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...
Ex Vivo Gene Therapydoi:10.1016/b978-0-12-394295-1.00001-9Lajos BaranyiVladimir SlepushkinBoro Dropulic
Ectopic osteogenesis by ex vivo gene therapy using beta tricalcium phosphate as a carrier. Injuries and other damage to large bone can result in defects that do not heal spontaneously and lead to severe functional impairment. Better therapies are......
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 ...
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 ...
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...
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...
The isolated cells were transduced with AAV2 vectors encoding eGFP, human BMP-2, and human TGF-β1 genes prior to implantation into hind limb muscles of SCID mice to assess osteogenesis in vivo. The results showed that rAMSCs were more efficiently transduced with GFP by AAV2 than by AAV6...
To investigate the efficacy of ex vivo gene therapy with self-inactivating lentiviral vectors (LVs) in correcting this complex phenotype, we used an ADA(-/-) mouse model characterized by early postnatal lethality. LV-mediated ADA gene transfer into bone marrow cells combined with low-dose ...
No effect of NGF-secreting NT2N cells on motor function deficits at 1-4 weeks post-transplantation was observed. These data suggest that NGF gene therapy using transduced NT2N neurons (as a source of delivery) may selectively improve cognitive function following TBI. 展开 ...