Treatment with trkC agonist antibodies delays disease progression in neuromuscular degeneration (nmd) mice

Human Molecular Genetics

Volumn 14, Issue 13, 2005, Pages 1825-1837

  Ruiz, Rocio ^a   Lin, John ^b   Forgie, Alison ^b   Foletti, Davide ^b   Shelton, David ^b   Rosenthal, Arnon ^b   Tabares, Lucia ^a  

^a UNIVERSITY OF SEVILLE   (Spain)

^b Rinat Neuroscience Corporation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY 2256; TYROSINE KINASE RECEPTOR; TYROSINE KINASE RECEPTOR C; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DEGENERATIVE DISEASE; DISEASE COURSE; DRUG EFFICACY; DRUG HALF LIFE; DRUG MECHANISM; ELECTROPHYSIOLOGY; FEMALE; IN VIVO STUDY; MALE; MOTOR PERFORMANCE; MOUSE; NEUROMUSCULAR DISEASE; NONHUMAN; PRIORITY JOURNAL; RESPIRATORY DISTRESS; SPINAL MUSCULAR ATROPHY; SURVIVAL;

ANIMALS; ANTIBODIES, MONOCLONAL; DISEASE MODELS, ANIMAL; HUMANS; MICE; MICE, NEUROLOGIC MUTANTS; MUSCULAR ATROPHY, SPINAL; RECEPTOR, TRKC; SIGNAL TRANSDUCTION;

EID: 26444606418     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddi189     Document Type: Article

References (52)

1. Lefebvre, S., Burglen, L., Reboullet, S., Clermont, O., Burlet, P., Viollet, L., Benichou, B., Cruaud, C., Millasseau, P., Zeviani, M. et al. (1995) Identification and characterization of a spinal muscular atrophy-determining gene. Cell, 80, 155-165.
2. Grohmann, K., Wienker, T.F., Saar, K., Rudnik-Schoneborn, S., Stoltenburg-Didinger, G., Rossi, R., Novelli, G., Nurnberg, G., Pfeufer, A., Wirth, B. et al. (1999) Diaphragmatic spinal muscular atrophy with respiratory distress is heterogeneous, and one form is linked to chromosome 11g13-q21. Am. J. Hum. Genet., 65, 1459-1462.
3. Grohmann, K., Schuelke, M., Diers, A., Hoffmann, K., Lucke, B., Adams, C., Bertini, E., Leonhardt-Horti, H., Muntoni, F., Ouvrier, R. et al. (2001) Mutations in the gene encoding immunoglobulin mu-binding protein 2 cause spinal muscular atrophy with respiratory distress type 1. Nat. Genet., 29, 75-77.
4. Grohmann, K., Varon, R., Stolz, P., Schuelke, M., Janetzki, C., Bertini, E., Bushby, K., Muntoni, F., Ouvrier, R., van Maldergem, L. et al. (2003) Infantile spinal muscular atrophy with respiratory distress type 1 (SMARD1). Ann. Neurol., 54, 719-724.
5. Pitt, M., Houlden, H., Jacobs, J., Mok, Q., Harding, B., Reilly, M. and Surtees, R. (2003) Severe infantile neuropathy with diaphragmatic weakness and its relationship to SMARD1. Brain, 126, 2682-2692.
6. Wirth, B. (2000) An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA). Hum. Mutat., 15, 228-237.
7. Mercuri, E., Bertini, E., Messina, S., Pelliccioni, M., D'Amico, A., Colitto, F., Mirabella, M., Tiziano, F.D., Vitali, T., Angelozzi, C. et al. (2004) Pilot trial of phenylbutyrate in spinal muscular atrophy. Neuromuscul. Disord., 14, 130-135.
8. Cook, S.A., Johnson, K.R., Bronson, R.T. and Davisson, M.T. (1995) Neuromuscular degeneration (nmd): A mutation on mouse chromosome 19 that causes motor neuron degeneration. Mamm. Genome, 6, 187-191.
9. Cox, G.A., Mahaffey, C.L. and Frankel, W.N. (1998) Identification of the mouse neuromuscular degeneration gene and mapping of a second site suppressor allele. Neuron, 21, 1327-1337.
10. Maddatu, T.P., Garvey, S.M., Schroeder, D.G., Hampton, T.G. and Cox, G.A. (2004) Transgenic rescue of neurogenic atrophy in the nmd mouse reveals a role for Ighmbp2 in dilated cardiomyopathy. Hum. Mol. Genet., 13, 1105-1115.
11. Grohmann, K., Rossoll, W., Kobsar, I., Holtmann, B., Jablonka, S., Wessig, C., Stoltenburg-Didinger, G., Fischer, U., Hubner, C., Martini, R. et al. (2004) Characterization of Ighmbp2 in motor neurons and implications for the pathomechanism in a mouse model of human spinal muscular atrophy with respiratory distress type 1 (SMARD1). Hum. Mol. Genet., 13, 2031-2042.
12. Arakawa, Y., Sendtner, M. and Thoenen, H. (1990) Survival effect of ciliary neurotrophic factor (CNTF) on chick embryonic motoneurons in culture: Comparison with other neurotrophic factors and cytokines. J. Neurosci., 10, 3507-3515.
13. Sendtner, M., Kreutzberg, G.W. and Thoenen, H. (1990) Ciliary neurotrophic factor prevents the degeneration of motor neurons after axotomy. Nature, 345, 440-441.
14. Sendtner, M., Schmalbruch, H., Stockli, K.A., Carroll, P., Kreutzberg, G. W. and Thoenen, H. (1992) Ciliary neurotrophic factor prevents degeneration of motor neurons in mouse mutant progressive motor neuronopathy. Nature, 358, 502-524.
15. Henderson, C.E., Camu, W., Mettling, C., Gouin, A., Poulsen, K., Karihaloo, M., Rullamas, J., Evans, T., McMahon, S.B., Armanini, M.P. et al. (1993) Neurotrophins promote motor neuron survival and are present in embryonic limb bud. Nature, 363, 266-270.
16. Sadick, M.D., Galloway, A., Shelton, D., Hale, V., Weck, S., Anicetti, V. and Wong, W.L. (1997) Analysis of neurotrophin/receptor interactions with a gD-flag-modified quantitative kinase receptor activation (gD.KIRA) enzyme-linked immunosorbent assay. Exp. Cell Res., 234, 354-361.
17. Poduslo, J.F. and Curran, G.L. (1996) Permeability at the blood-brain and blood-nerve barriers of the neurotrophic factors: NGF, CNTF, NT-3, BDNF. Brain Res. Mol. Brain Res., 36, 280-286.
18. Fernandez-Chacon, R., Wolfel, M., Nishimune, H., Tabares, L., Schmitz, F., Castellano-Munoz, M., Rosenmund, C.. Montesinos, M.L., Sanes, J.R., Schneggenburger, R. et al. (2004) The synaptic vesicle protein CSP alpha prevents presynaptic degeneration. Neuron, 42, 237-251.
19. Sagot, Y., Vejsada, R. and Kato, A.C. (1997) Clinical and molecular aspects of motoneurone diseases: Animal models, neurotrophic factors and Bcl-2 oncoprotein. Trends Pharmacol Sci., 18, 330-337.
20. Mitsumoto, H., Ikeda, K., Klinkosz, B., Cedarbaum, J.M., Wong, V. and Lindsay, R.M. (1994) Arrest of motor neuron disease in wobbler mice cotreated with CNTF and BDNF. Science, 265, 1107-1110.
21. Miller, R.G., Petajan, J.H., Bryan, W.W., Armon, C., Barohn, R.J., Goodpasture, J.C., Hoagland, R.J., Parry, G.J., Ross, M.A. and Stromatt, S.C. (1996) A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. rhCNTF ALS Study Group. Ann. Neurol., 39, 256-260.
22. Thoenen, H. and Sendtner, M. (2002) Neurotrophins: From enthusiastic expectations through sobering experiences to rational therapeutic approaches. Nat. Neurosci., 5 (suppl.), 1046-1050.
23. Diener, P.S. and Bregman, B.S. (1994) Neurotrophic factors prevent the death of CNS neurons after spinal cord lesions in newborn rats. Neuroreport, 5, 1913-1917.
24. Vejsada, R., Sagot, Y. and Kato, A.C. (1995) Quantitative comparison of the transient rescue effects of neurotrophic factors on axotomized motoneurons in vivo. Eur. J. Neurosci., 7, 108-115.
25. Sagot, Y., Tan, S.A., Baetge, E., Schmalbruch, H., Kato, A.C. and Aebischer, P. (1995) Polymer encapsulated cell lines genetically engineered to release ciliary neurotrophic factor can slow down progressive motor neuronopathy in the mouse. Eur. J. Neurosci., 7, 1313-1322.
26. Haase, G., Kennel, P., Pettmann, B., Vigne, E., Akli, S., Revah, F., Schmalbruch, H. and Kahn, A. (1997) Gene therapy of murine motor neuron disease using adenoviral vectors for neurotrophic factors. Nat. Med. 3, 429-436.
27. Bibel, M. and Barde, Y.A. (2000) Neurotrophins: Key regulators of cell fate and cell shape in the vertebrate nervous system. Genes Dev., 14, 2919-2937.
28. Knusel, B., Gao, H., Okazaki, T., Yoshida, T., Mori, N., Hefti, F. and Kaplan, D.R. (1997) Ligand-induced down-regulation of Trk messenger RNA, protein and tyrosine phosphorylation in rat cortical neurons. Neuroscience, 78, 851-862.
29. Sendtner, M. (1997) Gene therapy for motor neuron disease. Nat. Med. 3, 380-381.
30. Oppenheim, R.W. (1996) Neurotrophic survival molecules for motoneurons: an embarrassment of riches. Neuron, 17, 195-197.
31. Yan, Q., Elliott, J.L., Matheson, C., Sun, J., Zhang, L., Mu, X., Rex, K.L. and Snider, W.D. (1993) Influences of neurotrophins on mammalian motoneurons in vivo. J. Neurobiol., 24, 1555-1577.
32. Duberley, R.M., Johnson, I.P., Anand, P., Leigh, P.N. and Cairns, N.J. (1997) Neurotrophin-3-like immunoreactivity and Trk C expression in human spinal motoneurones in amyotrophic lateral sclerosis. J. Neurol. Sci., 148, 33-40.
33. Wang, T., Xie, K. and Lu, B. (1995) Neurotrophins promote maturation of developing neuromuscular synapses. J. Neurosci., 15, 4796-4805.
34. Liou, J.C. and Fu, W.M. (1997) Regulation of quantal secretion from developing motoneurons by postsynaptic activity-dependent release of NT-3. J. Neurosci., 17, 2459-2468.
35. Sendtner, M. (1998) Neurotrophic factors: Effects in modulating properties of the neuromuscular endplate. Cytokine Growth Factor Rev. 9, 1-7.
36. Poo, M.M. (2001) Neurotrophins as synaptic modulators. Nat. Rev. Neurosci., 2, 24-32.
37. Lohof, A.M., Ip, N.Y. and Poo, M.M. (1993) Potentiation of developing neuromuscular synapses by the neurotrophins NT-3 and BDNF. Nature, 363, 350-353.
38. Kang, H. and Schuman, E.M. (1995) Long-lasting neurotrophin-induced enhancement of synaptic transmission in the adult hippocampus. Science, 267, 1658-1662.
39. Loeb, J.A. and Fischbach, G.D. (1997) Neurotrophic factors increase neuregulin expression in embryonic ventral spinal cord neurons. J. Neurosci., 17, 1416-1424.
40. Griesbeck, O., Parsadanian, A.S., Sendtner, M. and Thoenen, H. (1995) Expression of neurotrophins in skeletal muscle: Quantitative comparison and significance for motoneuron survival and maintenance of function. J. Neurosci. Res., 42, 21-33.
41. 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. et al. (1994) GDNF: A potent survival factor for motoneurons present in peripheral nerve and muscle. Science, 266, 1062-1064.
42. Koliatsos, V.E., Clatterbuck, R.E., Winslow, J.W., Cayouette, M.H. and Price, D.L. (1993) Evidence that brain-derived neurotrophic factor is a trophic factor for motor neurons in vivo. Neuron, 10, 359-367.
43. Funakoshi, H., Frisen, J., Barbany, G., Timmusk, T., Zachrisson, O., Verge, V.M. and Persson, H. (1993) Differential expression of mRNAs for neurotrophins and their receptors after axotomy of the sciatic nerve. J. Cell Biol., 123, 455-465.
44. Loeb, J.A., Hmadcha, A., Fischbach, G.D., Land, S.J. and Zakarian, V.L. (2002) Neuregulin expression at neuromuscular synapses is modulated by synaptic activity and neurotrophic factors. J. Neurosci., 22 2206-2214.
45. Bommel, H., Xie, G., Rossoll, W., Wiese, S., Jablonka, S., Boehm, T. and Sendtner, M. (2002) Missense mutation in the tubulin-specific chaperone E (Tbce) gene in the mouse mutant progressive motor neuronopathy, a model of human motoneuron disease. J. Cell Biol., 159, 563-569.
46. Sagot, Y., Rosse, T., Vejsada, R., Perrelet, D. and Kato, A.C. (1998) Differential effects of neurotrophic factors on motoneuron retrograde labeling in a murine model of motoneuron disease. J. Neurosci., 18, 1132-1141.
47. DiStefano, P.S., Friedman, B., Radziejewski, C., Alexander, C., Boland, P., Schick, C.M., Lindsay, R.M. and Wiegand, S.J. (1992) The neurotrophins BDNF, NT-3, and NGF display distinct patterns of retrograde axonal transport in peripheral and central neurons. Neuron 8, 983-993.
48. Yan, Q., Elliott, J. and Snider, W.D. (1992) Brain-derived neurotrophic factor rescues spinal motor neurons from axotomy-induced cell death. Nature, 360, 753-755.
49. Davies, A.M., Horton, A., Burton, L.E., Schmelzer, C., Vandlen, R. and Rosenthal, A. (1993) Neurotophin-4/5 is a mammalian-specific survival factor for distinct populations of sensory neurons. J. Neurosci., 13, 4961-4967.
50. Rogers, S.D., Demaster, E., Catton, M., Ghilardi, J.R., Levin, L.A., Maggio, J.E. and Mantyh, P.W. (1997) Expression of endothelin-B receptors by glia in vivo is increased after CNS injury in rats, rabbits, and humans. Exp. Neurol., 145, 180-195.
51. McComas, A.J. (1991) Motor unit estimation: Methods, results, and present status. Muscle Nerve, 14, 585-597.
52. Shefner, J.M. and Gooch, C.L. (2002) Motor unit number estimation in neurologic disease. Adv. Neurol., 88, 33-52.