34 results on '"Meneghini, Vasco"'
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2. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
3. AAV vectors trigger DNA damage responses and STING-dependent inflammation in human CNS cells
4. Intracerebroventricular transplantation of human iPSC-derived neural stem cells (hiPSC-NSCs) into neonatal mice
5. GATA Factor-Mediated Gene Regulation in Human Erythropoiesis
6. Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe‐affected non‐human primates by intracerebral lentiviral gene therapy
7. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
8. An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
9. Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
10. Human iPSC-derived neural stem cells display a radial glia-like signaturein vitroand favorable long-term safety in transplanted mice
11. Editorial: Ex-vivo and in-vivo genome engineering for metabolic and neurometabolic diseases
12. Human iPSC-based models highlight defective glial and neuronal differentiation from neural progenitor cells in metachromatic leukodystrophy
13. Generation of Human Induced Pluripotent Stem Cell‐Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy
14. Chapter 7 - Intracerebroventricular transplantation of human iPSC-derived neural stem cells (hiPSC-NSCs) into neonatal mice
15. In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges
16. A novel role for the Receptor for Advanced Glycation End-products in neural progenitor cells derived from adult SubVentricular Zone
17. TBX3, the gene mutated in ulnar-mammary syndrome, promotes growth of mammary epithelial cells via repression of p19ARF, independently of p53
18. Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System
19. Novel TBX3 mutation data in families with Ulnar–Mammary syndrome indicate a genotype–phenotype relationship: mutations that do not disrupt the T-domain are associated with less severe limb defects
20. Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases
21. Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
22. Differential Requirements for the RAD51 Paralogs in Genome Repair and Maintenance in Human Cells
23. Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes
24. The Cohesin Release Factor WAPL Restricts Chromatin Loop Extension
25. Induction of Fetal Hemoglobin Synthesis By Crispr/Cas9-Mediated Disruption of the β-Globin Locus Architecture
26. Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy
27. 559. Induction of Fetal Hemoglobin in Adult Erythroblasts by Genome Editing of the Beta-Globin Locus
28. The Noradrenergic Component in Tapentadol Action Counteractsμ-Opioid Receptor–Mediated Adverse Effects on Adult Neurogenesis
29. α2δ Ligands Act as Positive Modulators of Adult Hippocampal Neurogenesis and Prevent Depression-Like Behavior Induced by Chronic Restraint Stress
30. High-Mobility Group Box-1 Protein and β-Amyloid Oligomers Promote Neuronal Differentiation of Adult Hippocampal Neural Progenitors via Receptor for Advanced Glycation End Products/Nuclear Factor-κB Axis: Relevance for Alzheimer's Disease.
31. The Noradrenergic Component in Tapentadol Action Counteracts μ-Opioid Receptor–Mediated Adverse Effects on Adult Neurogenesis
32. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
33. Editing a ?-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes
34. Intracerebroventricular transplantation of human iPSC-derived neural stem cells (hiPSC-NSCs) into neonatal mice.
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