89 results on '"Miccio, Annarita"'
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2. CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing
3. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
4. Genome editing approaches to β-hemoglobinopathies
5. A Genome Editing System for Therapeutical Targeting of Stem Cells
6. Human iPSC-derived neural stem cells display a radial glia-like signaturein vitroand favorable long-term safety in transplanted mice
7. Faculty Opinions recommendation of Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia.
8. Faculty Opinions recommendation of Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia.
9. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
10. Faculty Opinions recommendation of Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing.
11. Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation
12. An optimized SpCas9 high-fidelity variant for direct protein delivery
13. Epigenetic Regulation of β-Globin Genes and the Potential to Treat Hemoglobinopathies through Epigenome Editing
14. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
15. Faculty Opinions recommendation of Increased incidence of hematologic malignancies in SCD after HCT in adults with graft failure and mixed chimerism.
16. Targeted Base Editing Strategies for Beta-Hemoglobinopathies
17. Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing
18. Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
19. CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy
20. CRISPR's Path to the Clinic
21. Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
22. Novel genome-editing-based approaches to treat motor neuron diseases: Promises and challenges
23. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
24. 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
25. Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies
26. Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients
27. 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
28. Dysérythropoïèse dans la drépanocytose : une contribution d’origine centrale à l’anémie ?
29. Editorial: Mutation-Specific Gene Editing for Blood Disorders
30. Recent progress in genome editing for gene therapy applications: the French perspective
31. Special Issue: CRISPR's Path to the Clinic
32. Special Issue: CRISPR's Path to the Clinic
33. Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
34. Genome Editing for β-Hemoglobinopathies: Advances and Challenges
35. Base and Prime Editing Technologies for Blood Disorders
36. Reverse Phase-high-performance Liquid Chromatography (RP-HPLC) Analysis of Globin Chains from Human Erythroid Cells
37. Editing the LRF Repressor Binding Site in the γ-Globin Promoters Induces Therapeutically Relevant Fetal Hemoglobin Levels for the Treatment of β-Hemoglobinopathies
38. Fetal Hemoglobin Rescues Ineffective Erythropoiesis in Sickle Cell Disease
39. Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
40. Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins
41. GATA Factor-Mediated Gene Regulation in Human Erythropoiesis
42. Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
43. Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy
44. Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes
45. GATA factor transcriptional activity: Insights from genome‐wide binding profiles
46. Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy
47. Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies
48. Gene therapy of hemoglobinopathies: progress and future challenges
49. Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges
50. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
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