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1. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

8. Comprehensive characterization of a novel, oncogenic and targetable SEPTIN6::ABL2 fusion in T-ALL

9. A human genome editing–based MLL::AF4 ALL model recapitulates key cellular and molecular leukemogenic features

10. Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

11. Lentiviral Gene Therapy for the Correction of Congenital Dyserythropoietic Anemia Type II

12. Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

13. Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes

16. The Current Status of Mesenchymal Stromal Cells: Controversies, Unresolved Issues and Some Promising Solutions to Improve Their Therapeutic Efficacy

18. Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells

20. Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

22. A comprehensive strategy for the subtyping of patients with Fanconi anaemia: conclusions from the Spanish Fanconi Anemia Research Network

24. Premature aging and cancer development in transgenic mice lacking functional CYLD

25. Advances in the gene therapy of monogenic blood cell diseases.

26. Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

27. IKKβ-Mediated Resistance to Skin Cancer Development Is Ink4a/Arf-Dependent

29. 753. Lentiviral-Mediated Gene Correction of Mobilized Peripheral Blood Progenitors and Repopulating Cells from FA-A Patients

31. Mesenchymal stromal cells enhance the engraftment of hematopoietic stem cells in an autologous mouse transplantation model

32. The NFKB Inducing Kinase Modulates Hematopoiesis During Stress

36. Cardiac Bmi1+ cells contribute to myocardial renewal in the murine adult heart.

37. Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+cells from Fanconi anemia patients

38. Development of Lentiviral Vectors with Optimized Transcriptional Activity for the Gene Therapy of Patients with Fanconi Anemia

42. Constitutively Active Akt Induces Ectodermal Defects and Impaired Bone Morphogenetic Protein Signaling

44. Hematopoietic Dysfunction in a Mouse Model for Fanconi Anemia Group D1

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