81 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. Carbonyl iron and iron dextran therapies cause adverse effects on bone health in juveniles with chronic kidney disease
5. Heparanase Level and Procoagulant Activity Are Increased in Thalassemia and Attenuated by Janus Kinase 2 Inhibition
6. Cranberry A-type proanthocyanidins selectively target acute myeloid leukemia cells
7. Lobe specificity of iron binding to transferrin modulates murine erythropoiesis and iron homeostasis
8. Iron metabolism under conditions of ineffective erythropoiesis in β-thalassemia
9. Hepcidin agonists as therapeutic tools
10. Structure-function analysis of ferroportin defines the binding site and an alternative mechanism of action of hepcidin
11. Single vs. dual transgene ex vivo gene therapy for multiple sulfatase deficiency
12. Tropomodulin 1 controls erythroblast enucleation via regulation of F-actin in the enucleosome
13. Decreasing TfR1 expression reverses anemia and hepcidin suppression in β-thalassemic mice
14. Minihepcidin peptides as disease modifiers in mice affected by β-thalassemia and polycythemia vera
15. Distinct roles for hepcidin and interleukin-6 in the recovery from anemia in mice injected with heat-killed Brucella abortus
16. Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia
17. Establishment of High Titer Baboon Endogenous Retrovirus Envelope Pseudotyped Lentiviral Vector Production Protocol Focusing on Syncytium Formation and Exfoliation
18. Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?
19. Enhanced erythropoiesis in Hfe-KO mice indicates a role for Hfe in the modulation of erythroid iron homeostasis
20. Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in β-thalassemia
21. Protective role of calreticulin in HFE hemochromatosis
22. Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia
23. In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA
24. TNFα and Anti-IFNγ Rescues Chronic Anemia of Inflammation Phenotype in Tnfαko Mice Treated with Heat-Killed Brucella Abortus
25. Direct Delivery of mRNA-Loaded Lipid Nanoparticles to the Hematopoietic Stem Cell Niche
26. The Effect of Monoferric Transferrins on Hypoferremia in a Murine Model of Inflammation
27. Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6-ASO Is Superior for Correcting β-Thalassemia
28. Differential Effects of Monoferric Forms of Transferrin on Mouse Models of Anemia of Inflammation and Congenital Anemia
29. Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study
30. Improved Gene Therapy for Metachromatic Leukodystrophy
31. Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy
32. Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers
33. Elevated P21 (CDKN1a) Mediates Apoptosis of Beta-Thalassemic Erythroid Cells in Mice but Its Ablation Doesn't Improve Erythroid Maturation
34. Improved Lentiviral Vectors for the Cure of Hemoglobinopathies
35. Chapter 38 - Thalassemia Syndromes
36. Decreased hepcidin expression in murine β-thalassemia is associated with suppression of Bmp/Smad signaling
37. Generation of New Cell Models to Screen Improved Vectors for Functional Hemoglobin Expression
38. Absence of Interleukin-6 Protects Murine Bone Marrow Erythropoiesis Under Inflammation, in a Process Reversible By Iron Mediated Reactive Oxygen Species Upregulation
39. Influential Role of Macrophages in Hepcidin Regulation in Apo-Transferrin Treated β-Thalassemic Mice
40. Iron Binding to Specific Lobes of Transferrin Differentially Affects Erythropoiesis and Hepcidin Signaling
41. Ex Vivo Gene Therapy Approach By Targt Technology for the Treatment of β-Thalassemia Intermedia
42. Genetic Investigation of the Role of GDF11 in the Treatment of β-Thalassemia and MDS
43. A Double Knock out of Hepcidin and IL6 Demonstrates Independent Roles of the Two Genes in Anemia of Inflammation
44. Lack of Beta-1 Integrin Limits Stress Erythropoiesis and Splenomegaly in Beta-Thalassemia
45. Down-Regulation of TfR1 Increases Erythroid Precursor Enucleation and Hepatocyte Hepcidin Expression in ß-Thalassemic Mice
46. Targeting TMPRSS6 Using Antisense Technology for the Treatment of Beta-Thalassemia
47. Exogenous Apo-Transferrin Increases Monoferric Transferrin, Decreasing Cytosolic Iron Uptake and Heme and Globin Synthesis in β-Thalassemic Mice
48. Combination of Tmprss6-ASO and the Iron Chelator Deferiprone Improves Erythropoiesis and Reduces Iron Overload in a Mouse Model of Beta-Thalassemia
49. Use of Minihepcidins As a “Medical Phlebotomy” in the Treatment of Polycythemia Vera
50. Beta-1 Integrin Controls Homing and Expansion of Erythroid Cells in Stress Erythropoiesis and ß-Thalassemia
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