239 results on '"Mussolino, Claudio"'
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2. Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing
3. Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq
4. Automated generation of gene-edited CAR T cells at clinical scale
5. A versatile reporter system for multiplexed screening of effective epigenome editors
6. CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem Cells
7. Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets.
8. Multimodal HLA-I genotype regulation by human cytomegalovirus US10 and resulting surface patterning.
9. 3071 – EPIGENOME EDITING RESULTS IN SUCCESSFUL TRANSIENT REACTIVATION OF GAMMA GLOBIN GENE EXPRESSION AND OFFERS AN INNOVATIVE THERAPEUTIC OPTION FOR Β-HEMOGLOBINOPATHIES
10. Spotlight on gene therapy in Germany
11. Designer Effectors for Editing and Regulating Complex Genomes
12. Cas12a Ultra enables efficient genome editing in human multipotent and pluripotent stem cells
13. Traceless Targeting and Isolation of Gene-Edited Immortalized Keratinocytes from Epidermolysis Bullosa Simplex Patients
14. TALEN mediated gene editing in a mouse model of Fanconi anemia
15. The Use and Development of TAL Effector Nucleases
16. Streptococcus thermophilus CRISPR-Cas9 Systems Enable Specific Editing of the Human Genome
17. Editing CCR5: A Novel Approach to HIV Gene Therapy
18. Fast and Quantitative Identification of Ex Vivo Precise Genome Targeting-Induced Indel Events by IDAA
19. Inhibition of replication of hepatitis B virus using transcriptional repressors that target the viral DNA
20. A novel Cas9 fusion protein promotes targeted genome editing with reduced mutational burden in primary human cells
21. High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis
22. Proven and novel strategies for efficient editing of the human genome
23. Multimodal HLA-I genotype regulation by human cytomegalovirus US10 and resulting surface patterning
24. Generation of TALE-Based Designer Epigenome Modifiers
25. Erratum to: Delivery of Designer Epigenome Modifiers into Primary Human T Cells
26. Delivery of Designer Epigenome Modifiers into Primary Human T Cells
27. Allele-Specific Disruption of a Common STAT3 Autosomal Dominant Allele Is Not Sufficient to Restore Downstream Signaling in Patient-Derived T Cells
28. Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases
29. TALE nucleases: tailored genome engineering made easy
30. Transcription Activator-Like Effector (TALE) Nucleases and Repressor TALEs for Antiviral Gene Therapy
31. Genome Editing With TALEN, CRISPR-Cas9 and CRISPR-Cas12a in Combination With AAV6 Homology Donor Restores T Cell Function for XLP
32. Recent Approaches for Manipulating Globin Gene Expression in Treating Hemoglobinopathies
33. Zinc‐finger‐based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa
34. TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity
35. Preclinical Evaluation of a Novel TALEN Targeting CCR5 Confirms Efficacy and Safety in Conferring Resistance to HIV-1 Infection
36. Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells
37. Inhibition of Ocular Neovascularization by Hedgehog Blockade
38. Engineered zinc finger nickases induce homology-directed repair with reduced mutagenic effects
39. Efficient in vivo genome editing mediated by stem cells-derived extracellular vesicles carrying designer nucleases
40. HIV Gene Therapy: An Update
41. A novel TALE nuclease scaffold enables high genome editing activity in combination with low toxicity
42. Preclinical Evaluation of a Novel TALEN Targeting CCR5 Confirms Efficacy and Safety in Conferring Resistance to HIV‐1 Infection
43. DNA Damage: From Threat to Treatment
44. Clinically relevant CCR5 editing in primary CD4+ T cells with TALEN confers resistance to HIV-1 infection
45. Genome Editing of the SNAI1 Gene in Rhabdomyosarcoma: A Novel Model for Studies of Its Role
46. Gene Therapy "Made in Germany": A Historical Perspective, Analysis of the Status Quo, and Recommendations for Action by the German Society for Gene Therapy.
47. MOESM1 of Inhibition of replication of hepatitis B virus using transcriptional repressors that target the viral DNA
48. Chimerization Enables Gene Synthesis and Lentiviral Delivery of Customizable TALE-Based Effectors
49. Quantitative Evaluation of Chromosomal Rearrangements in Primary Gene-Edited Human Stem Cells by Preclinical CAST-Seq
50. The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
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