All Stories

  1. Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
  2. Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment
  3. Intensive care risk and long-term outcomes in pediatric allogeneic hematopoietic cell transplant recipients
  4. Bone Marrow Failure Associated with Short Telomeres and Digenic Variants of Uncertain Significance in Telomere Biology Genes
  5. Mediport use as an acceptable standard for CAR T cell infusion
  6. Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells
  7. Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
  8. Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion
  9. Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector
  10. Comparison of Outcomes in Patients with Cerebral Adrenoleukodystrophy (CALD) Receiving Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy in Clinical Trials Versus Those Receiving Allogeneic Hematopoietic Stem Cell Transplant in a Contemporaneous ...
  11. Post-Transplantation Cyclophosphamide Is Associated with an Increase in Non-Cytomegalovirus Herpesvirus Infections in Patients with Acute Leukemia and Myelodysplastic Syndrome
  12. A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results
  13. Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency
  14. Impact of cytogenetic abnormalities on outcomes of adult Philadelphia-negative acute lymphoblastic leukemia after allogeneic hematopoietic stem cell transplantation: a study by the Acute Leukemia Working Committee of the Center for International Blood ...
  15. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
  16. Elivaldogene Autotemcel (eli-cel, Lenti-D) Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy: Updated Results from the Phase 2/3 ALD-102 Study and First Report on Safety Outcomes from the Phase 3 ALD-104 Study
  17. The Efficacy and Safety of Pharmacokinetics Guided Once-Daily Intravenous Busulfan in Pediatric Patients Receiving Hematopoietic Stem Cell Transplant
  18. A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1
  19. First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency
  20. AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation
  21. Pediatric hematopoietic cell transplantation: Longitudinal trends in body mass index and outcomes
  22. Acoustofluidic sonoporation for gene delivery to human hematopoietic stem and progenitor cells
  23. Lenti-D Hematopoietic Stem Cell Gene Therapy Stabilizes Neurologic Function in Boys with Cerebral Adrenoleukodystrophy
  24. Management Blueprint for Acquired Long QT Syndrome in Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation
  25. Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
  26. Successful engraftment of haploidentical bone marrow with post‐transplantation cyclophosphamide in patients with aplastic anemia
  27. Dissecting the Immune Cell Progeny of CAR-Expressing Hematopoietic Stem Cells in a Humanized Mouse Model
  28. Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort
  29. Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer
  30. Impact of cytogenetic abnormalities on outcomes of adult Philadelphia-negative acute lymphoblastic leukemia after allogeneic hematopoietic stem cell transplantation: a study by the Acute Leukemia Working Committee of the Center for International Blood ...
  31. A Cetuximab-Mediated Suicide System in Chimeric Antigen Receptor–Modified Hematopoietic Stem Cells for Cancer Therapy
  32. Updated Safety and Efficacy Outcomes from an Ongoing Phase 2/3 Trial of Lenti-D Autologous Hematopoietic Stem Cell Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy
  33. Viral Infection Rates and Associated Serious Complications in Pediatric Umbilical Cord Blood Transplant Patients over the Last Decade
  34. Engineering Precision Medicine to Increase Graft-Versus-Lymphoma Activity: Hematopoietic Stem Cells Modified with Chimeric Antigen Receptors
  35. Activity of Anti-CD19 Chimeric Antigen Receptor T Cells Against B Cell Lymphoma Is Enhanced by Antibody-Targeted Interferon-Alpha
  36. Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome
  37. Lenti-D Hematopoietic Stem Cell Gene Therapy to Arrest Progression of Cerebral Adrenoleukodystrophy: Interim Results of an International Phase 2/3 Trial
  38. Isolated Testicular Recurrence of AML in Patients With Chronic GVHD >1 Year Following Allogeneic Stem Cell Transplant
  39. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
  40. Umbilical Cord Blood Transplantation in Children with Acute Leukemia: Impact of Conditioning on Transplantation Outcomes
  41. Use of Rapamycin in a Patient With Juvenile Myelomonocytic Leukemia: A Case Report
  42. Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency
  43. Pre-clinical development of gene modification of haematopoietic stem cells with chimeric antigen receptors for cancer immunotherapy
  44. Anti-CD19 Chimeric Antigen Receptor in Hematopoietic Stem Cells Controlled By the Suicide Gene HSV-sr39TK for Immunotherapy of B-Lineage Malignancies
  45. Gene Modification of Human Hematopoietic Stem Cells with Suicide Genes As a Safety Control
  46. second HSCT for relapsed APML
  47. In Vitro Generation of Human NK Cells Expressing Chimeric Antigen Receptor Through Differentiation of Gene-Modified Hematopoietic Stem Cells
  48. False-positive magnetic resonance imaging findings in follow-up of pediatric patients with tumors of the central nervous system
  49. Pre-Clinical Development of Gene Modification of Hematopoietic Stem Cells with Chimeric Antigen Receptors for Cancer Immunotherapy
  50. Development of Optimized Protocol for Generation of NK Cells Expressing Chimeric Antigen Receptors from Hematopoietic Stem Cells for Cancer Immunotherapy
  51. 30. Phase II Clinical Trial of Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA-SCID) Using a γ-Retroviral Vector
  52. Autologous Transplant/Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
  53. Second-Generation Chimeric Antigen Receptors Successfully Modify Hematopoietic Stem Cells for Immunotherapy of B-Lineage Malignancies
  54. Systematic Comparison of the EF-1 Alpha Short (EFS) and Viral Promoters for Gene Modification of Human Primary Cells for Clinical Applications
  55. A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
  56. Abstract B70: Modification of hematopoietic stem cells with CD19-specific chimeric antigen receptors as a novel approach for cancer immunotherapy
  57. Gene Therapy: Charting a Future Course—Summary of a National Institutes of Health Workshop, April 12, 2013
  58. Human Lymphoid Development in the Absence of Common  -Chain Receptor Signaling
  59. Gene-modified hematopoietic stem cells for cancer immunotherapy
  60. Hematopoietic stem cells for cancer immunotherapy
  61. Anti-CD19 Chimeric Antigen Receptor Controlled By The Suicide Gene HSVsr39TK In Hematopoietic Stem Cells For Immunotherapy Of B-Lineage Malignancies
  62. A Pre-Clinical Model Of Hematopoietic Stem Cell Based Immunotherapy For Cancer Utilizing The NY-ESO-1 T-Cell Receptor and sr39TK PET Reporter / Suicide Gene
  63. Modification of Hematopoietic Stem/Progenitor Cells with CD19-Specific Chimeric Antigen Receptors as a Novel Approach for Cancer Immunotherapy
  64. β-globin gene transfer to human bone marrow for sickle cell disease
  65. Stem Cell Transplantation in Primary Myelofibrosis of Childhood
  66. Same Sibling Marrow Following Cord Allogeneic Transplantation as Therapy for Second Relapse APL: A Pediatric Case Report
  67. A CD19/Fc fusion protein for detection of anti-CD19 chimeric antigen receptors
  68. Gene Transfer to Hematopoietic Stem/Progenitor Cells As a Novel Approach for Immunotherapy Against B-Lineage Malignancies: In Vivo Xenograft Model,
  69. Prognostic significance ofWT1 gene expression in pediatric acute myeloid leukemia
  70. A novel translocation (x;4)(p22.1;q32) in a child with malignant Schwannoma
  71. Radioterapia em adenocarcinoma de pâncreas
  72. AVALIAÇÃO EPIDEMIOLÓGICA DA HANSENÍASE E DOS SERVIÇOS RESPONSÁVEIS POR SEU ATENDIMENTO EM RIBEIRÃO PRETO - SP NO ANO DE 1992