Journal
CURRENT GENE THERAPY
Volume 15, Issue 3, Pages 266-276Publisher
BENTHAM SCIENCE PUBL LTD
DOI: 10.2174/1566523215666150126122317
Keywords
Adeno-virus; amyotrophic lateral sclerosis (ALS); gene-cell therapy; glial cell-derived neuro-trophic factor (GDNF); human umbilical cord blood cell (hUCBC); human umbilical cord blood mono-nuclear cell (hUCB-MC); neural cell adhesion molecule (NCAM); vascular endothelial growth factor (VEGF); viral vector
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Funding
- Russian Science Foundation [14-15-00847]
- Russian Foundation for Basic Research (RFBR) [13-04-02057]
- RFBR [13-04-97156]
- Tatarstan Republic Academy of Sciences [13-04-97156]
- Russian Government Program of Competitive Growth of Kazan Federal University
- RF [MD-433.2013.4]
- Asklepios-Med (Hungary)
- Federal Center of Collective Use and Pharmaceutical Research and Education Center of Kazan Federal University
- Russian Science Foundation [14-15-00847] Funding Source: Russian Science Foundation
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Amyotrophic lateral sclerosis (ALS) is an incurable, chronic, fatal neuro-degenerative disease characterized by progressive loss of moto-neurons and paralysis of skeletal muscles. Reactivating dysfunctional areas is under earnest investigation utilizing various approaches. Here we present an innovative gene-cell construct aimed at reviving inert structure and function. Human umbilical cord blood cells (hUCBCs) transduced with adeno-viral vectors encoding human VEGF, GDNF and/or NCAM genes were transplanted into transgenic ALS mice models. Significant improvement in behavioral performance (open-field and grip-strength tests), as well as increased life-span was observed in rodents treated with NCAM-VEGF or NCAM-GDNF co-transfected cells. Active trans-gene expression was found in the spinal cord of ALS mice 10 weeks after delivering genetically modified hUCBCs, and cells were detectable even 5 months following transplantation. Our gene-cell therapy model yielded prominent symptomatic control and prolonged life-time in ALS. Incredible survivability of xeno-transpanted cells was also observed without any immune-suppression. These results suggest that engineered hUCBCs may offer effective gene-cell therapy in ALS.
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