Symptomatic improvement, increased life-span and sustained cell homing in amyotrophic lateral sclerosis after transplantation of human umbilical cord blood cells genetically modified with adeno-viral vectors expressing a neuro-protective factor and a neural cell adhesion molecule

Current Gene Therapy

Volumn 15, Issue 3, 2015, Pages 266-276

  Islamov, Rustem Robertovich ^a   Rizvanov, Albert Anatolyevich ^b   Mukhamedyarov, Marat Alexandrovich ^a   Salafutdinov, Ilnur Ildusovich ^b   Garanina, Ekaterina Evgenevna ^b   Fedotova, Valeria Yuryevna ^b   Solovyeva, Valeria Vladimirovna ^b   Mukhamedshina, Yana Olegovna ^b   Safiullov, Zufar Zufarovich ^a   Izmailov, Andrey Alexandrovich ^a   Guseva, Daria Sergeevna ^a,c   Zefirov, Andrey Lvovich ^a   Kiyasov, Andrey Pavlovich ^b   Palotás, András ^b,d  

^a KAZAN STATE MEDICAL UNIVERSITY   (Russian Federation)

^b KAZAN FEDERAL UNIVERSITY   (Russian Federation)

^c HANNOVER MEDICAL SCHOOL   (Germany)

^d Asklepios Med (Private Medical Practice and Research Center)   (Hungary)

Author 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

Indexed keywords

ADENOVIRUS VECTOR; COMPLEMENTARY DNA; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; VASCULOTROPIN; NERVE CELL ADHESION MOLECULE; VASCULOTROPIN A;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL BEHAVIOR; ANIMAL CELL; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL HOMING; CONTROLLED STUDY; CORD BLOOD STEM CELL TRANSPLANTATION; FEMALE; FLUORESCENCE MICROSCOPY; GENE AMPLIFICATION; GENE EXPRESSION; GENETIC RECOMBINATION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; MALE; MOLECULAR CLONING; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; REAL TIME POLYMERASE CHAIN REACTION; STEM CELL GENE THERAPY; WESTERN BLOTTING; ANIMAL; CELL TRANSPLANTATION; CYTOLOGY; DEPENDOPARVOVIRUS; FETUS BLOOD; GENE THERAPY; GENE VECTOR; GENETICS; HEK293 CELL LINE; LIFE EXPECTANCY; PATHOPHYSIOLOGY; PROCEDURES; TRANSGENIC MOUSE;

ADENOVIRIDAE; MUS; RODENTIA;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; CELL TRANSPLANTATION; DEPENDOVIRUS; FEMALE; FETAL BLOOD; GENETIC THERAPY; GENETIC VECTORS; GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR; HEK293 CELLS; HUMANS; LIFE EXPECTANCY; MALE; MICE; MICE, TRANSGENIC; NEURAL CELL ADHESION MOLECULES; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 84928944466     PISSN: 15665232     EISSN: 18755631     Source Type: Journal    
DOI: 10.2174/1566523215666150126122317     Document Type: Article

References (27)

1. Abe K. Therapeutic potential of neurotrophic factors and neural stem cells against ischemic brain injury. J Cereb Blood Flow Metab 2000; 20(10): 1393-408.
2. Tomac A, Lindqvist E, Lin LF, et al. Protection and repair of the nigrostriatal dopaminergic system by GDNF in vivo. Nature 1995; 373(6512): 335-9.
3. Chen J, Bernreuther C, Dihne M, and Schachner M. Cell adhesion molecule l1-transfected embryonic stem cells with enhanced survival support regrowth of corticospinal tract axons in mice after spinal cord injury. J Neurotrauma 2005; 22(8): 896-906.
4. Ruiz de Almodovar C, Lambrechts D, Mazzone M, Carmeliet P. Role and therapeutic potential of VEGF in the nervous system. Physiol Rev 2009; 89(2): 607-48.
5. Taha MF. Cell based-gene delivery approaches for the treatment of spinal cord injury and neurodegenerative disorders. Curr Stem Cell Res Ther 2010; 5(1): 23-36.
6. Hester ME, Foust KD, Kaspar RW, and Kaspar BK. AAV as a gene transfer vector for the treatment of neurological disorders: novel treatment thoughts for ALS. Curr Gene Ther 2009; 9(5): 428-33.
7. Federici T, Boulis NM. Gene therapy for amyotrophic lateral sclerosis. Neurobiol Dis 2012; 48(2): 236-42.
8. Peel AL, Klein RL. Adeno-associated virus vectors: activity and applications in the CNS. J Neurosci Methods 2000; 98(2): 95-104.
9. Liu SP, Fu RH, Huang SJ, et al. Stem cell applications in regenerative medicine for neurological disorders. Cell Transplant 2013; 22(4): 631-7.
10. Sun T, Ma QH. Repairing neural injuries using human umbilical cord blood. Mol Neurobiol 2013; 47(3): 938-45.
11. Mukhamedyarov MA, Rizvanov AA, Safiullov ZZ, et al. Analysis of the efficiency of gene-cell therapy in transgenic mice with amyotrophic lateral sclerosis phenotype. Bull Exp Biol Med 2013; 154(4): 558-61.
12. Rizvanov AA, Kiyasov AP, Gaziziov IM, et al. Human umbilical cord blood cells transfected with VEGF and L(1)CAM do not differentiate into neurons but transform into vascular endothelial cells and secrete neuro-trophic factors to support neuro-genesis-a novel approach in stem cell therapy. Neurochem Int 2008; 53(6-8): 389-94.
13. Rizvanov AA, Guseva DS, Salafutdinov, II, et al. Genetically modified human umbilical cord blood cells expressing vascular endothelial growth factor and fibroblast growth factor 2 differentiate into glial cells after transplantation into amyotrophic lateral sclerosis transgenic mice. Exp Biol Med (Maywood) 2011; 236(1): 91-8.
14. Mukhamedshina YO, Solovieva VV, Salafutdinov II, et al. Analysis of recombinant VEGF gene expression by genetically modified umbilical cord blood mononuclear cells in experiment in vivo. Cell Transplant Tissue Eng 2012; 7(3): 130-4. (SCOPUS Cited Publication. In Russian)
15. Takahashi H, Shibuya M. The vascular endothelial growth factor (VEGF)/VEGF receptor system and its role under physiological and pathological conditions. Clin Sci (Lond) 2005; 109(3): 227-41.
16. Bokeriya LA, Golukhova EZ, Eremeeva MV, et al. Use of human VEGF(165) gene for therapeutic angiogenesis in coronary patients: first results. Bull Exp Biol Med 2005; 140(1): 106-12.
17. Hawley TS, Herbert DJ, Eaker SS, Hawley RG. Multiparameter flow cytometry of fluorescent protein reporters. Methods Mol Biol 2004; 263: 19-38.
18. Laemmli UK. Cleavage of structural proteins during the assembly of the head of bacteriophage T4. Nature 1970; 227(5259): 680-5.
19. Carson MJ, Doose JM, Melchior B, et al. CNS immune privilege: hiding in plain sight. Immunol Rev 2006; 213: 48-65.
20. Garbuzova-Davis S, Rodrigues MC, Mirtyl S, et al. Multiple intravenous administrations of human umbilical cord blood cells benefit in a mouse model of ALS. PLoS One 2012; 7(2): e31254.
21. Kiernan MC, Vucic S, Cheah BC, et al. Amyotrophic lateral sclerosis. Lancet 2011; 377(9769): 942-55.
22. Passweg JR, Baldomero H, Bregni M, et al. Hematopoietic SCT in Europe: data and trends in 2011. Bone Marrow Transplant 2013; 48(9): 1161-7.
23. Yang WZ, Zhang Y, Wu F, et al. Safety evaluation of allogeneic umbilical cord blood mononuclear cell therapy for degenerative conditions. J Transl Med 2010; 8: 75.
24. Harris DT, Rogers I. Umbilical cord blood: a unique source of pluripotent stem cells for regenerative medicine. Curr Stem Cell Res Ther 2007; 2(4): 301-9.
25. Neuhoff S, Moers J, Rieks M, et al. Proliferation, differentiation, and cytokine secretion of human umbilical cord blood-derived mononuclear cells in vitro. Exp Hematol 2007; 35(7): 1119-31.
26. Fan CG, Zhang QJ, Tang FW, et al. Human umbilical cord blood cells express neurotrophic factors. Neurosci Lett 2005; 380(3): 322-5.
27. Krakora D, Mulcrone P, Meyer M, et al. Synergistic effects of GDNF and VEGF on lifespan and disease progression in a familial ALS rat model. Mol Ther 2013; 21(8): 1602-10.