A genetic fusion GDNF-C fragment of tetanus toxin prolongs survival in a symptomatic mouse ALS model

Restorative Neurology and Neuroscience

Volumn 26, Issue 6, 2008, Pages 459-465

  Ciriza, J ^a   Moreno Igoa, M ^a   Calvo, A C ^a   Yague, G ^a   Palacio, J ^a   Miana Mena, F J ^a   Munoz M J ^a   Zaragoza, P ^a   Brulet P ^b   Pinzolas, R Y Osta ^a  

^a UNIVERSITY OF ZARAGOZA   (Spain)

^b CNRS   (France)

Author keywords

Amyotrophic lateral Sclerosis; apoptosis; fragment C of tetanus toxin; neurodegenerative diseases; neurotrophic factor; SOD1G93A animal model; survival

Indexed keywords

CASPASE 3; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; HYBRID PROTEIN; ONCOPROTEIN; PEPTIDE FRAGMENT; SOD1 G93A PROTEIN; SUPEROXIDE DISMUTASE; TETANUS TOXIN; TETANUS TOXIN FRAGMENT C;

AMYOTROPHIC LATERAL SCLEROSIS; ANALYSIS OF VARIANCE; ANIMAL; APOPTOSIS; ARTICLE; BRAIN CORTEX; CELL CULTURE; CYTOLOGY; DISEASE MODEL; DOSE RESPONSE; DRUG EFFECT; GENE TRANSFER; GENETIC TRANSFECTION; GENETICS; HUMAN; METABOLISM; MICROBIOLOGY; MOUSE; NERVE CELL; NEUROBLASTOMA; SURVIVAL; TRANSGENIC MOUSE;

AMYOTROPHIC LATERAL SCLEROSIS; ANALYSIS OF VARIANCE; ANIMALS; APOPTOSIS; CASPASE 3; CELLS, CULTURED; CEREBRAL CORTEX; DISEASE MODELS, ANIMAL; DOSE-RESPONSE RELATIONSHIP, DRUG; GENE TRANSFER TECHNIQUES; GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR; HUMANS; MICE; MICE, TRANSGENIC; NEUROBLASTOMA; NEURONS; ONCOGENE PROTEIN V-AKT; PEPTIDE FRAGMENTS; RECOMBINANT FUSION PROTEINS; SUPEROXIDE DISMUTASE; SURVIVAL ANALYSIS; TETANUS TOXIN; TRANSFECTION;

EID: 64049096441     PISSN: 09226028     EISSN: 18783627     Source Type: Journal    
DOI: None     Document Type: Article

References (30)

1. Andjelkovíc, M., Alessi, D. R., Meier, R., Fernandez, A., Lamb, N. J., Frech, M., Cron, P., Cohen, P., Lucocq, J. M., Hemmings, B. A. (1997). Role of translocation in the activation and function of protein kinase. B. J Biol Chem, 272(50), 31515-31524.
2. Alisky, J. M.&Davidson, B. L. (2000). Gene therapy for amyotrophic lateral sclerosis and other motor neuron diseases. Hum Gene Ther., 11(17), 2315-2329.
3. Bordet, T., Castelnau-Ptakhine, L., Fauchereau, F., Friocourt, G., Kahn, A., Haase, G. (2001). Neuronal targeting of cardiotrophin-1 by coupling with tetanus toxin C fragment. Mol Cell Neurosci., 17, 842-854.
4. Coen, L., Osta, R., Maury, M., Brulet, P. (1997). Construction of hybrid proteins that migrate retrogradely and transynaptically into the central nervous system. Proc Natl Acad Sci USA., 94, 9400-9405.
5. Figueiredo, D. M., Hallewell, R. A., Chen, L. L., Fairweather, N. F., Dougan, G., Savitt, J. M., Parks, D. A., Fishman, P. S. (1997). Delivery of recombinant tetanus-superoxide dismutase proteins to central nervous system neurons by retrograde axonal transport. Exp Neurol, 145(2), 546-554.
6. Fishman, P. S. and J. M. Savitt (1989). Transsynaptic transfer of retrogradely transported tetanus protein- peroxidase conjugates.Exp Neurol, 106(2), 197-203.
7. Francis, J. W., Hosler, B. A., Brown, R. H., Fishman, P. S. (1995). CuZn superoxide dismutase (SOD-1): tetanus toxin fragment C hybrid protein for targeted delivery of SOD-1 to neuronal cells. J Biol Chem, 270(25), 15434-15442.
8. Francis, J.W., Figueiredo, D., VanderSpek, J. C., Ayala, L. M., Kim, Y. S., Remington, M. P., Young, P. J., Lorson, C. L., Ikebe, S., Fishman, P. S., Brown, R. H. (2004). A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMNprotein to neurons. Brain Res., 995(1), 84-96.
9. Gil, C., Chaib-Oukadour, I., Aguilera, J.(2003). C-terminal fragment of the tetanus toxin heavy chain activates Akt and MEK/ERK signaling pathways in a Trk receptor-dependent manner in cultured cortical neurons. Biochem J, 373, 613-620.
10. Goldberg, R. L., Costa, T., Habig, W. H., Kohn, L. D., Hardegree, M. C. (1981). Characterization of fragment C and tetanus toxin binding to rat brain membranes. Mol Pharmacol, 20(3), 565-570.
11. Gurney, M. E., Pu, H., Chiu, A. Y., Dal Canto, M. C., Polchow, C. Y., Alexander, D. D., Caliendo, J., Hentati, A., Kwon, Y. W., Deng, H. X. (1994). Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation. Science, 264, 1772-1775.
12. Haase, G., Kennel, P., Pettmann, B., Vigne, E., Akli, S., Revah, F., Schmalbruch, H., Kahn, A. (1997). Gene therapy of murine motor neuron disease using adenoviral vectors for neurotrophic factors. Nat Med, 3(4), 429-36.
13. Henderson, C. E., Phillips, H. S., Pollock, R. A., Davies, A. M., Lemeulle, C., Armanini, M., Simmons, L., Moffet, B., Vandlen, R. A., Simpson, L. C. (1994). GDNF: a potent survival factor for motoneurons present in peripheral nerve and muscle. Science, 266, 1062-1064.
14. Herreros, J., Lalli, G.&Schiavo, G. (2000) C-terminal half of tetanus toxin fragment C is sufficient for neuronal binding and interaction with a putative protein receptor. Biochem J, 347(1), 199-204.
15. Kaspar, B. K., Llado, J., Sherkat, N., Rothstein, J. D., Gage, F. H. (2003). Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science, 301, 839-842.
16. Kennedy, S. G., Kandel, E. S., Cross, T. K., Hay, N. (1999). Akt/Protein kinase B inhibits cell death by preventing the release of cytochrome c from mitochondria. Mol Cell Biol, 19(8), 5800-5810.
17. Larsen, K. E., Benn, S. C., Ay, I., Chian, R. J., Celia, S. A., Remington, M. P., Bejarano, M., Liu, M., Ross, J., Carmillo, P., Sah, D., Phillips, K. A., Sulzer, D., Pepinsky, R. B., Fishman, P. S., Brown, R. H., Francis, J. W. (2006). A glial cell line-derived neurotrophic factor (GDNF): tetanus toxin fragment C protein conjugate improves delivery of GDNF to spinal cord motor neurons in mice. Brain Res, 1120, 1-12.
18. Li, W., Brakefield, D., Pan, Y., Hunter, D., Myckatyn, T. M., Parsadanian, A. (2007). Muscle-derived but not centrally derived transgene GDNF is neuroprotective in SOD1G93A mouse model of ALS. Exp Neurol, 203, 457-71.
19. Matthews, C. C., Figueiredo, D. M., Wollack, J. B., Fairweather, N. F., Dougan, G., Hallewell, R. A., Cadet, J. L., Fishman, P. S. (2000). Protective effect of supplemental superoxide dismutase on survival of neuronal cells during starvation. Requirement for cytosolic distribution. J Mol Neurosci., 14(3), 155-66.
20. Meier, R., Alessi, D. R., Cron, P., Andjelkovíc, M., Hemmings, B. A. (1997). Mitogenic activation, phosphorylation, and nuclear translocation of protein kinase beta. J Biol Chem, 272(48), 30491-30497.
21. Miana-Mena, F. J., Roux, S., Benichou, J. C., Osta, R., Brulet, P. (2002). Neuronal activity-dependent membrane traffic at the neuromuscular junction. Proc Natl Acad Sci USA. 99, 3234- 3239.
22. Miana-Mena, F. J., Muñoz, M. J., Ciriza, J., Soria, J., Br̂ulet, P., Zaragoza, P., Osta, R. (2003). Fragment C tetanus toxin: a putative activity-dependent neuroanatomical tracer. Acta Neurobiol Exp (Wars), 63(3), 211-218.
23. Miana-Mena, F. J., Mnoz, M. J., Roux, S., Ciriza, J., Zaragoza, P., Br̂ulet, P., Osta, R.(2004). A non-viral vector for targeting gene therapy to motorneurons in the CNS. Neurodegener Dis, 1(2-3), 101-108.
24. Nicklas, W., Baneux, P., Boot, R., Decelle, T., Deeny, A. A., Fumanelli, M., Illgen-Wilcke, B. (FELASA) Working group on health monitoring of rodent and rabbit colonies) (2002). Recommendations for the health monitoring of rodent and rabbit colonies in breeding and experimental units. Lab Anim, 36, 20-42.
25. Oppenheim, R. W., Houenou, L. J., Johnson, J. E., Lin, L. F., Li, L., Lo, A. C., Newsome, A. L., Prevette, D. M., Wang, S. (1995). Developing motor neurons rescued from programmed and axotomy-induced cell death by GDNF. Nature, 373, 344- 346.
26. Pezeshki, G., Franke, B., Engele, J. (2001). Evidence for a ligandspecific signaling through GFRalpha-1, but not GFRalpha-2, in the absence of Ret. J Neurosci Res, 66(3), 390-395.
27. Sagot, Y., Tan, S. A., Hammang, J. P., Aebischer, P., Kato, A. C. (1996). GDNF slows loss of motoneurons but not axonal degeneration or premature death of pmn/pmn mice. J Neurosci, 16(7), 2335-2341.
28. Storkebaum, E., Lambrechts, D., Dewerchin, M., Moreno-Murciano, M. P., Appelmans, S., Oh, H., Van Damme, P., Rutten, B., Man, W. Y., De Mol, M., Wyns, S., Manka, D., Vermeulen, K., Van DenBosch, L., Mertens, N., Schmitz, C., Robberecht, W., Conway, E. M., Collen, D., Moons, L., Carmeliet, P. (2005). Treatment of motorneuron degeneration by intracerebroventricular delivery of VEGF in a rat model of ALS. Nat Neurosci, 8, 85-92.
29. Wang, H. J., Cao, J. P.,Yu, J. K., Gao, D. S. (2007). Role of PI3-K/Akt pathway and its effect on glial cell line-derived neurotrophic factor in midbrain dopamine cells. Acta Pharmacol Sin, 28, 166-172.
30. Yan, Q., Matheson, C., Lopez, O. T. (1995). In vivo neurotrophic effects of GDNF on neonatal and adult facial motor neurons. Nature, 373, 341-344.