Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

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

Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206

(2019) Markus Y. Mapara et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Bone marrow characterization in sickle cell disease: inflammation and stress erythropoiesis lead to suboptimal CD34 recovery

(2019) Alexis Leonard et al.   BRITISH JOURNAL OF HAEMATOLOGY

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

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

Ethical Challenges in Hematopoietic Cell Transplantation for Sickle Cell Disease

(2018) Robert Sheppard Nickel et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

HLA Haploidentical Stem Cell Transplant with Pretransplant Immunosuppression for Patients with Sickle Cell Disease

(2018) Anna B. Pawlowska et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

(2018) Chiara Antoniani et al.   BLOOD

Haploidentical hematopoietic stem cell transplant for patients with sickle cell disease using thiotepa, fludarabine, thymoglobulin, low dose cyclophosphamide, 200 cGy tbi and post transplant cyclophosphamide

(2018) Haydar Frangoul et al.   BONE MARROW TRANSPLANTATION

The European Society for Blood and Marrow Transplantation (EBMT) Consensus Guidelines for the Detection and Treatment of Donor-specific Anti-HLA Antibodies (DSA) in Haploidentical Hematopoietic Cell Transplantation

(2018) Stefan O. Ciurea et al.   BONE MARROW TRANSPLANTATION

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

When there is no match, the game is not over: Alternative donor options for hematopoietic stem cell transplantation in sickle cell disease

(2018) Jacinth J. Joseph et al.   SEMINARS IN HEMATOLOGY

Genetic therapies for sickle cell disease

(2018) Erica B. Esrick et al.   SEMINARS IN HEMATOLOGY

Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced

(2018) Alexis Leonard et al.   Expert Review of Hematology

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

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

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

Gene therapy for blood diseases

(2018) Donald B Kohn   CURRENT OPINION IN BIOTECHNOLOGY

Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases

(2017) Staci D. Arnold et al.   HAEMATOLOGICA

Gene Therapy in a Patient with Sickle Cell Disease

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

Clinical risks and healthcare utilization of hematopoietic cell transplantation for sickle cell disease in the USA using merged databases

(2017) Staci D. Arnold et al.   HAEMATOLOGICA

Treosulfan-based conditioning regimen in sibling and alternative donor hematopoietic stem cell transplantation for children with sickle cell disease

(2017) Antonio Marzollo et al.   Mediterranean Journal of Hematology and Infectious Diseases

Mortality Rates and Age at Death from Sickle Cell Disease: U.S., 1979–2005

(2017) Sophie Lanzkron et al.   PUBLIC HEALTH REPORTS

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

(2016) Eliane Gluckman et al.   BLOOD

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

(2016) Shalini Shenoy et al.   BLOOD

Prognostic factors and outcomes for pediatric patients receiving an haploidentical relative allogeneic transplant using CD3/CD19-depleted grafts

(2016) M A Diaz et al.   BONE MARROW TRANSPLANTATION

Use of genome-editing tools to treat sickle cell disease

(2016) Ipek Tasan et al.   HUMAN GENETICS

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

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

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

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 Sickle Cell Disease Mortality Using a Population-Based Surveillance System, 2004 through 2008

(2016) Susan T. Paulukonis et al.   PUBLIC HEALTH REPORTS

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

(2016) M. A. DeWitt et al.   Science Translational Medicine

Assessing Costs, Benefits, and Risks in Chronic Disease: Taking the Long View

(2015) Carla Saenz et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

   T-cell reconstitution after HLA-haploidentical hematopoietic transplantation depleted of TCR-  +/CD19+ lymphocytes

(2015) I. Airoldi et al.   BLOOD

Rapid memory T-cell reconstitution recapitulating CD45RA-depleted haploidentical transplant graft content in patients with hematologic malignancies

(2015) B M Triplett et al.   BONE MARROW TRANSPLANTATION

Allogeneic donor availability for hematopoietic stem cell transplantation in children with sickle cell disease

(2015) David Justus et al.   PEDIATRIC BLOOD & CANCER

Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

(2015) Xiaosong Huang et al.   STEM CELLS

Nonmyeloablative HLA-Matched Sibling Allogeneic Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Phenotype

(2014) Matthew M. Hsieh et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg et al.   NATURE

HLA Match Likelihoods for Hematopoietic Stem-Cell Grafts in the U.S. Registry

(2014) Loren Gragert et al.   NEW ENGLAND JOURNAL OF MEDICINE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

(2014) C. J. Braun et al.   Science Translational Medicine

Long-Term Outcome and Evaluation of Organ Function in Pediatric Patients Undergoing Haploidentical and Matched Related Hematopoietic Cell Transplantation for Sickle Cell Disease

(2013) Mari H. Dallas et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

β-globin gene transfer to human bone marrow for sickle cell disease

(2013) Zulema Romero et al.   JOURNAL OF CLINICAL INVESTIGATION

Unrelated Donor Cord Blood Transplantation for Children with Severe Sickle Cell Disease: Results of One Cohort from the Phase II Study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN)

(2012) Naynesh R. Kamani et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Race and ethnicity in decisions about unrelated hematopoietic stem cell donation

(2012) G. E. Switzer et al.   BLOOD

HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease

(2012) J. Bolanos-Meade et al.   BLOOD

Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

(2012) F. Candotti et al.   BLOOD

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

Accelerated lymphocyte reconstitution and long-term recovery after transplantation of lentiviral-transduced rhesus CD34+ cells mobilized by G-CSF and plerixafor

(2011) Naoya Uchida et al.   EXPERIMENTAL HEMATOLOGY

The Use of Chromatin Insulators to Improve the Expression and Safety of Integrating Gene Transfer Vectors

(2011) David W. Emery   HUMAN GENE THERAPY

Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma

(2010) Manuel Grez et al.   MOLECULAR THERAPY

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

The cost of health care for patients with sickle cell disease

(2009) Samir K. Ballas   AMERICAN JOURNAL OF HEMATOLOGY

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

(2009) Courtney D. Fitzhugh et al.   CYTOTHERAPY

Cyclophosphamide and cancer: golden anniversary

(2009) Ashkan Emadi et al.   Nature Reviews Clinical Oncology

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Allogeneic Hematopoietic Stem-Cell Transplantation for Sickle Cell Disease

(2009) Matthew M. Hsieh et al.   NEW ENGLAND JOURNAL OF MEDICINE

High Risk of Graft Failure in Patients With Anti-HLA Antibodies Undergoing Haploidentical Stem-Cell Transplantation

(2009) Stefan O. Ciurea et al.   TRANSPLANTATION

HLA-Haploidentical Bone Marrow Transplantation for Hematologic Malignancies Using Nonmyeloablative Conditioning and High-Dose, Posttransplantation Cyclophosphamide

(2008) Leo Luznik et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Correction of Murine Sickle Cell Disease Using γ-Globin Lentiviral Vectors to Mediate High-level Expression of Fetal Hemoglobin

(2008) Tamara I Pestina et al.   MOLECULAR THERAPY