Treatment of the amyotrophic lateral sclerosis using of genetically modified umbilical cord bloodmononuclear cells in the preclinical studies

Abstract

Development of the fundamental and clinical «regenerativemedicine» is based on the progress of gene, stem cell andgene-cell biotechnologies. However, the reliable preclinicalinvestigations on animal models and more over clinical trialsstay far away from the available nowadays gene and cellconstructions. Neuroscience is one of the fast growing fieldsof knowledge in biology and medicine. Pioneer experimentsin neuroscience promises breakthrough in the innovativemethods for treatment of neurodegenerative diseases innear future. This review addresses strategies for gene-celltherapy of neurodegenerative diseases by the example ofamyotrophic lateral sclerosis. Precisely gene modification ofmononuclear fraction of umbilical cord blood cells (UCBC)by dual cassette plasmid vectors is observed. Based onour own results of transplantation of genetically modifiedUCBC overexpressing recombinant neural cell adhesionmolecule L1, vascular endothelial growth factor, fibroblastgrowth factor 2, and glial derived neurotrophic factor indifferent combinations we provide the experimental datafor usefulness of transplantation of gene modified UCBCfor treating neurodegenerative diseases. In the review wediscuss the efficacy of gene modification of UCBC not onlyfor secretion of recombinant proteins, but in increasing oftransplanted cells survivability, their migration possibilitiesand capability to differentiate in endothelial, microglial andmacroglial cell types.

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