Gene and stem-cell therapy for neurodegenerative diseases

Abstract

Currently there are no available effective therapies for treating neurodegenerative diseases. In animal models, the loss of neurons, caused by mutations in known genes, can be alleviated by genetically modifying target cells to increase their regeneration and viability, or by replacing dead neurons with new healthy neural cells by neurotransplantation of stem or progenitor cells, differentiated in neural pathway. Here we summarized results for gene therapy using antisense oligonucleotides, siRNA, and viral vectors. Critically reviewed advantages and disadvantages of neurotransplantation of embryonic and different adult stem cells. To date we found no reports of using genetically modified stem cells from umbilical cord blood for cell therapy of neurodegenerative diseases. We state a hypothesis, that stem cells from umbilical cord blood, genetically modified by transfection with plasmid vector, simultaneously expressing neural cell adhesion molecule L1 and vascular endothelial growth factor [VEGF), could have a significantly enhanced therapeutic effect in transgenic mice G93A, which serve as animal model for amyotrophic lateral sclerosis [ALS).

Keywords

neurodegenerative diseases / stem cells / cell therapy / gene therapy

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