315 results on '"Miccio, Annarita"'
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2. Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells
3. CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing
4. Revolutionising healing: Gene Editing's breakthrough against sickle cell disease
5. Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses
6. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
7. An optimized SpCas9 high-fidelity variant for direct protein delivery
8. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
9. Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation
10. Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
11. Cyclosporin H Improves the Transduction of CD34+ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease.
12. Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
13. Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
14. GATA Factor-Mediated Gene Regulation in Human Erythropoiesis
15. Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies
16. Genome editing approaches to β-hemoglobinopathies
17. A Genome Editing System for Therapeutical Targeting of Stem Cells
18. Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
19. Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges
20. Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
21. Human iPSC-derived neural stem cells display a radial glia-like signaturein vitroand favorable long-term safety in transplanted mice
22. Faculty Opinions recommendation of Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia.
23. Faculty Opinions recommendation of Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia.
24. Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
25. Faculty Opinions recommendation of Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing.
26. Progress and harmonization of gene editing to treat human diseases:Proceeding of COST Action CA21113 GenE-HumDi
27. Epigenetic Regulation of β-Globin Genes and the Potential to Treat Hemoglobinopathies through Epigenome Editing
28. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
29. Faculty Opinions recommendation of Increased incidence of hematologic malignancies in SCD after HCT in adults with graft failure and mixed chimerism.
30. Targeted Base Editing Strategies for Beta-Hemoglobinopathies
31. Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing
32. CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy
33. Concise Review: Epigenetic Regulation of Hematopoiesis: Biological Insights and Therapeutic Applications
34. Sickle Cell Disease: From Genetics to Curative Approaches.
35. In vivo Selection of Genetically Modified Erythroblastic Progenitors Leads to Long-Term Correction of β-Thalassemia
36. CRISPR's Path to the Clinic
37. Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges
38. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
39. FOG1 requires NuRD to promote hematopoiesis and maintain lineage fidelity within the megakaryocytic-erythroid compartment
40. Pre-Clinical Development of a Highly Efficient TALEN ®-Based Correction of the β-Globin Gene in Patient-Derived Hematopoietic Stem and Progenitor Cells (HSPCs) to Treat Sickle Cell Disease
41. Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies
42. Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients
43. Base Editing-Mediated Dissection of the -200 Region of the γ-Globin Promoters to Induce Fetal Hemoglobin and Rescue Sickle Cell Disease and β-Thalassemia
44. Dysérythropoïèse dans la drépanocytose : une contribution d’origine centrale à l’anémie ?
45. Editorial: Mutation-Specific Gene Editing for Blood Disorders
46. Recent progress in genome editing for gene therapy applications: the French perspective
47. Special Issue: CRISPR's Path to the Clinic
48. Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts
49. NuRD mediates activating and repressive functions of GATA‐1 and FOG‐1 during blood development
50. In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of [beta]-thalassemia
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