title: Analysis of the efficiency of gene-cell therapy in transgenic mice with amyotrophic lateral sclerosis phenotype.
abstract: Amyotrophic lateral sclerosis is a neurodegenerative disease characterized by progressive death of cerebral and spinal motorneurons. Using behavioral tests we studied the efficiency of gene-cell therapy in SOD1 G93A transgenic mice receiving xenotransplantation of human umbilical cord blood mononuclear cells genetically modified with adenoviral vectors encoding vascular endothelial growth factor (VEGF) and reporter green fluorescent protein (EGFP) genes. The cells were transplanted to mice on week 27 of life (preclinical stage of the disease). Behavioral tests (open field, grip strength test) showed that transplantation of umbilical cord blood mononuclear cells expressing VEGF significantly improved the parameters of motor and explorative activity, grip strength, and animal survival. Thus, gene-cell therapy based on genetically modified mononuclear cells expressing VEGF can be efficient for the treatment of amyotrophic lateral sclerosis.
authors.affiliation: Kazan State Medical University, Ministry of Health Care of the Russian Federation, Moscow, Russia.
authors.forename: M A
authors.initials: MA
authors.lastname: Mukhamedyarov
authors.forename: A A
authors.initials: AA
authors.lastname: Rizvanov
authors.forename: Z Z
authors.initials: ZZ
authors.lastname: Safiullov
authors.forename: A A
authors.initials: AA
authors.lastname: Izmailov
authors.forename: G A
authors.initials: GA
authors.lastname: Sharifullina
authors.forename: V V
authors.initials: VV
authors.lastname: Solovieva
authors.forename: V Yu
authors.initials: VY
authors.lastname: Fedotova
authors.forename: I I
authors.initials: II
authors.lastname: Salafutdinov
authors.forename: E E
authors.initials: EE
authors.lastname: Cherenkova
authors.forename: F V
authors.initials: FV
authors.lastname: Bashirov
authors.forename: M S
authors.initials: MS
authors.lastname: Kaligin
authors.forename: S R
authors.initials: SR
authors.lastname: Abdulkhakov
authors.forename: M M
authors.initials: MM
authors.lastname: Shmarov
authors.forename: D Yu
authors.initials: DY
authors.lastname: Logunov
authors.forename: B S
authors.initials: BS
authors.lastname: Naroditsky
authors.forename: A P
authors.initials: AP
authors.lastname: Kiyasov
authors.forename: A L
authors.initials: AL
authors.lastname: Zefirov
authors.forename: R R
authors.initials: RR
authors.lastname: Islamov
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000503:physiopathology* / Q000628:therapy*; D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000379:methods; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic; D013482:Superoxide Dismutase / Q000235:genetics / Q000378:metabolism*; D014183:Transplantation, Heterologous; D042461:Vascular Endothelial Growth Factor A / Q000235:genetics / Q000378:metabolism
chemical_list: D042461:Vascular Endothelial Growth Factor A; C109415:SOD1 G93A protein; D013482:Superoxide Dismutase