Neurotrophic factors: From neurodevelopmental regulators to novel therapies for Parkinson’s disease

Neural Regeneration Research

Volumn 9, Issue 19, 2014, Pages 1708-1711

  Hegarty, Shane V ^a   O'Keeffe, Gerard W ^a   Sullivan, Aideen M ^a  

^a UNIVERSITY COLLEGE CORK   (Ireland)

Author keywords

Glial cell line derived neurotrophic factor; Growth differentiation factor 5; Nervous system development; Neuroprotection; Neurotrophic factors; Neurturin; Nigrostriatal dopaminergic neurons; Parkinson s disease

Indexed keywords

ALPHA SYNUCLEIN; GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR RECEPTOR; GROWTH DIFFERENTIATION FACTOR 5; NEUROTROPHIC FACTOR; NEURTURIN; TRANSFORMING GROWTH FACTOR BETA;

ARTICLE; DOPAMINERGIC NERVE CELL; GENE THERAPY; GENE TRANSFER; HUMAN; IMMUNOCYTOCHEMISTRY; MOTOR PERFORMANCE; NERVE CELL GROWTH; NERVE DEGENERATION; NEUROPROTECTION; NONHUMAN; OPEN STUDY; PARKINSON DISEASE; PROTEIN EXPRESSION;

EID: 84908615939     PISSN: 16735374     EISSN: 18767958     Source Type: Journal    
DOI: 10.4103/1673-5374.143410     Document Type: Article

References (34)

1. Bartus RT, Baumann TL, Siffert J, Herzog CD, Alterman R, Boulis N, Turner DA, Stacy M, Lang AE, Lozano AM, Olanow CW (2013) Safety/ feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology 80:1698-1701.
2. Choi-Lundberg DL, Bohn MC (1995) Ontogeny and distribution of glial cell line-derived neurotrophic factor (GDNF) mRNA in rat. Brain Res Dev Brain Res 85:80-88.
3. Clarkson ED, Zawada WM, Freed CR (1997) GDNF improves survival and reduces apoptosis in human embryonic dopaminergic neurons in vitro. Cell Tissue Res 289:207-210.
4. Clayton KB, Sullivan AM (2007) Differential effects of GDF5 on the medial and lateral rat ventral mesencephalon. Neurosci Lett 427:132-137.
5. Costello DJ, O’Keeffe GW, Hurley FM, Sullivan AM (2012) Transplantation of novel human GDF-5-expressing CHO cells is neuroprotective in models of Parkinson’s disease. J Cell Mol Med 16:2451-2460.
6. Decressac M, Ulusoy A, Mattsson B, Georgievska B, Romero-Ramos M, Kirik D, Bjorklund A (2011) GDNF fails to exert neuroprotection in a rat alpha-synuclein model of Parkinson’s disease. Brain 134:2302-2311.
7. Eggert K, Schlegel J, Oertel W, Wurz C, Krieg JC, Vedder H (1999) Glial cell line-derived neurotrophic factor protects dopaminergic neurons from 6-hydroxydopamine toxicity in vitro. Neurosci Lett 269:178-182.
8. Gavin AM, Walsh S, Wyatt SL, O’ Keeffe GW, Sullivan AM (2013) 6-Hydroxydopamine induces distinct alterations in GDF5 and GDNF mRNA expression in the rat nigrostriatal system in vivo. Neurosci Lett 561:176-181.
9. Gill SS, Patel NK, Hotton GR, O’Sullivan K, McCarter R, Bunnage M, Brooks DJ, Svendsen CN, Heywood P (2003) Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease. Nat Med 9:589-595.
10. Granholm AC, Mott JL, Bowenkamp K, Eken S, Henry S, Hoffer BJ, Lapchak PA, Palmer MR, van Horne C, Gerhardt GA (1997) Glial cell line-derived neurotrophic factor improves survival of ventral mesencephalic grafts to the 6-hydroxydopamine lesioned striatum. Exp Brain Res 116:29-38.
11. Granholm AC, Reyland M, Albeck D, Sanders L, Gerhardt G, Hoernig G, Shen L, Westphal H, Hoffer B (2000) Glial cell line-derived neurotrophic factor is essential for postnatal survival of midbrain dopamine neurons. J Neurosci 20:3182-3190.
12. Hegarty SV, Sullivan AM, O’Keeffe GW (2014a) Roles for the TGFbeta Superfamily in the Development and Survival of Midbrain Dopaminergic Neurons. Mol Neurobiol 50:559-573.
13. Hegarty SV, Collins LM, Gavin AM, Roche SL, Wyatt SL, Sullivan AM, O’Keeffe GW (2014b) Canonical BMP-Smad signalling promotes neurite growth in rat midbrain dopaminergic neurons. Neuromolecular Med 16:473-489.
14. Herzog CD, Brown L, Kruegel BR, Wilson A, Tansey MG, Gage FH, Johnson EM, Jr., Bartus RT (2013) Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120). Neurobiol Dis 58:38-48.
15. Horger BA, Nishimura MC, Armanini MP, Wang LC, Poulsen KT, Rosenblad C, Kirik D, Moffat B, Simmons L, Johnson E, Jr., Milbrandt J, Rosenthal A, Bjorklund A, Vandlen RA, Hynes MA, Phillips HS (1998) Neurturin exerts potent actions on survival and function of midbrain dopaminergic neurons. J Neurosci 18:4929-4937.
16. Hurley FM, Costello DJ, Sullivan AM (2004) Neuroprotective effects of delayed administration of growth/differentiation factor-5 in the partial lesion model of Parkinson’s disease. Exp Neurol 185:281-289.
17. Kholodilov N, Yarygina O, Oo TF, Zhang H, Sulzer D, Dauer W, Burke RE (2004) Regulation of the development of mesencephalic dopaminergic systems by the selective expression of glial cell line-derived neurotrophic factor in their targets. J Neurosci 24:3136-3146.
18. Krieglstein K, Suter-Crazzolara C, Hotten G, Pohl J, Unsicker K (1995) Trophic and protective effects of growth/differentiation factor 5, a member of the transforming growth factor-beta superfamily, on midbrain dopaminergic neurons. J Neurosci Res 42:724-732.
19. Lang AE, Gill S, Patel NK, Lozano A, Nutt JG, Penn R, Brooks DJ, Hotton G, Moro E, Heywood P, Brodsky MA, Burchiel K, Kelly P, Dalvi A, Scott B, Stacy M, Turner D, Wooten VG, Elias WJ, Laws ER et al. (2006) Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease. Ann Neurol 59:459-466.
20. Lin LF, Doherty DH, Lile JD, Bektesh S, Collins F (1993) GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science 260:1130-1132.
21. Marks WJ, Jr., Ostrem JL, Verhagen L, Starr PA, Larson PS, Bakay RA, Taylor R, Cahn-Weiner DA, Stoessl AJ, Olanow CW, Bartus RT (2008) Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson’s disease: an open-label, phase I trial. Lancet Neurol 7:400-408.
22. Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA et al. (2010) Gene delivery of AAV2-neurturin for Parkinson’s disease: a double-blind, randomised, controlled trial. Lancet Neurol 9:1164-1172.
23. Nishitoh H, Ichijo H, Kimura M, Matsumoto T, Makishima F, Yamaguchi A, Yamashita H, Enomoto S, Miyazono K (1996) Identification of type I and type II serine/threonine kinase receptors for growth/ differentiation factor-5. J Biol Chem 271:21345-21352.
24. Nutt JG, Burchiel KJ, Comella CL, Jankovic J, Lang AE, Laws ER, Jr., Lozano AM, Penn RD, Simpson RK, Jr., Stacy M, Wooten GF (2003) Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology 60:69-73.
25. O’Keeffe GW, Dockery P, Sullivan AM (2004a) Effects of growth/differentiation factor 5 on the survival and morphology of embryonic rat midbrain dopaminergic neurones in vitro. J Neurocytol 33:479-488.
26. O’Keeffe GW, Hanke M, Pohl J, Sullivan AM (2004b) Expression of growth differentiation factor-5 in the developing and adult rat brain. Brain Res Dev Brain Res 151:199-202.
27. O’Sullivan DB, Harrison PT, Sullivan AM (2010) Effects of GDF5 overexpression on embryonic rat dopaminergic neurones in vitro and in vivo. J Neural Transm 117:559-572.
28. Slevin JT, Gerhardt GA, Smith CD, Gash DM, Kryscio R, Young B (2005) Improvement of bilateral motor functions in patients with Parkinson disease through the unilateral intraputaminal infusion of glial cell line-derived neurotrophic factor. J Neurosurg 102:216-222.
29. Storm EE, Huynh TV, Copeland NG, Jenkins NA, Kingsley DM, Lee SJ (1994) Limb alterations in brachypodism mice due to mutations in a new member of the TGF beta-superfamily. Nature 368:639-643.
30. Sullivan AM, Pohl J, Blunt SB (1998) Growth/differentiation factor 5 and glial cell line-derived neurotrophic factor enhance survival and function of dopaminergic grafts in a rat model of Parkinson’s disease. Eur J Neurosci 10:3681-3688.
31. Sullivan AM, Opacka-Juffry J, Hotten G, Pohl J, Blunt SB (1997) Growth/differentiation factor 5 protects nigrostriatal dopaminergic neurones in a rat model of Parkinson’s disease. Neurosci Lett 233:73-76.
32. Tomac A, Lindqvist E, Lin LF, Ogren SO, Young D, Hoffer BJ, Olson L (1995) Protection and repair of the nigrostriatal dopaminergic system by GDNF in vivo. Nature 373:335-339.
33. Toulouse A, Sullivan AM (2008) Progress in Parkinson’s disease-where do we stand? Prog Neurobiol 85:376-392.
34. Tseng JL, Bruhn SL, Zurn AD, Aebischer P (1998) Neurturin protects dopaminergic neurons following medial forebrain bundle axotomy. Neuroreport 9:1817-1822.