Genetic therapies for the first molecular disease

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

(2021) Adam C. Wilkinson et al.   Nature Communications

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

(2020) Julie Kanter et al.   BLOOD

Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency

(2020) Rajni Agarwal et al.   BLOOD

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

(2020) John F. Tisdale et al.   BLOOD

Pathologic angiogenesis in the bone marrow of humanized sickle cell mice is reversible by blood transfusion

(2020) Shin-Young Park et al.   BLOOD

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

(2020) Emmanuelle Six et al.   BLOOD

Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor

(2020) Naoya Uchida et al.   HAEMATOLOGICA

The promise and challenge of therapeutic genome editing

(2020) Jennifer A. Doudna   NATURE

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors

(2020) Jordan L. Doman et al.   NATURE BIOTECHNOLOGY

Continuous evolution of SpCas9 variants compatible with non-G PAMs

(2020) Shannon M. Miller et al.   NATURE BIOTECHNOLOGY

Expanding the genome-targeting scope and the site selectivity of high-precision base editors

(2020) Junjie Tan et al.   Nature Communications

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

(2020) Leslie Weber et al.   Science Advances

Development of gene editing strategies for human β-globin (HBB) gene mutations

(2020) Batuhan Mert Kalkan et al.   GENE

Optimizing lentiviral vector transduction of hematopoietic stem cells for gene therapy

(2020) Yoonjeong Jang et al.   GENE THERAPY

Cas9 activates the p53 pathway and selects for p53-inactivating mutations

(2020) Oana M. Enache et al.   NATURE GENETICS

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

A rationally engineered cytosine base editor retains high on-target activity while reducing both DNA and RNA off-target effects

(2020) Erwei Zuo et al.   NATURE METHODS

Timed inhibition of CDC7 increases CRISPR-Cas9 mediated templated repair

(2020) Beeke Wienert et al.   Nature Communications

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

(2020) Yi Yu et al.   Nature Communications

Direct quantification of single red blood cell hemoglobin concentration with multiphoton microscopy

(2020) Andrew T Francis et al.   ANALYTICAL CHEMISTRY

Motivations and Decision-Making of Adult Sickle Cell Patients in High-Risk Clinical Research

(2020) Hae Lin Cho et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Rationally Designed APOBEC3B Cytosine Base Editors with Improved Specificity

(2020) Shuai Jin et al.   MOLECULAR CELL

Current Status and Challenges of DNA Base Editing Tools

(2020) You Kyeong Jeong et al.   MOLECULAR THERAPY

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Characterization of red blood cell microcirculatory parameters using a bioimpedance microfluidic device

(2020) Tieying Xu et al.   Scientific Reports

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy

(2020) Christian Brendel et al.   Molecular Therapy-Methods & Clinical Development

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

(2020) Selami Demirci et al.   Molecular Therapy-Methods & Clinical Development

Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences

(2020) Richard A. Morgan et al.   Molecular Therapy-Methods & Clinical Development

Single C-to-T substitution using engineered APOBEC3G-nCas9 base editors with minimum genome- and transcriptome-wide off-target effects

(2020) Sangsin Lee et al.   Science Advances

Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing

(2020) Muhammad Naeem et al.   Cells

AcrIIA5 Suppresses Base Editors and Reduces Their Off-Target Effects

(2020) Mingming Liang et al.   Cells

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

(2020) Matthew M. Hsieh et al.   Blood Advances

Fetal Hemoglobin in Sickle Cell Anemia

(2020) Martin H Steinberg   BLOOD

Methods Favoring Homology-Directed Repair Choice in Response to CRISPR/Cas9 Induced-Double Strand Breaks

(2020) Han Yang et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Unbiased investigation of specificities of prime editing systems in human cells

(2020) Do Yon Kim et al.   NUCLEIC ACIDS RESEARCH

Prime editing for functional repair in patient-derived disease models

(2020) Imre F. Schene et al.   Nature Communications

CRISPR GUARD protects off-target sites from Cas9 nuclease activity using short guide RNAs

(2020) Matthew A. Coelho et al.   Nature Communications

Controlled Cycling and Quiescence Enables Efficient HDR in Engraftment-Enriched Adult Hematopoietic Stem and Progenitor Cells

(2020) Jiyung J. Shin et al.   Cell Reports

Allele-Specific Chromosome Removal after Cas9 Cleavage in Human Embryos

(2020) Michael V. Zuccaro et al.   CELL

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

(2020) Erica B. Esrick et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing

(2020) Josu de la Fuente et al.   Lancet Haematology

Genetic engineering of hematopoiesis: current stage of clinical translation and future perspectives

(2019) Luigi Naldini   EMBO Molecular Medicine

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

(2019) Sarah Marktel et al.   NATURE MEDICINE

Genome-wide profiling of adenine base editor specificity by EndoV-seq

(2019) Puping Liang et al.   Nature Communications

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

(2019) Agnieszka Czechowicz et al.   Nature Communications

Hematopoietic chimerism and donor-specific skin allograft tolerance after non-genotoxic CD117 antibody-drug-conjugate conditioning in MHC-mismatched allotransplantation

(2019) Zhanzhuo Li et al.   Nature Communications

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Genome-wide target specificity of CRISPR RNA-guided adenine base editors

(2019) Daesik Kim et al.   NATURE BIOTECHNOLOGY

Toxicity-Free Hematopoietic Stem Cell Engraftment Achieved with Anti-CD117 Monoclonal Antibody Conditioning

(2019) Rajni Agarwal et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Evaluating and Enhancing Target Specificity of Gene-Editing Nucleases and Deaminases

(2019) Daesik Kim et al.   Annual Review of Biochemistry

Aberrant Clonal Hematopoiesis following Lentiviral Vector Transduction of HSPCs in a Rhesus Macaque

(2019) Diego A. Espinoza et al.   MOLECULAR THERAPY

Editing the Sickle Cell Disease Mutation in Primary Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

(2019) Zulema Romero et al.   MOLECULAR THERAPY

Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors

(2019) Julian Grünewald et al.   NATURE

The next generation of CRISPR–Cas technologies and applications

(2019) Adrian Pickar-Oliver et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Effect of increased dose of total body irradiation on graft failure associated with HLA-haploidentical transplantation in patients with severe haemoglobinopathies: a prospective clinical trial

(2019) Javier Bolaños-Meade et al.   Lancet Haematology

Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis

(2019) Changyang Zhou et al.   NATURE

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

(2019) So Hyun Park et al.   NUCLEIC ACIDS RESEARCH

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

(2019) Olivier Humbert et al.   Science Translational Medicine

PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector

(2019) Katelyn E. Masiuk et al.   Molecular Therapy-Methods & Clinical Development

Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes

(2019) Stosh Ozog et al.   BLOOD

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

(2019) Elisa Magrin et al.   BLOOD

Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors

(2019) Chang Li et al.   MOLECULAR THERAPY

Title: Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements

(2019) Richard A. Morgan et al.   MOLECULAR THERAPY

In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

(2019) Sowmya Pattabhi et al.   Molecular Therapy-Nucleic Acids

Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study

(2019) Mary Eapen et al.   Lancet Haematology

Enhancing Lentiviral and Alpharetroviral Transduction of Human Hematopoietic Stem Cells for Clinical Application

(2019) Juliane W. Schott et al.   Molecular Therapy-Methods & Clinical Development

Antibody Therapies for Acute Myeloid Leukemia: Unconjugated, Toxin-Conjugated, Radio-Conjugated and Multivalent Formats

(2019) Brent A. Williams et al.   Journal of Clinical Medicine

An All Antibody Approach for Conditioning Bone Marrow for Hematopoietic Stem Cell Transplantation with Anti-cKIT and Anti-CD47 in Non-Human Primates

(2019) Kristopher D Marjon et al.   BLOOD

Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

(2019) Angela R. Smith et al.   BLOOD

Are genetic approaches still needed to cure sickle cell disease?

(2019) Robert A. Brodsky et al.   JOURNAL OF CLINICAL INVESTIGATION

Off-target effects of cytidine base editor and adenine base editor: what can we do?

(2019) Puping Liang et al.   Journal of Genetics and Genomics

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

High-throughput assessment of hemoglobin polymer in single red blood cells from sickle cell patients under controlled oxygen tension

(2019) Giuseppe Di Caprio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gates and NIH join forces on HIV and sickle cell diseases

(2019) Jon Cohen et al.   SCIENCE

Impact of Conditioning Intensity of Allogeneic Transplantation for Acute Myeloid Leukemia With Genomic Evidence of Residual Disease

(2019) Christopher S. Hourigan et al.   JOURNAL OF CLINICAL ONCOLOGY

Gene therapy for severe combined immunodeficiencies and beyond

(2019) Alain Fischer et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Single-cell modeling of routine clinical blood tests reveals transient dynamics of human response to blood loss

(2019) Anwesha Chaudhury et al.   eLife

The Role of the Health Care Provider in Building Trust Between Patients and Precision Medicine Research Programs

(2018) Anitra Persaud et al.   AMERICAN JOURNAL OF BIOETHICS

Beyond Consent: Building Trusting Relationships With Diverse Populations in Precision Medicine Research

(2018) Stephanie A. Kraft et al.   AMERICAN JOURNAL OF BIOETHICS

Experiences and Decision Making in Hematopoietic Stem Cell Transplant in Sickle Cell Disease: Patients' and Caregivers' Perspectives

(2018) Kirshma Khemani et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Variability of homozygous sickle cell disease: The role of alpha and beta globin chain variation and other factors

(2018) Graham R. Serjeant et al.   BLOOD CELLS MOLECULES AND DISEASES

UDiTaS™, a genome editing detection method for indels and genome rearrangements

(2018) Georgia Giannoukos et al.   BMC GENOMICS

Rapid Mobilization Reveals a Highly Engraftable Hematopoietic Stem Cell

(2018) Jonathan Hoggatt et al.   CELL

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

(2018) Nan Liu et al.   CELL

Safety and efficacy of plerixafor dose escalation for the mobilization of CD34 + hematopoietic progenitor cells in patients with sickle cell disease: interim results

(2018) Farid Boulad et al.   HAEMATOLOGICA

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

(2018) Chantal Lagresle-Peyrou et al.   HAEMATOLOGICA

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding

(2018) Gabriella E. Martyn et al.   NATURE GENETICS

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety and efficacy of plerixafor dose escalation for the mobilization of CD34 + hematopoietic progenitor cells in patients with sickle cell disease: interim results

(2018) Farid Boulad et al.   HAEMATOLOGICA

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

(2018) Chantal Lagresle-Peyrou et al.   HAEMATOLOGICA

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

(2018) Carsten T. Charlesworth et al.   Molecular Therapy-Nucleic Acids

Sickle cell disease

(2018) Gregory J. Kato et al.   Nature Reviews Disease Primers

Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans

(2018) Serena Scala et al.   NATURE MEDICINE

A limited number of double-strand DNA breaks is sufficient to delay cell cycle progression

(2018) Jeroen van den Berg et al.   NUCLEIC ACIDS RESEARCH

Emerging Genetic Therapy for Sickle Cell Disease

(2018) Stuart H. Orkin et al.   Annual Review of Medicine

Haploidentical Bone Marrow Transplantation with Post-Transplantation Cyclophosphamide Plus Thiotepa Improves Donor Engraftment in Patients with Sickle Cell Anemia: Results of an International Learning Collaborative

(2018) Josu de la Fuente et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

(2018) Hongjie Wang et al.   JOURNAL OF CLINICAL INVESTIGATION

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

(2018) Annalisa Lattanzi et al.   MOLECULAR THERAPY

Wake-up Sleepy Gene: Reactivating Fetal Globin for β-Hemoglobinopathies

(2018) Beeke Wienert et al.   TRENDS IN GENETICS

Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins

(2018) Philippe E. Mangeot et al.   Nature Communications

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 for blood diseases

(2018) Donald B Kohn   CURRENT OPINION IN BIOTECHNOLOGY

Mobilization of hematopoietic stem cells with the novel CXCR4 antagonist POL6326 (balixafortide) in healthy volunteers—results of a dose escalation trial

(2017) Darja Karpova et al.   Journal of Translational Medicine

Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells

(2017) Katelyn E. Masiuk et al.   MOLECULAR THERAPY

Genome-wide target specificities of CRISPR RNA-guided programmable deaminases

(2017) Daesik Kim et al.   NATURE BIOTECHNOLOGY

Gene Therapy in a Patient with Sickle Cell Disease

(2017) Jean-Antoine Ribeil et al.   NEW ENGLAND JOURNAL OF MEDICINE

Sickle Cell Disease

(2017) Frédéric B. Piel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients

(2017) Naoya Uchida et al.   Human Gene Therapy Clinical Development

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

Proponent or collaborative: Physician perspectives and approaches to disease modifying therapies in sickle cell disease

(2017) Nitya Bakshi et al.   PLoS One

Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease

(2016) Philipp M. Altrock et al.   AMERICAN JOURNAL OF HEMATOLOGY

Mistrust of Pediatric Sickle Cell Disease Clinical Trials Research

(2016) Evelyn M. Stevens et al.   AMERICAN JOURNAL OF PREVENTIVE MEDICINE

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

(2016) Eliane Gluckman et al.   BLOOD

Clinical and haematological risk factors for cerebral macrovasculopathy in a sickle cell disease newborn cohort: a prospective study

(2016) Julie Sommet et al.   BRITISH JOURNAL OF HAEMATOLOGY

New agents in HSC mobilization

(2016) Mélanie J. Domingues et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

(2016) Christian Brendel et al.   JOURNAL OF CLINICAL INVESTIGATION

Fetal haemoglobin in sickle-cell disease: from genetic epidemiology to new therapeutic strategies

(2016) Guillaume Lettre et al.   LANCET

Enhancer Regulation of Transcriptional Bursting Parameters Revealed by Forced Chromatin Looping

(2016) Caroline R. Bartman et al.   MOLECULAR CELL

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

Defining and improving the genome-wide specificities of CRISPR–Cas9 nucleases

(2016) Shengdar Q. Tsai et al.   NATURE REVIEWS GENETICS

Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin

(2016) T. Masuda et al.   SCIENCE

Hematopoietic stem cell transplantation in immunocompetent hosts without radiation or chemotherapy

(2016) Akanksha Chhabra et al.   Science Translational 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

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

(2015) M. D. Hoban et al.   BLOOD

Risk factor analysis of cerebral white matter hyperintensities in children with sickle cell disease

(2015) Veronica van der Land et al.   BRITISH JOURNAL OF HAEMATOLOGY

211Astatine-Conjugated Monoclonal CD45 Antibody-Based Nonmyeloablative Conditioning for Stem Cell Gene Therapy

(2015) Christopher R. Burtner et al.   HUMAN GENE THERAPY

miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction

(2015) Swaroopa Guda et al.   MOLECULAR THERAPY

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Radiolabeled Anti-CD45 Antibody with Reduced-Intensity Conditioning and Allogeneic Transplantation for Younger Patients with Advanced Acute Myeloid Leukemia or Myelodysplastic Syndrome

(2014) Raya Mawad et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Peculiar Elongated and Sickle-Shaped Red Blood Corpuscles in a Case of Severe Anemia

(2014) James B. Herrick   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Management of Sickle Cell Disease

(2014) Barbara P. Yawn et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Role of TP53 mutations in the origin and evolution of therapy-related acute myeloid leukaemia

(2014) Terrence N. Wong et al.   NATURE

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

(2013) M. H. Steinberg et al.   BLOOD

The High-Affinity CXCR4 Antagonist BKT140 Is Safe and Induces a Robust Mobilization of Human CD34+ Cells in Patients with Multiple Myeloma

(2013) A. Peled et al.   CLINICAL CANCER RESEARCH

Radioimmunotherapy for hematopoietic cell transplantation

(2013) Joseph G Jurcic   Immunotherapy

Genetic modifiers of sickle cell disease

(2012) Martin H. Steinberg et al.   AMERICAN JOURNAL OF HEMATOLOGY

Associated risk factors for silent cerebral infarcts in sickle cell anemia: low baseline hemoglobin, sex, and relative high systolic blood pressure

(2011) M. R. DeBaun et al.   BLOOD

Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin

(2011) Duyen A. Ngo et al.   BRITISH JOURNAL OF HAEMATOLOGY

Human bone marrow hematopoietic stem cells are increased in frequency and myeloid-biased with age

(2011) W. W. Pang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

THE RATE OF MUTATION OF HUMAN GENES

(2010) J. B. S. HALDANE   HEREDITAS

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Busulfan in Hematopoietic Stem Cell Transplantation

(2009) Stefan O. Ciurea et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Granulocyte colony-stimulating factor (G-CSF) administration in individuals with sickle cell disease: time for a moratorium?

(2009) Courtney D. Fitzhugh et al.   CYTOTHERAPY