520 results on '"Holmes, Michael C"'
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2. In vivo partial cellular reprogramming enhances liver plasticity and regeneration
3. ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice
4. Sleeping BeautymRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency
5. Gene Correction of iPSCs from a Wiskott-Aldrich Syndrome Patient Normalizes the Lymphoid Developmental and Functional Defects
6. Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo
7. NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease
8. Promoter-Selective Properties of the TBP-Related Factor TRF1
9. Engineering hematopoietic stem cells toward a functional cure of human immunodeficiency virus infection
10. Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates
11. In vivo genome editing of the albumin locus as a platform for protein replacement therapy
12. Off-the-shelf, steroid-resistant, IL13Rα2-specific CAR T cells for treatment of glioblastoma
13. Blood flow through sutured and coupled microvascular anastomoses: A comparative computational study
14. Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4+ T cells from HIV-1 infection
15. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
16. Genome editing for scalable production of alloantigen‐free lentiviral vectors for in vivo gene therapy
17. Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients
18. Characterization of Surfactant Water Systems by X-Ray Scattering and 2H NMR
19. Novel Approaches to Controlling Transcription
20. A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR
21. Targeted Gene Knockout in Mammalian Cells by Using Engineered Zinc-Finger Nucleases
22. Targeted genome editing in human repopulating haematopoietic stem cells
23. Targeted gene addition to human mesenchymal stromal cells as a cell-based plasma-soluble protein delivery platform
24. Supplement to: Genome Editing with Zinc Finger Nuclease Modified Autologous CD4 T-cells
25. GENE EDITING OF CCR5 IN HEMATOPOIETIC STEM CELLS IN A NONHUMAN PRIMATE MODEL OF HIV/AIDS: O1.03
26. A Southern Blot Protocol to Detect Chimeric Nuclease-Mediated Gene Repair
27. Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer
28. Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells
29. In vivo genome editing restores haemostasis in a mouse model of haemophilia
30. Targeted gene therapy and cell reprogramming in Fanconi anemia
31. Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV
32. Targeted transgene integration in plant cells using designed zinc finger nucleases
33. Persistent repression of tau in the brain using engineered zinc finger protein transcription factors
34. (super 2)H NMR evidence for the formation of random mesh phases in nonionic surfactant-water systems
35. A Rapid and General Assay for Monitoring Endogenous Gene Modification
36. TRF2 associates with DREF and directs promoter-selective gene expression in Drosophila
37. AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction
38. Characterization of Surfactant Water Systems by X-Ray Scattering and 2H NMR
39. Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology
40. Highly efficient endogenous human gene correction using designed zinc-finger nucleases
41. Humanized Mouse Model of HIV-1 Latency with Enrichment of Latent Virus in PD-1 + and TIGIT + CD4 T Cells
42. Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
43. Diversifying the structure of zinc finger nucleases for high-precision genome editing
44. ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
45. Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+ Cells
46. Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major
47. Differential impact of transplantation on peripheral and tissue-associated viral reservoirs: Implications for HIV gene therapy
48. Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
49. A Potential Therapy for Beta-Thalassemia (ST-400) and Sickle Cell Disease (BIVV003)
50. Liver-based expression of the human alpha-galactosidase A gene in a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction
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