
Professor In Residence
M_PEDS-HEMATOLOGY
The over-arching goal of my research career has been to develop and expand curative therapies for hemoglobin disorders in particular and non-malignant hematopoietic disorders more broadly. I have focused on genome editing approaches most recently, which rely upon modification of autologous hematopoietic cells to elicit a curative effect. In collaboration with colleagues at UC Berkeley, BCHO and UCLA, we are pursuing genomic editing of the sickle mutation in hematopoietic stem cells using the CRSPR/Cas9 ribonucleoprotein system. I have a long track record of experience in cellular therapies for pediatric acquired and hereditary conditions and in bone marrow transplantation.
Publications
Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease.
Blood advances
Incremental Eligibility Criteria for the BMT CTN 1507 Haploidentical Trial for Children with Sickle Cell Disease.
Blood advances
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.
Lancet (London, England)
Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.
Scientific reports
A Cure for Sickle Cell Disease.
Transplantation and cellular therapy
Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial.
Frontiers in neurology
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
The New England journal of medicine
Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.
American journal of hematology
Defining curative endpoints for transfusion-dependent β-thalassemia in the era of gene therapy and gene editing.
American journal of hematology
Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing.
American journal of hematology
Allogeneic haematopoietic stem-cell transplantation versus gene therapy for haemoglobinopathies.
The Lancet. Haematology
Access to CAR T-cell Clinical Trials in Underrepresented Populations: a Multicenter Cohort Study of Pediatric and Young Adult ALL Patients.
Transplantation and cellular therapy
Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials.
Transplantation and cellular therapy
Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease.
Journal of clinical oncology : official journal of the American Society of Clinical Oncology
Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.
Hematology/oncology clinics of North America
Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials.
Transplantation and cellular therapy
Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.
American journal of hematology
High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation.
iScience
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease.
The New England journal of medicine
Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.
The New England journal of medicine
American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.
Blood advances
BMT CTN State of the Science Symposium 2021: Looking Forward as the Network Celebrates its 20th Year.
Transplantation and cellular therapy
Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation.
Bone marrow transplantation
Induction of Fetal Hemoglobin by Gene Therapy.
The New England journal of medicine
Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi-center HGB-206 trial.
American journal of hematology
Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease.
Blood advances
End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.
Blood advances
Related and unrelated donor transplantation for ß-thalassemia major: results of an international survey.
Blood advances
Effect of donor type and conditioning regimen intensity on allogeneic transplantation outcomes in patients with sickle cell disease: a retrospective multicentre, cohort study.
The Lancet. Haematology
Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.
American journal of hematology
Promise of gene therapy to treat sickle cell disease.
Expert opinion on biological therapy
CIRM Alpha Stem Cell Clinics: Collaboratively Addressing Regenerative Medicine Challenges.
Cell stem cell
Gene Therapy in Patients with Transfusion-Dependent ß-Thalassemia.
The New England journal of medicine
Late Effects Screening Guidelines after Hematopoietic Cell Transplantation (HCT) for Hemoglobinopathy: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric HCT.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
The case for HLA-identical sibling hematopoietic stem cell transplantation in children with symptomatic sickle cell anemia.
Blood advances
Relationship between Mixed Donor-Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia.
Bone marrow transplantation
Current Results and Future Research Priorities in Late Effects after Hematopoietic Stem Cell Transplantation for Children with Sickle Cell Disease and Thalassemia: A Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium Inte
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.
Science translational medicine
The Second Pediatric Blood and Marrow Transplant Consortium International Consensus Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation: Defining the Unique Late Effects of Children Undergoing Hematopoietic Cell Transplantation f
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.
Blood
Ankyrin-B directs membrane tethering of periaxin and is required for maintenance of lens fiber cell hexagonal shape and mechanics.
American journal of physiology. Cell physiology
Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Update of hematopoietic cell transplantation for sickle cell disease.
Current opinion in hematology
In utero hematopoietic cell transplantation for hemoglobinopathies.
Frontiers in pharmacology
One-unit versus two-unit cord-blood transplantation for hematologic cancers.
The New England journal of medicine
Phenotyping: targeting genotype's rich cousin for diagnosis.
Journal of paediatrics and child health
Combined umbilical cord blood and bone marrow from HLA-identical sibling donors for hematopoietic stem cell transplantation in children with hemoglobinopathies.
Pediatric blood & cancer
Evaluation of the Spectra Optia apheresis system for mononuclear cell (MNC) collection in G-CSF mobilized and nonmobilized healthy donors: results of a multicenter study.
Journal of clinical apheresis
ß-globin gene transfer to human bone marrow for sickle cell disease.
The 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).
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Hematopoietic cell transplantation for nonmalignant disorders.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Improved engraftment with minimal graft-versus-host disease after major histocompatibility complex-mismatched cord blood transplantation with photochemically treated donor lymphocytes.
Experimental biology and medicine (Maywood, N.J.)
Umbilical cord blood transplantation for children with thalassemia and sickle cell disease.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Umbilical cord blood transplantation for thalassemia major.
Hematology/oncology clinics of North America
Gene therapy and bone marrow transplantation for thalassemia: changing of the guard?
Molecular therapy : the journal of the American Society of Gene Therapy
Stem-cell transplantation for sickle cell disease.
The New England journal of medicine
Transplant outcomes in bone marrow failure syndromes and hemoglobinopathies.
Seminars in Hematology
Pulmonary, gonadal, and central nervous system status after bone marrow transplantation for sickle cell disease.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Translational stability for sickle gene therapy.
Gene therapy
Human term placenta as a source of hematopoietic cells.
Experimental biology and medicine (Maywood, N.J.)
Risk factors affecting outcome of second HLA-matched sibling donor transplantations for graft failure in severe acquired aplastic anemia.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Newborn screening for hemoglobinopathies in California.
Pediatric blood & cancer
Recent advances in bone marrow transplantation in hemoglobinopathies.
Current molecular medicine
Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies.
Pediatric blood & cancer
Hematopoietic cell transplantation for thalassemia and sickle cell disease: past, present and future.
Bone marrow transplantation
Mixed haematopoietic chimerism for sickle cell disease prevents intravascular haemolysis.
British journal of haematology
Cord blood transplantation for sickle cell anemia: bust or boom?
Pediatric transplantation
Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research.
British journal of haematology
Effect of myeloablative bone marrow transplantation on growth in children with sickle cell anaemia: results of the multicenter study of haematopoietic cell transplantation for sickle cell anaemia.
British journal of haematology
Clinical hemoglobinopathies: iron, lungs and new blood.
Current opinion in hematology
The prevention and management of stroke in sickle cell anaemia.
Expert opinion on biological therapy
Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin.
Bone marrow transplantation
Outcomes of preimplantation genetic diagnosis therapy in treatment of beta-thalassemia: A retrospective analysis.
Annals of the New York Academy of Sciences
Sibling donor cord blood transplantation for thalassemia major: Experience of the Sibling Donor Cord Blood Program.
Annals of the New York Academy of Sciences
Stem cell therapy for sickle cell disease: transplantation and gene therapy.
Hematology. American Society of Hematology. Education Program
Stem cell transplantation with S-59 photochemically treated T-cell add-backs to establish allochimerism in murine thalassemia.
Annals of the New York Academy of Sciences
Treatment of hepatitis C virus infection in thalassemia.
Annals of the New York Academy of Sciences
Sickle cell anemia and hematopoietic cell transplantation: When is a pound of cure worth more than an ounce of prevention?
Pediatric transplantation
Haematopoietic cell transplantation in the treatment of sickle cell disease.
Expert opinion on biological therapy
Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease.
Blood
Novel therapeutic approaches in sickle cell disease.
Hematology. American Society of Hematology. Education Program
Bone marrow transplantation in sickle cell anemia.
Current opinion in oncology
Sibling donor cord blood banking for children with sickle cell disease.
Pediatric pathology & molecular medicine
Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Stem cell transplantation for sickle cell disease: can we reduce the toxicity?
Pediatric pathology & molecular medicine
Collection of sibling donor cord blood for children with thalassemia.
Journal of pediatric hematology/oncology
Bone Marrow Transplantation for Non-Malignant Disease.
Hematology. American Society of Hematology. Education Program
Bone marrow transplantation for sickle cell disease: where do we go from here?
Journal of pediatric hematology/oncology
The chicken beta-globin 5'HS4 boundary element blocks enhancer-mediated suppression of silencing.
Molecular and cellular biology
Current and future preparative regimens for bone marrow transplantation in thalassemia.
Annals of the New York Academy of Sciences
Unrelated and HLA-nonidentical related donor marrow transplantation for thalassemia and leukemia. A combined report from the Seattle Marrow Transplant Team and the International Bone Marrow Transplant Registry.
Annals of the New York Academy of Sciences
Collaborative multicenter investigation of marrow transplantation for sickle cell disease: current results and future directions.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Bone marrow transplantation for sickle cell disease.
The New England journal of medicine
Barriers to bone marrow transplantation for sickle cell anemia.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
Transcriptional enhancers act in cis to suppress position-effect variegation.
Genes & development
Enhancers increase the probability but not the level of gene expression.
Proceedings of the National Academy of Sciences of the United States of America
Bone marrow transplantation for thalassemia. The USA experience.
The American journal of pediatric hematology/oncology