More recently, lentiviral vectors have been used for the correction of human - thalassaemia and adrenoleukodystrophy. They have also been successfully used in the treatment of leukaemia by modifying cytotoxic T cells directed to cancer cells. Here we describe the production of clinical grade retro- ...
Vector titers in excess of 106 transducing units/ml can be harvested from the final producer clones, which can be increased to 108 TU/ml by concentration. This method will be of use to all basic and clinical investigators who wish to produce large batches of lentiviral vectors....
However, viral vectors have several critical problems in in vivo application. Especially, retroviral and lentiviral vectors have major concerns of insertional ... E Check - 《Nature》 被引量: 377发表: 2002年 Lentiviral Vectors and Gene Therapy Gene therapy was conceived during the early and mid ...
Here, we have investigated factors influencing the extent of lentiviral vector (LV) shedding upon ex vivo transduction of human hematopoietic stem and progenitor cells. Our results indicate that, although vector carry-over is detectable when using laboratory-grade vector stocks, the use of clinical-...
Biosafety Challenges for Use of Lentiviral Vectors in Gene Therapy Lentiviral vectors are promising tools for the genetic modification of cells in biomedical research and gene therapy. Their use in recent clinical trials f... A Schambach,U Modlich,M Rothe - 《Current Gene Therapy》 被引量: 73发...
Development of a replication-competent lentivirus assay for dendritic cell-targeting lentiviral vectors It is a current regulatory requirement to demonstrate absence of detectable replication-competent lentivirus (RCL) in lentiviral vector products prior to use in clinical trials. Immune Design previously des...
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of NK cell therapies include the use of allogenic cell sources, off-the-shelf availability, and no risk of graft-versus-host disease (GvHD). Allogeneic NK cell therapies have reached the clinical stage as ex vivo expanded and differentiated non-engineered cells, as chimeric antigen receptor (...
It is anticipated that the use of AAV9 or similar AAV capsids will broaden the application of gene therapy to more CNS disorders in the near future. However, whereas AAV9 has greatly expanded the ability to treat a larger number of CNS disorders, it still targets a minority of cells ...
Another group established CD19/22 dual targeting CAR T cells by transducing T cells with two separate lentiviral vectors that direct the expression of two separate CARs targeting CD19 and CD22. This approach led to a cocktail of three distinct populations of CAR T cells (anti-CD19, anti-CD...