693 results on '"Cathomen, Toni"'
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2. Effective genome editing with an enhanced ISDra2 TnpB system and deep learning-predicted ωRNAs
3. Gene editing of NCF1 loci is associated with homologous recombination and chromosomal rearrangements
4. CRISPR/Cas9 editing of NKG2A improves the efficacy of primary CD33-directed chimeric antigen receptor natural killer cells
5. Hypoxic extracellular vesicles from hiPSCs protect cardiomyocytes from oxidative damage by transferring antioxidant proteins and enhancing Akt/Erk/NRF2 signaling
6. Deciphering bat influenza H18N11 infection dynamics in male Jamaican fruit bats on a single-cell level
7. Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells
8. Modulation of TCR stimulation and pifithrin-α improve the genomic safety profile of CRISPR-engineered human T cells
9. Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1
10. Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases
11. Engineering of potent CAR NK cells using non-viral Sleeping Beauty transposition from minimalistic DNA vectors
12. Integration of ζ-deficient CARs into the CD3ζ gene conveys potent cytotoxicity in T and NK cells
13. CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia
14. On- and off-target effects of paired CRISPR-Cas nickase in primary human cells
15. Hypoxia enhances anti-fibrotic properties of extracellular vesicles derived from hiPSCs via the miR302b-3p/TGFβ/SMAD2 axis
16. Gene editing of hematopoietic stem cells restores T-cell response in familial hemophagocytic lymphohistiocytosis
17. Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi
18. Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies
19. Adaptive Immunity in Genitourinary Cancers
20. Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa
21. Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques
22. Adoptive T cell therapy cures mice from active hemophagocytic lymphohistiocytosis (HLH)
23. FRI-152-YI Gene editing to treat liver metabolic disorders: harnessing paired Cas9-Nickases for efficient and safe treatment of primary hyperoxaluria type 1
24. Generating universal anti-CD19 CAR T cells with a defined memory phenotype by CRISPR/Cas9 editing and safety evaluation of the transcriptome
25. A non-viral and selection-free COL7A1 HDR approach with improved safety profile for dystrophic epidermolysis bullosa
26. Anti-CD19 CARs displayed at the surface of lentiviral vector particles promote transduction of target-expressing cells
27. Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq
28. The potential of CAR T cell therapy for prostate cancer
29. Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells
30. Automated generation of gene-edited CAR T cells at clinical scale
31. Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article.
32. PSMA-Directed CAR T Cells Combined with Low-Dose Docetaxel Treatment Induce Tumor Regression in a Prostate Cancer Xenograft Model
33. Gene editing ofCD3 epsilongene to redirect regulatory T cells for adoptive T cell transfer
34. Integration of ζ-deficient CARs into the CD3-zeta gene conveys potent cytotoxicity in T and NK cells
35. CRISPR/Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia
36. Artificial Targets: a versatile cell-free platform to characterize CAR T cell function in vitro
37. A versatile reporter system for multiplexed screening of effective epigenome editors
38. Assessing the differential impact of chronic CMV and treated HIV infection on CD8+ T-cell differentiation in a matched cohort study: is CMV the key?
39. The Human Genome Editing Race: Loosening Regulatory Standards for Commercial Advantage?
40. CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem Cells
41. Integration of ζ-deficient CARs into the CD3-zeta gene conveys potent cytotoxicity in T and NK cells
42. Cell-Based Models of ‘Cytokine Release Syndrome’ Endorse CD40L and Granulocyte–Macrophage Colony-Stimulating Factor Knockout in Chimeric Antigen Receptor T Cells as Mitigation Strategy
43. The Use and Development of TAL Effector Nucleases
44. Cas12a Ultra enables efficient genome editing in human multipotent and pluripotent stem cells
45. Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy
46. P1357: MILESTONE (MODIFYING ELANE GOLDBERG–HOGNES BOX TO INHIBIT EXPRESSION), A UNIVERSAL, SAFE AND EFFECTIVE CRISPR/CAS9N-MEDIATED GENOME EDITING STRATEGY FOR ELANE RELATED SEVERE CONGENITAL NEUTROPENIA
47. P1354: CRISPR/CAS9 GENE EDITING OF IMMUNE CHECKPOINT RECEPTOR NKG2A IMPROVES THE ANTI-LEUKEMIC EFFICACY OF PRIMARY CD33-TARGETING CAR-NK CELLS.
48. Gene editing of hematopoietic stem cells restores T cell response in familial hemophagocytic lymphohistiocytosis
49. Traceless Targeting and Isolation of Gene-Edited Immortalized Keratinocytes from Epidermolysis Bullosa Simplex Patients
50. TALEN mediated gene editing in a mouse model of Fanconi anemia
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