A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

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Items 101–200 of 367

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   Selami Demirci, Alexis Kristine Leonard, Khaled Essawi, John F. Tisdale · 2021 · Molecular Therapy — Methods & Clinical Development
2. Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34+ cells in vitro and in vivo
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3. CRISPR/Cas9 System: A Bacterial Tailor for Genomic Engineering
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4. Immune mechanisms involved in sickle cell disease pathogenesis: current knowledge and perspectives
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5. A long noncoding RNA from the HBS1L-MYB intergenic region on chr6q23 regulates human fetal hemoglobin expression
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6. Therapy Development by Genome Editing of Hematopoietic Stem Cells
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7. Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?
   Leonard, Alexis, Tisdale, John, Abraham, Allistair · 2020 · British Journal of Haematology
8. A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies
   Olivier Humbert, Christopher W. Peterson, Zachary K. Norgaard, Stefan Radtke, Hans‐Peter Kiem · 2017 · Molecular Therapy — Methods & Clinical Development
9. Switching from fetal to adult hemoglobin
   Xunde Wang, Swee Lay Thein · 2018 · Nature Genetics
10. An All-in-One Nanomedicine Consisting of CRISPR-Cas9 and an Autoantigen Peptide for Restoring Specific Immune Tolerance
    Ying‐Li Luo, Lifang Liang, Yun-Jiu Gan, Jing Liu, Yue Zhang, Ya-Nan Fan, Gui Zhao, Anna Czarna, Zi-Dong Lu, Xiao‐Jiao Du et al. · 2020 · ACS Applied Materials & Interfaces
11. Sickle cell disease: progress towards combination drug therapy
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12. Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients
    Lucia Carmela Cosenza, Jessica Gasparello, Nicola Romanini, Matteo Zurlo, Cristina Zuccato, Roberto Gambari, Alessia Finotti · 2021 · Molecular Therapy — Methods & Clinical Development
13. Concise Review: Epigenetic Regulation of Hematopoiesis: Biological Insights and Therapeutic Applications
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14. Gene Therapy and Gene Editing for β-Thalassemia
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15. Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies
    Chang Li, Nikoletta Psatha, Hongjie Wang, Manvendra Singh, Himanshu Bhusan Samal, Wenli Zhang, Anja Ehrhardt, Zsuzsanna Izsvák, Thalia Papayannopoulou, André Lieber · 2018 · Molecular Therapy — Methods & Clinical Development
16. CRISPR to fix bad blood: a new tool in basic and clinical hematology
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17. Designed Two- and Three-Dimensional Protein Nanocage Networks Driven by Hydrophobic Interactions Contributed by Amyloidogenic Motifs
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18. Therapeutic gene editing in haematological disorders with CRISPR /Cas9
    Trine I. Jensen, Esben Axelgaard, Rasmus Otkjær Bak · 2019 · British Journal of Haematology
19. Hematopoietic Stem Cell-Targeted Gene-Addition and Gene-Editing Strategies for β-hemoglobinopathies
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20. Pharmacological and molecular approaches for the treatment of β‐hemoglobin disorders
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21. CRISPR/Cas9-based gene-editing technology for sickle cell disease
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22. Revolutionising healing: Gene Editing's breakthrough against sickle cell disease
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23. Genetic therapies for sickle cell disease
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24. Gene therapies for transfusion dependent β‐thalassemia: Current status and critical criteria for success
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25. Gene Therapy of the Hemoglobinopathies
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26. Impact of Genetic Variation on CRISPR-Cas Targeting
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27. CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantation
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28. Current advances of CRISPR-Cas technology in cell therapy
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29. Sickle Cell Disease: From Genetics to Curative Approaches
    Hardouin, Giulia, Magrin, Elisa, Corsia, Alice, Cavazzana, Marina, Miccio, Annarita, Semeraro, Michaela · 2023 · Annual Review of Genomics and Human Genetics
30. Exosomes for CRISPR-Cas9 Delivery: The Cutting Edge in Genome Editing
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31. Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells
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32. Gene therapy and gene editing strategies for hemoglobinopathies
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33. Synthetic Vehicles for Encapsulation and Delivery of CRISPR/Cas9 Gene Editing Machinery
    Valentina Carboni, Carine Maaliki, Mram Alyami, Shahad K. Alsaiari, Niveen M. Khashab · 2019 · Advanced Therapeutics
34. Epigenetic inactivation of ERF reactivates γ-globin expression in β-thalassemia
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35. Diverse Approaches to Gene Therapy of Sickle Cell Disease
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36. Potential new approaches to the management of the Hb Bart’s hydrops fetalis syndrome: the most severe form of α-thalassemia
    Andrew John King, Douglas R. Higgs · 2018 · Hematology
37. Molecular Medicine: Found in Translation
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38. A unified model of human hemoglobin switching through single-cell genome editing
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39. Illuminating the genome-wide activity of genome editors for safe and effective therapeutics
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40. Genome Editing for β-Hemoglobinopathies: Advances and Challenges
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41. Current advances in overcoming obstacles of CRISPR/Cas9 off-target genome editing
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42. CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia
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43. Commentary: Programmable base editing of A·T to G·C in genomic DNA without DNA cleavage
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44. Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses
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45. Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges
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46. Genetic therapies for the first molecular disease
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47. Genome editing strategies for fetal hemoglobin induction in beta-hemoglobinopathies
    Selami Demirci, Alexis Kristine Leonard, John F. Tisdale · 2020 · Human Molecular Genetics
48. CRISPR-based therapeutic genome editing for inherited blood disorders
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49. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease
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50. Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders
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51. Genome editing approaches to β-hemoglobinopathies
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52. Preclinical evaluation for engraftment of CD34+ cells gene-edited at the sickle cell disease locus in xenograft mouse and non-human primate models
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53. Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy
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54. Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies
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55. Promise of gene therapy to treat sickle cell disease
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56. Gene editing of PKLR gene in human hematopoietic progenitors through 5’ and 3’ UTR modified TALEN mRNA
    Oscar Quintana Bustamante, Sara Fañanas-Baquero, Israel Orman, Raúl Torres-Ruiz, Philippe N. Duchateau, Laurent Poirot, Agnès Gouble, Juan Antonio Bueren, Jose Carlos Segovia · 2019 · PLoS ONE
57. Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches
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58. Fluorescent labeling of CRISPR/Cas9 RNP for gene knockout in HSPCs and iPSCs reveals an essential role for GADD45b in stress response
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59. Immunoresponse to Gene-Modified Hematopoietic Stem Cells
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60. CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells
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61. BCL11A-deficient human erythropoiesis is impaired in vitro and after xenotransplantation into mice
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62. Investigational drugs in phase I and phase II clinical trials for thalassemia
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63. High-level embryonic globin production with efficient erythroid differentiation from a K562 erythroleukemia cell line
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64. Robust CRISPR/Cas9 Genome Editing of the HUDEP-2 Erythroid Precursor Line Using Plasmids and Single-Stranded Oligonucleotide Donors
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65. Multiplexed tat-Targeting CRISPR-Cas9 Protects T Cells from Acute HIV-1 Infection with Inhibition of Viral Escape
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66. Genome and Epigenome Editing to Treat Disorders of the Hematopoietic System
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67. CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside
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68. Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies
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69. Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations
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70. Nanoparticles targeting hematopoietic stem and progenitor cells: Multimodal carriers for the treatment of hematological diseases
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71. The future of sickle cell disease therapeutics rests in genomics
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72. Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies
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73. CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression
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74. A Versatile Tool for the Quantification of CRISPR/Cas9-Induced Genome Editing Events in Human Hematopoietic Cell Lines and Hematopoietic Stem/Progenitor Cells
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76. Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies
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77. 3′HS1 CTCF binding site in human β-globin locus regulates fetal hemoglobin expression
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79. Genome-based therapeutic interventions for β-type hemoglobinopathies
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80. In vivo genome editing of human haematopoietic stem cells for treatment of blood disorders using mRNA delivery
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81. HRI depletion cooperates with pharmacologic inducers to elevate fetal hemoglobin and reduce sickle cell formation
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83. Simultaneous high-efficiency base editing and reprogramming of patient fibroblasts
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84. In Vivo Hematopoietic Stem Cell Genome Editing: Perspectives and Limitations
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86. Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment
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87. A Cas9-mediated adenosine transient reporter enables enrichment of ABE-targeted cells
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88. SEC23A rescues SEC23B-deficient congenital dyserythropoietic anemia type II
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90. Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease
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94. Genome editing of immune cells using CRISPR/Cas9
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95. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
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96. Editing human hematopoietic stem cells: advances and challenges
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97. CRISPR-Directed Therapeutic Correction at the NCF1 Locus Is Challenged by Frequent Incidence of Chromosomal Deletions
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99. Targeting fetal hemoglobin expression to treat β hemoglobinopathies
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