507 results on '"Mavilio F"'
Search Results
2. OP29 Haematopoietic stem cell gene therapy as a treatment for severe Crohn’s Disease associated with pathogenic NOD2 genetic variants
3. Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry
4. Anti-Hiv Viral Interference Induced by Retroviral Vectors Expressing a Non-Producer Hiv-1 Variant
5. Differential Activation of Homeobox Genes by Retinoic Acid in Human Embryonal Carcinoma Cells
6. Expression of Cellular Oncogenes in Primary Cells from Human Acute Leukemias
7. Translocation and Rearrangement of c-myc into Immunoglobulin α Heavy Chain Locus in Primary Cells from Acute Lymphocytic Leukemia
8. Molecular Mechanisms of Human Hemoglobin Switching: Selective Undermethylation and Expression of Globin Genes in Embryonic, Fetal, and Adult Erythroblasts
9. Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes
10. Risk assessment in skin gene therapy: viral–cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors
11. IL4 gene delivery to the CNS recruits regulatory T cells and induces clinical recovery in mouse models of multiple sclerosis
12. Absence of an intrathecal immune reaction to a helper-dependent adenoviral vector delivered into the cerebrospinal fluid of non-human primates
13. Erratum: Genomic analysis of sleeping beauty transposon integration in human somatic cells (PLoS ONE (2014) 9:11 (e112712) DOI: 10.1371/journal.pone.0112712)
14. AUTOPHAGIC MYOPATHIES / MYOFIBRILLAR MYOPATHIES / DISTAL MYOPATHIES / POMPE DISEASE
15. Gene therapy of inherited skin adhesion disorders: a critical overview
16. Growth Inhibition and Differentiation of a Human Embryonal Carcinoma (EC) Cell Line by Osteogenic Protein-1 (OP-1)
17. Differential Activation of Homeobox Genes by Retinoic Acid in Human Embryonal Carcinoma Cells
18. Safety of retroviral gene marking with a truncated NGF receptor
19. Gene Therapy Approaches to Hemoglobinopathies
20. 808 Ex vivo COL7A1 correction for recessive dystrophic epidermolysis bullosa using CRISPR/Cas9 and homology directed repair
21. IL-7 and IL-15 instruct the generation of human memory stem T cells from na?¯ve precursors
22. Genetic Polymorphism of the α-Globin Haplotypes in a Population from Calabria (Southern Italy): Studies with Variants of the α-Globin Chain
23. Rearrangement and Abnormal Expression of Human c-myc in Acute Lymphocytic Leukemia
24. Correction of the exon 2 duplication in DMD myoblasts by a single CRISPR/Cas9 system
25. Targeted Genome Editing in Spinal Muscular Atrophy
26. Naive-Derived Memory Stem T Cells: A Novel Promising Platform for Cancer Immune-Gene Therapy
27. The importance of be(ginn)ing Naive: implications for cancer immune-gene therapy
28. In vivo selection of hematopoietic stem cells by a truncated erythropoietin receptor (tEpoR)
29. Cell-Specific Transcriptional Regulatory Domains Attract Gamma Retroviral Integration in the Human Genome
30. Preclinical Assessment of a Gene Therapy Approach to beta-Thalassemia
31. Characterization of potential genomic 'safe harbor' for efficient targeted gene addition with zinc finger nucleases
32. Transcription Factor Binding Sites Are Genetic Determinant of Retroviral Integration in the Human Genome
33. Vector design influences retroviral integration in human hematopoietic cells
34. The influence of vector design on integration site selection by gamma-retroviral and lentiviral vectors
35. 819. Molecular Follow-Up of Patients Treated with Allogeneic Hematopoietic Stem Cell Transplantation and Donor Lymphocytes Transduced with a Retroviral Vector Expressing HSV-TK and ∆LNGFR
36. Retroviral vector integration deregulates gene expression but has no consequences on the biology and function of transplanted T cells
37. Adeno-associated virus vector-mediated liver gene therapy for Crigler-Najjar syndrome
38. Transduction of human hematopoietic stem cells and T-lymphocytes with RD114-TR-pseudotyped lentiviral vectors
39. Molecular follow-up of patients treated with HSV-TK/Delta LNGFR engineered donor lymphocytes after allogeneic stem cell transplantation
40. Gene therapy of Duchenne muscular dystrophy using rAAV vectors: Exon skipping and microdystrophin approaches in GRMD dogs
41. Heterocellular hereditary persistence of fetal hemoglobin (HPFH). Molecular mechanisms of abnormal γ-gene expression in association with β thalassemia and linkage relationship with the β-globin gene cluster
42. Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication
43. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases
44. Self-inactivating alpharetroviral vectors have a low genotoxic integration profile in human CD34(+) hematopoietic progenitor cells
45. Defining the lentiviral integrome in human hematopoietic cells
46. Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)
47. Peripheral vein injection of AAV8-MTM1 leads to long-term survival and correction of severe muscle pathology in a canine model of X-linked myotubular myopathy: Results from a dose escalation study
48. P1213 : Adeno-associated virus vector-mediated liver gene therapy for Crigler-Najjar syndrome
49. Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted retroviral vectors
50. P.300 - Correction of the exon 2 duplication in DMD myoblasts by a single CRISPR/Cas9 system
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