Human umbilical cord blood mononuclear cells transfected with dual cassette plasmids(vegf + neurotrophic factor) for the treatment of amyotrophic lateral sclerosis

Abstract

To increase the viability of neural cells in neurodegenerativediseases, after neurotraumas and ischemic strokes themost important neurotrophic and neuroprotective factors,which can be used as therapeutic agents were identifiedin long-term studies in vitro and in vivo. These includebrain-derived neurotrophic factor (BDNF), glial-derivedneurotrophic factor (GDNF), insulin-like growth factor (IGF)and vascular endothelial growth factor (VEGF). One of thepromising ways of the delivery of supporting neuron survivalfactors is considered to be transplantation of geneticallymodified cells overexpressing recombinant therapeuticgenes. This article describes generation of cellular deliveryvectors of therapeutic genes - human umbilical cord bloodmononuclear cells genetically modified by dual cassetteplasmids, expressing two therapeutic genes. Efficiency oftransgene expression was confirmed in vitro using RT-PCR.Analysis of survival, migration, and phenotype of geneticallymodified cells was performed 2 weeks after transplantationinto transgenic mice with amyotrophic lateral sclerosisphenotype.

Keywords

gene-cell therapy / amyotrophic lateral sclerosis / human umbilical cord blood mononuclear cells / vascular endothelial growth factor / basic fibroblast growth factor / glial cell-derived neurotrophic factor / neural adhesion molecule L1

References

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