard A. Voit et al. NUCLEIC ACIDS RESEARCH
- CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity
- (2013) Thomas J. Cradick et al. NUCLEIC ACIDS RESEARCH
- LDL receptor and its family members serve as the cellular receptors for vesicular stomatitis virus
- (2013) D. Finkelshtein et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia
- (2013) Laura Breda et al. Expert Review of Hematology
- Concurrent Measures of Fusion and Transduction Efficiency of Primary CD34+ Cells with Human Immunodeficiency Virus 1-Based Lentiviral Vectors Reveal Different Effects of Transduction Enhancers
- (2013) Dina Ingrao et al. Human Gene Therapy Methods
- Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates
- (2012) Frédéric B Piel et al. LANCET
- Therapeutic Hemoglobin Levels after Gene Transfer in β-Thalassemia Mice and in Hematopoietic Cells of β-Thalassemia and Sickle Cells Disease Patients
- (2012) Laura Breda et al. PLoS One
- Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities
- (2011) A. Wilber 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
- 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
- Therapeutic levels of fetal hemoglobin in erythroid progeny of -thalassemic CD34+ cells after lentiviral vector-mediated gene transfer
- (2010) A. Wilber et al. BLOOD
- Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients
- (2010) Emanuela Anna Roselli et al. EMBO Molecular Medicine
- Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease
- (2010) M. Andreani et al. HAEMATOLOGICA
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
- (2010) Marina Cavazzana-Calvo et al. NATURE
- Haploinsufficiency for the erythroid transcription factor KLF1 causes hereditary persistence of fetal hemoglobin
- (2010) Joseph Borg et al. NATURE GENETICS
- A genome-wide association identified the common genetic variants influence disease severity in β0-thalassemia/hemoglobin E
- (2009) Manit Nuinoon et al. HUMAN GENETICS
- Cocal-pseudotyped Lentiviral Vectors Resist Inactivation by Human Serum and Efficiently Transduce Primate Hematopoietic Repopulating Cells
- (2009) Grant D Trobridge et al. MOLECULAR THERAPY
- Global epidemiology of haemoglobin disorders and derived service indicators
- (2008) Bernadette Modell BULLETIN OF THE WORLD HEALTH ORGANIZATION
- In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of -thalassemia
- (2008) A. Miccio et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of -thalassemia
- (2008) M. Uda et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific RepressorBCL11A
- (2008) Vijay G. Sankaran et al. SCIENCE
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