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5. Clinical Implications of p53 Dysfunction in Patients with Myelodysplastic Syndromes

7. Safe therapeutic gene expression by a lentiviral vector for the gene therapy of autosomal recessive osteopetrosis

9. PBX1-directed stem cell transcriptional program drives tumor progression in myeloproliferative neoplasm

11. Exploitation of circulating CD34+ cells and non-genotoxic conditioning to overcome major limitations to treatment for autosomal recessive osteopetrosis

13. 3072 – MICRORNA-127-3P CONTROLS MURINE HEMATOPOIETIC STEM CELL MAINTENANCE BY LIMITING DIFFERENTIATION

14. Generation of an immunodeficient mouse model of tcirg1-deficient autosomal recessive osteopetrosis

15. Expanded circulating hematopoietic stem/progenitor cells as novel cell source for the treatment of TCIRG1 osteopetrosis

16. Chromosome Transplantation: Correction of the Chronic Granulomatous Disease Defect in Mouse Induced Pluripotent Stem Cells

17. MicroRNA-127-3p controls murine hematopoietic stem cell maintenance by limiting differentiation

18. ACKR2 in hematopoietic precursors as a checkpoint of neutrophil release and anti-metastatic activity

20. Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts

24. Osteopetrosis rescue upon RANKL administration toRankl−/−mice: A new therapy for human RANKL-dependent ARO

25. Anti-CD3ε mAb improves thymic architecture and prevents autoimmune manifestations in a mouse model of Omenn syndrome: therapeutic implications

32. Molecular purging of multiple myeloma cells by ex-vivo culture and retroviral transduction of mobilized-blood CD34+ cells

33. Prognostic Evaluation of Myelodysplastic Syndromes (MDS): Analysis of Deaths Due to Age-Related Causes.

34. IL-3 or IL-7 Increases ex Vivo Gene Transfer Efficiency in ADA-SCID BM CD34+ Cells while Maintaining in Vivo Lymphoid Potential

35. Telomere Length Has Prognostic Impact in B-Cell Chronic Lymphocytic Leukemia (B-CLL).

36. Mobilized Blood CD34+Cells Transduced and Selected with a Clinically Applicable Protocol Reconstitute Lymphopoiesis in SCID-Hu Mice

39. Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning

40. Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

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