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49 results on '"lentiviral vectors"'

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1. Development and validation of a droplet digital PCR method for quantifying lentiviral vector infectious titer

2. Genetic alteration of SJ293TS cells and modification of serum-free media enhances lentiviral vector production

3. Lentiviral delivered aflibercept OXB-203 for treatment of neovascular AMD

4. Optimizing and developing a scalable, chemically defined, animal component-free lentiviral vector production process in a fixed-bed bioreactor

5. Evaluation of diversity indices to estimate clonal dominance in gene therapy studies

6. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

7. Process intensification for lentiviral vector manufacturing using tangential flow depth filtration

8. Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy

9. Physiological lentiviral vectors for the generation of improved CAR-T cells

10. Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

11. Targeting transgenic proteins to alpha granules for platelet-directed gene therapy

12. Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment

13. Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy

14. Induced dendritic cells co-expressing GM-CSF/IFN-α/tWT1 priming T and B cells and automated manufacturing to boost GvL

15. A human surfactant B deficiency air-liquid interface cell culture model suitable for gene therapy applications

16. Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering

17. Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency

18. Lentiviral Vector Production Titer Is Not Limited in HEK293T by Induced Intracellular Innate Immunity

19. The C-terminal domain of LRRK2 with the G2019S mutation is sufficient to produce neurodegeneration of dopaminergic neurons in vivo

20. Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis

21. Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

22. The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes

23. Formation of hippocampal mHTT aggregates leads to impaired spatial memory, hippocampal activation and adult neurogenesis

24. Genetic Engineering and Manufacturing of Hematopoietic Stem Cells

25. Recent advances in hematopoietic gene therapy for genetic disorders.

26. Vpx mediated degradation of SAMHD1 has only a very limited effect on lentiviral transduction rate in ex vivo cultured HSPCs

27. Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy

28. Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering

29. Improved GaLV-TR Glycoproteins to Pseudotype Lentiviral Vectors: Impact of Viral Protease Activity in the Production of LV Pseudotypes

30. Cytoplasmic gelsolin increases mitochondrial activity and reduces Aβ burden in a mouse model of Alzheimer's disease

31. Comparison Between Several Integrase-defective Lentiviral Vectors Reveals Increased Integration of an HIV Vector Bearing a D167H Mutant

32. Lentiviral Vector Purification Using Genetically Encoded Biotin Mimic in Packaging Cell

33. Novel bicistronic lentiviral vectors correct β-Hexosaminidase deficiency in neural and hematopoietic stem cells and progeny: implications for in vivo and ex vivo gene therapy of GM2 gangliosidosis

34. WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation

35. Transition from serum-supplemented monolayer to serum-free suspension lentiviral vector production for generation of chimeric antigen receptor T cells.

36. Assessment of Integration-defective HIV-1 and EIAV Vectors In Vitro and In Vivo

37. Formation of hippocampal mHTT aggregates leads to impaired spatial memory, hippocampal activation and adult neurogenesis

38. New methods for disease modeling using lentiviral vectors

39. Cytoplasmic gelsolin increases mitochondrial activity and reduces Aβ burden in a mouse model of Alzheimer's disease

40. Current and innovative emerging therapies for porphyrias with hepatic involvement.

41. Manufacturing chimeric antigen receptor T cells: issues and challenges.

42. Immune modulation by genetic modification of dendritic cells with lentiviral vectors

43. Assessment of Integration-defective HIV-1 and EIAV Vectors In Vitro and In Vivo

44. Closed-system manufacturing of CD19 and dual-targeted CD20/19 chimeric antigen receptor T cells using the CliniMACS Prodigy device at an academic medical center.

45. Preclinical studies for a phase 1 clinical trial of autologous hematopoietic stem cell gene therapy for sickle cell disease.

46. Tumor targeting by lentiviral vectors combined with magnetic nanoparticles in mice.

47. Human Wharton's jelly-derived mesenchymal stromal cells engineered to secrete Epstein-Barr virus interleukin-10 show enhanced immunosuppressive properties.

48. Vpx mediated degradation of SAMHD1 has only a very limited effect on lentiviral transduction rate in ex vivo cultured HSPCs.

49. In vitro generation of glucose-responsive insulin producing cells using lentiviral based pdx-1 gene transduction of mouse (C57BL/6) mesenchymal stem cells.

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