86 results on '"Rivella, Stefano"'
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2. Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy
3. Elevated CDKN1A (P21) mediates β-thalassemia erythroid apoptosis, but its loss does not improve β-thalassemic erythropoiesis
4. Protocol for a high titer of BaEV-Rless pseudotyped lentiviral vector: Focus on syncytium formation and detachment
5. Carbonyl iron and iron dextran therapies cause adverse effects on bone health in juveniles with chronic kidney disease
6. Heparanase Level and Procoagulant Activity Are Increased in Thalassemia and Attenuated by Janus Kinase 2 Inhibition
7. Cranberry A-type proanthocyanidins selectively target acute myeloid leukemia cells
8. Lobe specificity of iron binding to transferrin modulates murine erythropoiesis and iron homeostasis
9. Iron metabolism under conditions of ineffective erythropoiesis in β-thalassemia
10. Hepcidin agonists as therapeutic tools
11. What can we learn from ineffective erythropoiesis in thalassemia?
12. Structure-function analysis of ferroportin defines the binding site and an alternative mechanism of action of hepcidin
13. Single vs. dual transgene ex vivo gene therapy for multiple sulfatase deficiency
14. Tropomodulin 1 controls erythroblast enucleation via regulation of F-actin in the enucleosome
15. Decreasing TfR1 expression reverses anemia and hepcidin suppression in β-thalassemic mice
16. Minihepcidin peptides as disease modifiers in mice affected by β-thalassemia and polycythemia vera
17. Distinct roles for hepcidin and interleukin-6 in the recovery from anemia in mice injected with heat-killed Brucella abortus
18. Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia
19. Establishment of High Titer Baboon Endogenous Retrovirus Envelope Pseudotyped Lentiviral Vector Production Protocol Focusing on Syncytium Formation and Exfoliation
20. Gene therapy for hemoglobinopathies: progress and challenges
21. The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia
22. Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?
23. Enhanced erythropoiesis in Hfe-KO mice indicates a role for Hfe in the modulation of erythroid iron homeostasis
24. Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in β-thalassemia
25. Protective role of calreticulin in HFE hemochromatosis
26. Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia
27. In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA
28. TNFα and Anti-IFNγ Rescues Chronic Anemia of Inflammation Phenotype in Tnfαko Mice Treated with Heat-Killed Brucella Abortus
29. Direct Delivery of mRNA-Loaded Lipid Nanoparticles to the Hematopoietic Stem Cell Niche
30. The Effect of Monoferric Transferrins on Hypoferremia in a Murine Model of Inflammation
31. Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6-ASO Is Superior for Correcting β-Thalassemia
32. Differential Effects of Monoferric Forms of Transferrin on Mouse Models of Anemia of Inflammation and Congenital Anemia
33. Globin gene transfer for treatment of the β-thalassemias and sickle cell disease
34. Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study
35. Improved Gene Therapy for Metachromatic Leukodystrophy
36. Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy
37. Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers
38. Elevated P21 (CDKN1a) Mediates Apoptosis of Beta-Thalassemic Erythroid Cells in Mice but Its Ablation Doesn't Improve Erythroid Maturation
39. Improved Lentiviral Vectors for the Cure of Hemoglobinopathies
40. Chapter 38 - Thalassemia Syndromes
41. Decreased hepcidin expression in murine β-thalassemia is associated with suppression of Bmp/Smad signaling
42. Generation of New Cell Models to Screen Improved Vectors for Functional Hemoglobin Expression
43. Absence of Interleukin-6 Protects Murine Bone Marrow Erythropoiesis Under Inflammation, in a Process Reversible By Iron Mediated Reactive Oxygen Species Upregulation
44. Influential Role of Macrophages in Hepcidin Regulation in Apo-Transferrin Treated β-Thalassemic Mice
45. Iron Binding to Specific Lobes of Transferrin Differentially Affects Erythropoiesis and Hepcidin Signaling
46. Ex Vivo Gene Therapy Approach By Targt Technology for the Treatment of β-Thalassemia Intermedia
47. Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS
48. A Double Knock out of Hepcidin and IL6 Demonstrates Independent Roles of the Two Genes in Anemia of Inflammation
49. Lack of Beta-1 Integrin Limits Stress Erythropoiesis and Splenomegaly in Beta-Thalassemia
50. Down-Regulation of TfR1 Increases Erythroid Precursor Enucleation and Hepatocyte Hepcidin Expression in ß-Thalassemic Mice
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