244 results on '"Rivella, Stefano"'
Search Results
2. Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations
3. Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of Multiple Sulfatase Deficiency
4. Optimizing lentiviral genomic integrations to cure beta-thalassemia: The least required for success?
5. Single vs. dual transgene gene therapy for multiple sulfatase deficiency
6. Elevated CDKN1a (p21) mediates β-thalassemia erythroid apoptosis but its loss does not improve β-thalassemic erythropoiesis
7. Normal and dysregulated crosstalk between iron metabolism and erythropoiesis
8. Novel potential therapeutics to modify iron metabolism and red cell synthesis in diseases associated with defective erythropoiesis
9. Pathogenic Mechanisms in Thalassemia I
10. Protocol for a high titer of BaEV-Rless pseudotyped lentiviral vector: Focus on syncytium formation and detachment
11. Optimizing cross-correction to overcome limitations of gene therapy in multiple sulfatase deficiency
12. TMPRSS6 as a Therapeutic Target for Disorders of Erythropoiesis and Iron Homeostasis
13. In Vivo Modification of Hematopoietic Stem Cells By Targeted Lipid Nanoparticles Encapsulating mRNA
14. Direct Delivery of mRNA-Loaded Lipid Nanoparticles to the Hematopoietic Stem Cell Niche
15. The Effect of Monoferric Transferrins on Hypoferremia in a Murine Model of Inflammation
16. Establishment of High Titer Baboon Endogenous Retrovirus Envelope Pseudotyped Lentiviral Vector Production Protocol Focusing on Syncytium Formation and Exfoliation
17. Differential Effects of Monoferric Forms of Transferrin on Mouse Models of Anemia of Inflammation and Congenital Anemia
18. Combination of a TGF-β Ligand Trap (RAP-GRL) and TMPRSS6-ASO Is Superior for Correcting β-Thalassemia
19. TNFα and Anti-IFNγ Rescues Chronic Anemia of Inflammation Phenotype in Tnfαko Mice Treated with Heat-Killed Brucella Abortus
20. Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia
21. Emergent treatments for β-thalassemia and orphan drug legislations
22. Transferrin receptor 2 (Tfr2) genetic deletion makes transfusion‐independent a murine model of transfusion‐dependent β‐thalassemia
23. Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia
24. Elevated p21 (CDKN1a) mediates β-thalassemia erythroid apoptosis but its loss does not improve β-thalassemic erythropoiesis
25. Tmprss6-ASO as a tool for the treatment of Polycythemia Vera mice
26. Tfr2 Genetic Deletion Makes Transfusion-Independent a Murine Model of Transfusion-Dependent β-Thalassemia
27. Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy
28. Lack of IL6 Improves Recovery from Anemia of Inflammation Which Gets Hampered in Presence of Excess Iron
29. Combination of a Luspatercept-like Drug (RAP-GRL) and Tmprss6-ASO Is Superior to Either Drug Alone for Correcting β-Thalassemia
30. A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment
31. TNFα Controls the Delicate Balance between Erythropoiesis and Stem Cell Exhaustion during Inflammatory Stress
32. Obligate N-Terminal but Not C-Terminal Monoferric Transferrin Ameliorates Anemia in β-Thalassemic Mice
33. Improved Gene Therapy for Metachromatic Leukodystrophy
34. Dietary Iron Increases Expression of Liver Hepcidin Relative to BMP6 By a Mechanism Involving Transferrin Receptor 2 and Specificity of Transferrin Lobe Iron Occupancy
35. DNA binding to TLR9 expressed by red blood cells promotes innate immune activation and anemia
36. Gene Therapy for Hemoglobinopathies
37. Contributors
38. The Role of Iron in Benign and Malignant Hematopoiesis
39. The EHA Research Roadmap: Anemias
40. Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy
41. Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin
42. The hepcidin regulator erythroferrone is a new member of the erythropoiesis-iron-bone circuitry
43. Author response: The hepcidin regulator erythroferrone is a new member of the erythropoiesis-iron-bone circuitry
44. Pleckstrin-2 is essential for erythropoiesis in β-thalassemic mice, reducing apoptosis and enhancing enucleation
45. The Hepcidin Regulator Erythroferrone is a New Member of the Erythropoiesis–Iron–Bone Circuitry
46. Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies
47. Red Blood Cells Function as DNA Sensors
48. CYP450 Mediates Reactive Oxygen Species Production in a Mouse Model of β-Thalassemia through an Increase in 20-HETE Activity
49. Management of non‐transfusion‐dependent β‐thalassemia ( NTDT ): The next 5 years
50. Revisiting the non‐transfusion‐dependent (NTDT) vs. transfusion‐dependent (TDT) thalassemia classification 10 years later
Catalog
Books, media, physical & digital resources
Discovery Service for Jio Institute Digital Library
For full access to our library's resources, please sign in.