170 results on '"Segovia, José C."'
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2. Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes
3. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia
4. Non-Homologous End-Joining Defect in Fanconi Anemia Hematopoietic Cells Exposed to Ionizing Radiation
5. Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients
6. Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency
7. Comprehensive characterization of a novel, oncogenic and targetable SEPTIN6::ABL2 fusion in T-ALL
8. A human genome editing–based MLL::AF4 ALL model recapitulates key cellular and molecular leukemogenic features
9. Figure S1 from IKKβ-Mediated Resistance to Skin Cancer Development Is Ink4a/Arf-Dependent
10. Supplementary Table 1 from IKKβ-Mediated Resistance to Skin Cancer Development Is Ink4a/Arf-Dependent
11. Supplementary Information from IKKβ-Mediated Resistance to Skin Cancer Development Is Ink4a/Arf-Dependent
12. Data from IKKβ-Mediated Resistance to Skin Cancer Development Is Ink4a/Arf-Dependent
13. Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells
14. Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study
15. Lentiviral Gene Therapy for the Correction of Congenital Dyserythropoietic Anemia Type II
16. Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients
17. Rescue of Pyruvate Kinase Deficiency in Mice by Gene Therapy Using the Human Isoenzyme
18. Increased Intraocular Insulin-like Growth Factor-I Triggers Blood-Retinal Barrier Breakdown
19. In vivo proliferation advantage of genetically corrected hematopoietic stem cells in a mouse model of Fanconi anemia FA-D1
20. Squalene in a sex-dependent manner modulates atherosclerotic lesion which correlates with hepatic fat content in apoE-knockout male mice
21. Characteristics of Lentiviral Vectors Harboring the Proximal Promoter of the vav Proto-oncogene: A Weak and Efficient Promoter for Gene Therapy
22. Olive oil preparation determines the atherosclerotic protection in apolipoprotein E knockout mice
23. The Current Status of Mesenchymal Stromal Cells: Controversies, Unresolved Issues and Some Promising Solutions to Improve Their Therapeutic Efficacy
24. Hematopoietic Dysfunction in a Mouse Model for Fanconi Anemia Group D1
25. Optimising stable retroviral transduction of primary human synovial fibroblasts
26. Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells
27. Modelling Congenital Dyserythropoietic Anemia Type II through Gene Editing in Hematopoietic Stem and Progenitor Cells
28. Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients
29. A comprehensive strategy for the subtyping of patients with Fanconi anaemia: conclusions from the Spanish Fanconi Anemia Research Network
30. Genetic modification of hematopoietic stem cells: recent advances in the gene therapy of inherited diseases
31. Advances in the gene therapy of monogenic blood cell diseases
32. In vitro and in vivo susceptibility of mouse megakaryocytic progenitors to strain i of parvovirus minute virus of mice
33. Premature aging and cancer development in transgenic mice lacking functional CYLD
34. IKKβ-Mediated Resistance to Skin Cancer Development Is Ink4a/Arf-Dependent
35. Advances in the gene therapy of monogenic blood cell diseases.
36. Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy
37. Long-term skin regeneration in xenografts from iPSC teratoma-derived human keratinocytes
38. 753. Lentiviral-Mediated Gene Correction of Mobilized Peripheral Blood Progenitors and Repopulating Cells from FA-A Patients
39. Efficient CRISPR/Cas9-Mediated Gene Editing of Pklr in Human Hematopoietic Progenitors and Stem Cells for the Gene Therapy of Pyruvate Kinase Deficiency
40. Estrogens Enhance Human Hematopoietic Stem Cell Engraftment in a Xenogenic Transplantation Model
41. Advances in the Gene Therapy of Patients with Fanconi Anemia
42. Cardiac Bmi1 + cells contribute to myocardial renewal in the murine adult heart
43. Mesenchymal stromal cells enhance the engraftment of hematopoietic stem cells in an autologous mouse transplantation model
44. The NFKB Inducing Kinase Modulates Hematopoiesis During Stress
45. Interacting Resident Epicardium-Derived Fibroblasts and Recruited Bone Marrow Cells Form Myocardial Infarction Scar
46. Brief Report: Reduced Expression of CD18 Leads to the In Vivo Expansion of Hematopoietic Stem Cells in Mouse Bone Marrow
47. Gene Editing of the Human Pklr Gene in Human Hematopoietic Progenitors to Correct Pyruvate Kinase Deficiency
48. 228. Characterization of Hematopoietic Progenitors from Pyruvate Kinase Deficient (PKD) Patients and Transduction of PKD CD34+ Cells with a Therapeutic Lentiviral Vector
49. Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+cells from Fanconi anemia patients
50. Development of Lentiviral Vectors with Optimized Transcriptional Activity for the Gene Therapy of Patients with Fanconi Anemia
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