All Stories

  1. Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials
  2. European Joint Programme on Rare Diseases workshop: LAMA2-muscular dystrophy: paving the road to therapy March 17–19, 2023, Barcelona, Spain
  3. Dysbiosis: An Indicator of COVID-19 Severity in Critically Ill Patients
  4. Oxidative Stress in Structural Valve Deterioration: A Longitudinal Clinical Study
  5. Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy
  6. Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy
  7. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia
  8. Variable readthrough responsiveness of nonsense mutations in hemophilia A
  9. Absence of p.R50X Pygm read-through in McArdle disease cellular models
  10. Expression of microRNA-155 in inflammatory cells modulates liver injury
  11. Advanced cell-based modeling of the royal disease: characterization of the mutated F9 mRNA
  12. Response of the human myocardium to ischemic injury and preconditioning: The role of cardiac and comorbid conditions, medical treatment, and basal redox status
  13. Myeloid-derived suppressor cells can be efficiently generated from human hematopoietic progenitors and peripheral blood monocytes
  14. Myeloid-derived suppressor cells expressing a self-antigen ameliorate experimental autoimmune encephalomyelitis
  15. A reproducible method for the isolation and expansion of ovine mesenchymal stromal cells from bone marrow for use in regenerative medicine preclinical studies
  16. Long-Term Restoration of Thymidine Phosphorylase Function and Nucleoside Homeostasis Using Hematopoietic Gene Therapy in a Murine Model of Mitochondrial Neurogastrointestinal Encephalomyopathy
  17. Prospective therapeutic approaches in mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)
  18. 279. Efficient and Safe Lentiviral Vector-Mediated Hematopoietic Stem Cell Gene Therapy in MNGIE Mice
  19. Molecular characterization of ten F8 splicing mutations in RNA isolated from patient's leucocytes: assessment of in silico prediction tools accuracy
  20. Hematopoietic chimerisms: friends or foes?
  21. Thymidine phosphorylase is both a therapeutic and a suicide gene in a murine model of mitochondrial neurogastrointestinal encephalomyopathy
  22. Gene therapy using a liver-targeted AAV vector restores nucleoside and nucleotide homeostasis in a murine model of MNGIE
  23. Myeloid-Derived Suppressor Cells Are Generated During Retroviral Transduction of Murine Bone Marrow
  24. Bone Marrow Transplantation in Dysferlin-Deficient Mice Results in a Mild Functional Improvement
  25. Notch signals contribute to preserve the multipotentiality of human CD34+CD38−CD45RA−CD90+ hematopoietic progenitors by maintaining T cell lineage differentiation potential
  26. Myeloid-derived suppressor cells (MDSC): Another player in the orchestra
  27. Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE
  28. Dendritic and tumor cell fusions transduced with adenovirus encoding CD40L eradicate B-cell lymphoma and induce a Th17-type response
  29. Identification of multipotent mesenchymal stromal cells in the reactive stroma of a prostate cancer xenograft by side population analysis
  30. Transgene Expression Levels Determine the Immunogenicity of Transduced Hematopoietic Grafts in Partially Myeloablated Mice
  31. Bone Marrow Transplantation Induces Normoglycemia in a Type 2 Diabetes Mellitus Murine Model
  32. Corrigendum to “Tolerance Induction in Experimental Autoimmune Encephalomyelitis Using Non-myeloablative Hematopoietic Gene Therapy With Autoantigen”
  33. Tolerance Induction in Experimental Autoimmune Encephalomyelitis Using Non-myeloablative Hematopoietic Gene Therapy With Autoantigen
  34. The Hoechst low-fluorescent profile of the side population: clonogenicity versus dye retention
  35. Terapia génica: realidades actuales y expectativas
  36. Aplicaciones terapéuticas de las células madre
  37. A rare fraction of human hematopoietic stem cells with large telomeres
  38. Células madre en el tratamiento de la sordera
  39. Flow cytometry-based approach to ABCG2 function suggests that the transporter differentially handles the influx and efflux of drugs
  40. Retroviral vectors: new applications for an old tool
  41. Antiphospholipid antibodies and stroke
  42. Myeloablation enhances engraftment of transduced murine hematopoietic cells, but does not influence long-term expression of the transgene
  43. Terapia génica en células madre hematopoyéticas: mitos y realidades
  44. Efficient transduction of human hematopoietic repopulating cells generating stable engraftment of transgene-expressing cells in NOD/SCID mice
  45. Highly Efficient Transduction of the Green Fluorescent Protein Gene in Human Umbilical Cord Blood Stem Cells Capable of Cobblestone Formation in Long-Term Cultures and Multilineage Engraftment of Immunodeficient Mice
  46. High-Titer Retroviral Vectors Containing the Enhanced Green Fluorescent Protein Gene for Efficient Expression in Hematopoietic Cells
  47. Allogeneic marrow grafts from donors with congenital chromosomal abnormalities in marrow cells
  48. Myelosuppressive conditioning improves autologous engraftment of genetically marked hematopoietic repopulating cells in dogs
  49. Antibodies Against Platelet-Activating Factor in Patients with Antiphospholipid Antibodies
  50. Effect of Human IgG Antiphospholipid Antibodies on an In Vivo Thrombosis Model in Mice
  51. Primary antiphospholipid syndrome
  52. Valvular Heart Disease in the Primary Antiphospholipid Syndrome
  53. Anticardiolipin antibodies and binding of anionic phospholipids and serum protein
  54. Fetal Loss Treatment in Patients with Antiphospholipid Antibodies
  55. Polymyositis and cyclosporin A.
  56. Anticorps antiphospholipide et rickettsioses
  57. The “Primary” Antiphospholipid Syndrome
  58. Fetal loss treatment in patients with antiphospholipid antibodies.
  59. Anticardiolipin Antibodies and Migraine-Related Strokes
  60. Sneddon's syndrome and anticardiolipin antibodies.
  61. Lupus anticoagulant and portal hypertension
  62. Lupus Anticoagulant and Stroke
  63. Serum Thrombocytopenia and High-Dose Immunoglobulin Treatment