As LEKTI is a secreted protein, the genetically modified graft may provide not only an immediate local protective barrier, but also act as a source of secreted LEKTI providing a generalized benefit following ex-vivo gene therapy. 机译:Netherton综合征(NS)是一种令人衰弱的先天性皮肤疾病,由编码...
In vivo and ex vivo gene therapy strategies to treat tumors using adenovirus gene transfer vectors. Cancer Chemother Pharmacol 1999; 43 Suppl: S90-S99In vivo and ex vivo gene therapy strategies to treat tumors using adenovirus gene transfer vectors[J] . Ronald G. Crystal.Cancer Chemotherapy and...
Chapter 1-Ex Vivo Gene Therapy: Utilization of Genetic Vectors for the Generation of Genetically Modified Cell Products for Therapy. Baranyi L,Slepushkin V,Dropulic B. Gene Therapy of Cancer . 2014Baranyi L, Slepushkin V, Dropulic B. Ex vivo gene therapy: utilization of genetic vectors for ...
A therapeutic option for monogenic disorders is gene therapy with ex vivo-transduced autologous hematopoietic stem cells (HSCs). Safety or efficacy studies of ex vivo-modified HSCs are conducted in humanized mouse models after ablation of the murine bone marrow and transfer of human CD34+ HSCs. En...
SummaryBackgroundCell-cycle blockade by ex-vivo gene therapy of experimental vein grafts inhibits the neointimal hyperplasia and subsequent accelerated atherosclerosis that lead to human bypass-graft failure. In a prospective, randomised, controlled trial, we investigated the safety and biological efficacy ...
Until recently, progress in ex vivo gene therapy (GT) for human immunodeficiency virus-1 (HIV-1) treatment has been incremental. Long-term HIV-1 remission in a patient who received a heterologous stem cell transplant for acquired immunodeficiency syndrome-related lymphoma from a CCR5−/– donor...
Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Familial hypercholesterolemia (FH) is an inherited disorder in humans that is caused by a deficiency of low density lipoprotein receptors (LDLRs). An anima... Chowdhury,M Grossman,S Gupta,... - ...
Ex vivo autologous hematopoietic stem cell (HSC) gene therapy may be an alternative to haploidentical BMT. In a French trial, 9 of 10 XSCID infants had immune reconstitution following ex vivo transduction of autologous HSC with a retroviral vector encoding γc. Selective development and expansion ...
Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invent...
The ex vivo gene therapy could be a promising tool for the treatment of osteoporotic fractures and critically sized defects. The reduced number of complications (nonunions, loss of reduction, and fragment dislocation), shortening of hospitalization period, and improvement of bone strength are decisive...