Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

Cited by (272)

Items 101–200 of 272

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9. The Committee for Advanced Therapies' of the European Medicines Agency Reflection Paper on Management of Clinical Risks Deriving from Insertional Mutagenesis
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10. Solving the Problem of γ-Retroviral Vectors Containing Long Terminal Repeats
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11. Alpharetroviral Vectors: From a Cancer-Causing Agent to a Useful Tool for Human Gene Therapy
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12. Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection
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13. Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside
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14. Sense-and-Respond Payload Delivery Using a Novel Antigen-Inducible Promoter Improves Suboptimal CAR-T Activation
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15. Synthetic Biology—Toward Therapeutic Solutions
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16. Cell and Gene Therapy for Friedreich Ataxia: Progress to Date
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17. Promoter Choice for Retroviral Vectors: Transcriptional Strength Versus Trans-Activation Potential
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18. Alpharetroviral Vector-mediated Gene Therapy for X-CGD: Functional Correction and Lack of Aberrant Splicing
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19. CTF/NF1 transcription factors act as potent genetic insulators for integrating gene transfer vectors
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20. Lentiviral Vectors: A Versatile Tool to Fight Cancer
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21. Stem Cell Marking With Promotor-deprived Self-inactivating Retroviral Vectors Does Not Lead to Induced Clonal Imbalance
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22. Self-inactivating retroviral vector-mediated gene transfer induces oncogene activation and immortalization of primary murine bone marrow cells.
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23. The genomic risk of somatic gene therapy
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24. Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy
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25. Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia
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26. Correction of murine hemophilia A following nonmyeloablative transplantation of hematopoietic stem cells engineered to encode an enhanced human factor VIII variant using a safety-augmented retroviral vector
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27. A Self-inactivating γ-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice
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28. The transformative potential of HSC gene therapy as a genetic medicine
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29. The long and winding road that leads to a cure for epidermolysis bullosa.
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30. Human miR223 Promoter as a Novel Myelo-Specific Promoter for Chronic Granulomatous Disease Gene Therapy
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31. Gene Therapy of Mpl Deficiency: Challenging Balance Between Leukemia and Pancytopenia
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32. Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia
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33. Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes
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34. Progress and prospects: Foamy virus vectors enter a new age
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35. Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy
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36. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease
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37. Lentiviral vectors incorporating a human elongation factor 1α promoter for the treatment of canine leukocyte adhesion deficiency
    Everette Jacob Remington Nelson, Laura M. Tuschong, M.J. Hunter, Thomas R. Bauer, Tanya H. Burkholder, Dennis D. Hickstein · 2010 · Gene Therapy
38. Towards a Safer, More Randomized Lentiviral Vector Integration Profile Exploring Artificial LEDGF Chimeras
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39. Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential
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40. Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy
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41. Use of human MAR elements to improve retroviral vector production
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42. Epidermal growth factor improves lentivirus vector gene transfer into primary mouse hepatocytes
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43. Treatment of canine leukocyte adhesion deficiency by foamy virus vectors expressing CD18 from a PGK promoter
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44. Gene Therapy for Canine Leukocyte Adhesion Deficiency with Lentiviral Vectors Using the Murine Stem Cell Virus and Human Phosphoglycerate Kinase Promoters
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45. Generation of Genetically Engineered Precursor T-Cells From Human Umbilical Cord Blood Using an Optimized Alpharetroviral Vector Platform
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46. From Bench to Bedside: Preclinical Evaluation of a Self-Inactivating Gammaretroviral Vector for the Gene Therapy of X-linked Chronic Granulomatous Disease
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47. Factor VIII delivered by haematopoietic stem cell-derived B cells corrects the phenotype of haemophilia A mice
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48. An inducible Tet-Off-H2B-GFP lentiviral reporter vector for detection and in vivo isolation of label-retaining cells
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49. TheSleeping Beautytransposon system for clinical applications
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50. Gene therapy for β-thalassaemia: the continuing challenge
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51. Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia
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52. Practical considerations for chimeric antigen receptor design and delivery
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53. Delivery of large transgene cassettes by foamy virus vector
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54. The biochemical basis of interactions between Glucocerebrosidase and alpha‐synuclein in GBA 1 mutation carriers
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55. Integration Site and Clonal Expansion in Human Chronic Retroviral Infection and Gene Therapy
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56. Generation of High-Titer Self-Inactivated γ-Retroviral Vector Producer Cells
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57. Foamy Virus Vectors for HIV Gene Therapy
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58. Ex Vivo Selection of Transduced Hematopoietic Stem Cells for Gene Therapy of β-Hemoglobinopathies
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59. Emerging Therapeutic Approaches for Diamond Blackfan Anemia
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60. Gene Therapy Strategies: Can We Eradicate HIV?
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61. Assembly of infectious Kaposi’s sarcoma-associated herpesvirus progeny requires formation of a pORF19 pentamer
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62. Gene Targeting in Neuroendocrinology
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63. Correcting inborn errors of immunity: From viral mediated gene addition to gene editing
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64. Advancements in Viral Gene Therapy for Gaucher Disease
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65. Identification and Characterization of Enhancer-Blocking Insulators to Reduce Retroviral Vector Genotoxicity
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66. Comprehensive Investigation of Parameter Choice in Viral Integration Site Analysis and Its Effects on the Gene Annotations Produced
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67. Deletion of the LTR Enhancer/Promoter Has No Impact on the Integration Profile of MLV Vectors in Human Hematopoietic Progenitors
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68. Gene Therapy - Developments and Future Perspectives
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69. Genetic Strategies for HIV Treatment and Prevention
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70. Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency
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71. Lentiviral vectors encoding human MUC1-specific, MHC-unrestricted single-chain TCR and a fusion suicide gene: potential for universal and safe cancer immunotherapy
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72. Gene Therapy for Primary Immunodeficiency
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73. Retroviral Vector Performance in Defined Chromosomal Loci of Modular Packaging Cell Lines
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74. Curative approaches for sickle cell disease: A review of allogeneic and autologous strategies.
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75. Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases
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76. Lack of genotoxicity due to foamy virus vector integration in human iPSCs
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77. Improving TCR Gene Therapy for Treatment of Haematological Malignancies
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78. Critical Variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency
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79. Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis
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80. Insulated Foamy Viral Vectors
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81. Supplying Clotting Factors From Hematopoietic Stem Cell–derived Erythroid and Megakaryocytic Lineage Cells
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82. Evidence for the in vivo safety of insulated foamy viral vectors
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83. Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal
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84. Partially corrected X-linked severe combined immunodeficiency: long-term problems and treatment options
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85. Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing
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86. Gene and Cellular Therapies for Leukodystrophies
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87. Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective
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88. Barcoding of Macaque Hematopoietic Stem and Progenitor Cells: A Robust Platform to Assess Vector Genotoxicity
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89. Lentivirus technologies for modulation of the immune system
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90. Lentiviral MGMTP140K-mediated in vivo selection employing a ubiquitous chromatin opening element (A2UCOE) linked to a cellular promoter
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91. Rapid and Efficient Stable Gene Transfer to Mesenchymal Stromal Cells Using a Modified Foamy Virus Vector
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92. Time-course study of the protection induced by an interferon-inducible DNA vaccine against viral haemorrhagic septicaemia in rainbow trout
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93. Stem Cell Biology in Health and Disease
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94. Development of an in vitro genotoxicity assay to detect retroviral vector-induced lymphoid insertional mutants
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95. Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD
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96. 3′ Self-Inactivating Long Terminal Repeat Inserts for the Modulation of Transgene Expression from Lentiviral Vectors
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97. Gene Therapy for Primary Immunodeficiency
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98. Innovative Therapies for Hemoglobin Disorders
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99. Retroviral Vector Technology for Gene Therapy: History, Current Landscape, and Future Prospects
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100. Gene therapy for epilepsy: An emerging, promising approach for a serious neurological disorder
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