| Autor: |
Islamov RR, Rizvanov AA, Mukhamedyarov MA, Salafutdinov II, Garanina EE, Fedotova VY, Solovyeva VV, Mukhamedshina YO, Safiullov ZZ, Izmailov AA, Guseva DS, Zefirov AL, Kiyasov AP, Palotas A; Department of Medical Biology and Genetics, Kazan State Medical University, ul. Butlerova 49, R- 420012 Kazan, Russia. islamru@yahoo.com. |
| Jazyk: |
angličtina |
| Zdroj: |
Current gene therapy [Curr Gene Ther] 2015; Vol. 15 (3), pp. 266-76. |
| DOI: |
10.2174/1566523215666150126122317 |
| Abstrakt: |
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. |
| Databáze: |
MEDLINE |
| Externí odkaz: |
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