GDNF delivery for Parkinson's disease

Acta Neurochirurgica, Supplementum

Volumn , Issue 97 PART 2, 2007, Pages 135-154

  Patel, N K ^a   Gill, Steven S ^a  

^a FRENCHAY HOSPITAL   (United Kingdom)

Author keywords

convection enhanced delivery; drug delivery; GDNF; Neuromodulation; PD

Indexed keywords

GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR; NEUROPROTECTIVE AGENT;

ANIMAL; DRUG DELIVERY SYSTEM; HUMAN; INSTRUMENTATION; METHODOLOGY; PARKINSON DISEASE; REVIEW;

ANIMALS; DRUG DELIVERY SYSTEMS; GLIAL CELL LINE-DERIVED NEUROTROPHIC FACTOR; HUMANS; NEUROPROTECTIVE AGENTS; PARKINSON DISEASE;

EID: 85052608961     PISSN: 00651419     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-3-211-33081-4_16     Document Type: Review

References (157)

1. ALS CNTF Treatment Study Group (1996) A double-blind placebo-controlled clinical trial of subcutaneous recombinant human ciliary neurotrophic factor (rHCNTF) in amyotrophic lateral sclerosis. Neurology 46:1244-1249
2. Acsadi G, Anguelov RA, Yang H, Toth G, Thomas R, Jani A, Wang Y, Ianakova E, Mohammad S, Lewis RA, Shy ME (2002) Increased survival and function of SOD1 mice after glial cell-derived neurotrophic factor gene therapy. Hum Gene Ther 13:1047-1059
3. Aebischer P, Ridet J (2001) Recombinant proteins for neurodegenerative diseases: the delivery issue. Trends Neurosci 24:533-540
4. Ai Y, Markesbery W, Zhang Z, Grondin R, Elseberry D, Gerhardt GA, Gash DM (2003) Intraputamenal infusion of GDNF in aged rhesus monkeys: distribution and dopaminergic effects. J Comp Neurol 461:250-261
5. Airaksinen MS, Saarma M (2002) The GDNF family: signalling, biological functions and therapeutic value. Nat Rev Neurosci 3:383-394
6. Alexi T, Borlongan CV, Faull RL, Williams CE, Clark RG, Gluckman PD, Hughes PE (2000) Neuroprotective strategies for basal ganglia degeneration: Parkinson's and Huntington's diseases. Prog Neurobiol 60:409-470
7. Altar CA, Boylan CB, Fritsche M, Jackson C, Hyman C, Lindsay RM (1994) The neurotrophins NT-4/5 and BDNF augment serotonin, dopamine, and GABAergic systems during behaviorally effective infusions to the substantia nigra. Exp Neurol 130:31-40
8. Anderson KD, Alderson RF, Altar CA, Di Stefano PS, Corcoran TL, Lindsay RM, Wiegand SJ (1995) Differential distribution of exogenous BDNF, NGF, and NT-3 in the brain corresponds to the relative abundance and distribution of high-affinity and lowaffinity neurotrophin receptors. J Comp Neurol 357:296-317
9. Apfel SC (2001) Neurotrophic factor therapy - prospects and problems. Clin Chem Lab Med 39:351-355
10. Appel SH (1981) A unifying hypothesis for the cause of amyotrophic lateral sclerosis, parkinsonism, and Alzheimer disease. Ann Neurol 10:499-505
11. Arenas E (2002) Stem cells in the treatment of Parkinson's disease. Brain Res Bull 57:795-808
12. Arenas E, Trupp M, Akerud P, Ibanez CF (1995) GDNF prevents degeneration and promotes the phenotype of brain noradrenergic neurons in vivo. Neuron 15:1465-1473
13. Baloh RH, Tansey MG, Johnson EM Jr, Milbrandt J (2000) Functional mapping of receptor specificity domains of glial cell line-derived neurotrophic factor (GDNF) family ligands and production of GFRalpha1 RET-specific agonists. J Biol Chem 275:3412-3420
14. Baudet C, Mikaels A, Westphal H, Johansen J, Johansen TE, Ernfors P (2000) Positive and negative interactions of GDNF, NTN and ART in developing sensory neuron subpopulations, and their collaboration with neurotrophins. Development 127:4335-4344
15. Beck KD, Irwin I, Valverde J, Brennan TJ, Langston JW, Hefti F (1996) GDNF induces a dystonia-like state in neonatal rats and stimulates dopamine and serotonin synthesis. Neuron 16:665-673
16. Beck KD, Valverde J, Alexi T, Poulsen K, Moffat B, Vandlen RA, Rosenthal A, Hefti F (1995) Mesencephalic dopaminergic neurons protected by GDNF from axotomy-induced degeneration in the adult brain. Nature 373:339-341
17. Beck M, Karch C, Wiese S, Sendtner M (2001) Motoneuron cell death and neurotrophic factors: basic models for development of new therapeutic strategies in ALS. Amyotroph Lateral Scler Other Motor Neuron Disord 2 Suppl 1:S55-S68
18. Bennett DA, Beckett LA, Murray AM, Shannon KM, Goetz CG, Pilgrim DM, Evans DA (1996) Prevalence of parkinsonian signs and associated mortality in a community population of older people. N Engl J Med 334:71-76
19. Bennett DL, Michael GJ, Ramachandran N, Munson JB, Averill S, Yan Q, McMahon SB, Priestley JV (1998) A distinct subgroup of small DRG cells express GDNF receptor components and GDNF is protective for these neurons after nerve injury. J Neurosci 18:3059-3072
20. Bjorklund A, Kirik D, Rosenblad C, Georgievska B, Lundberg C, Mandel RJ (2000) Towards a neuroprotective gene therapy for Parkinson's disease: use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in the rat Parkinson model. Brain Res 886:82-98
21. Blesch A, Grill RJ, Tuszynski MH (1998) Neurotrophin gene therapy in CNS models of trauma and degeneration. In: Van Leeuwen FW, Salchi A, Giger RJ, Holtmaat AJGD, Verhaagen J (eds) Progress in brain research. Elsevier Science BV, Amsterdam, pp 473-484
22. Bobo RH, Laske DW, Akbasak A, Morrison PF, Dedrick RL, Oldfield EH (1994) Convection-enhanced delivery of macromolecules in the brain. Proc Natl Acad Sci USA 91:2076-2080
23. Boucher TJ, Okuse K, Bennett DL, Munson JB, Wood JN, McMahon SB (2000) Potent analgesic effects of GDNF in neuropathic pain states. Science 290:124-127
24. Bowers WJ, Howard DF, Federoff HJ (1997) Gene therapeutic strategies for neuroprotection: implications for Parkinson's disease. Exp Neurol 144:58-68
25. Buj-Bello A, Buchman VL, Horton A, Rosenthal A, Davies AM (1995) GDNF is an age-specific survival factor for sensory and autonomic neurons. Neuron 15:821-828
26. Cacalano G, Farinas I, Wang LC, Hagler K, Forgie, A, Moore M, Armanini M, Phillips H, Ryan AM, Reichardt LF, Hynes M, Davies A, Rosenthal A (1998) GFRalpha1 is an essential receptor component for GDNF in the developing nervous system and kidney. Neuron 21:53-62
27. Casper D, Mytilineou C, Blum M (1991) EGF enhances the survival of dopamine neurons in rat embryonic mesencephalon primary cell culture. J Neurosci Res 30:372-381
28. Chauhan NB, Siegel GJ, Lee JM (2001) Depletion of glial cell line-derived neurotrophic factor in substantia nigra neurons of Parkinson's disease brain. J Chem Neuroanat 21:277-288
29. Chen MY, Lonser RR, Morrison PF, Governale LS, Oldfield EH (1999) Variables affecting convection-enhanced delivery to the striatum: a systematic examination of rate of infusion, cannula size, infusate concentration, and tissue-cannula sealing time. J Neurosurg 90:315-320
30. Cheng H, Fraidakis M, Blomback B, Lapchak P, Hoffer B, Olson L (1998) Characterization of a fibrin glue-GDNF slow-release preparation. Cell Transplant 7:53-61
31. 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
32. Clauss MA, Jai RK (1990) Interstitial transport of rabbit and sheep antibodies in normal and neoplastic tissues. Cancer Res 50:3487-3492
33. Cohen S, Levi-Montalcini R, Hamburger V (1954) A nerve growth stimulating factor isolated from sarcomas 37 and 180. Proc Natl Acad Sci USA 40:1014-1018
34. Collier TJ, Sortwell CE (1999) Therapeutic potential of nerve growth factors in Parkinson's disease. Drugs Aging 14:261-287
35. Connor B, Dragunow M (1998) The role of neuronal growth factors in neurodegenerative disorders of the human brain. Brain Res Rev 27:1-39
36. Date I, Aoi M, Tomita S, Collins F, Ohmoto T (1998) GDNF administration induces recovery of the nigrostriatal dopaminergic system both in young and aged parkinsonian mice. Neuroreport 9:2365-2369
37. Date I, Notter MF, Felten SY, Felten DL (1990) MPTP-treated young mice but not aging mice show partial recovery of the nigrostriatal dopaminergic system by stereotaxic injection of acidic fibroblast growth factor (aFGF). Brain Res 526:156-160
38. de Rijk MC, Tzourio C, Breteler MM, Dartigues JF, Amaducci L, Lopez-Pousa S, Manubens-Bertran JM, Alperovitch A, Rocca WA (1997) Prevalence of parkinsonism and Parkinson's disease in Europe: the EUROPARKINSON collaborative study. European community concerted action on the epidemiology of Parkinson's disease. J Neurol Neurosurg Psychiatry 62:10-15
39. Del Fiacco M, Quartu M, Serra MP, Follesa P, Lai ML, Bachis A (2002) Topographical localization of glial cell line-derived neurotrophic factor in the human brain stem: an immunohistochemical study of prenatal, neonatal and adult brains. J Chem Neuroanat 23:29-48
40. Eigenbrot C, Gerber N (1997) X-ray structure of glial cell-derived neurotrophic factor at 1.9A resolution and implications for receptor binding. Nat Struct Biol 4:435-438
41. Engele J, Bohn MC (1991) The neurotrophic effects of fibroblast growth factors on dopaminergic neurons in vitro are mediated by mesencephalic glia. J Neurosci 11:3070-3078
42. Engele J, Franke B (1996) Effects of glial cell line-derived neurotrophic factor (GDNF) on dopaminergic neurons require concurrent activation of cAMP-dependent signaling pathways. Cell Tissue Res 286:235-240
43. Eriksdotter Jonhagen M, Nordberg A, Amberla K, Backman L, Ebendal T, Meyerson B, Olson L, Seiger Shigeta M, Theodorsson E, Viitanen M, Winblad B, Wahlund LO (1998) Intracerebroventricular infusion of nerve growth factor in three patients with Alzheimer's disease. Dement Geriatr Cogn Disord 9:246-257
44. Fearnley JM, Lees AJ (1991) Ageing and Parkinson's disease: substantia nigra regional selectivity. Brain 114(Pt 5):2283-2301
45. Francois C, Savy C, Jan C, Tande D, Hirsch EC, Yelnik J (2000) Dopaminergic innervation of the subthalamic nucleus in the normal state, in MPTP-treated monkeys, and in Parkinson's disease patients. J Comp Neurol 425:121-129
46. Francois C, Yelnik J, Tande D, Agid Y, Hirsch EC (1999) Dopaminergic cell group A8 in the monkey: anatomical organization and projections to the striatum. J Comp Neurol 414:334-347
47. Frim DM, Uhler TA, Galpern WR, Beal MF, Breakefield XO, Isacson O (1994) Implanted fibroblasts genetically engineered to produce brain-derived neurotrophic factor prevent 1-methyl-4-phenylpyridinium toxicity to dopaminergic neurons in the rat. Proc Natl Acad Sci USA 91:5104-5108
48. Gash DM, Gerhardt GA, Hoffer BJ (1998a) Effects of glial cell line-derived neurotrophic factor on the nigrostriatal dopamine system in rodents and nonhuman primates. Adv Pharmacol 42:911-915
49. Gash DM, Zhang Z, Gerhardt G (1998b) Neuroprotective and neurorestorative properties of GDNF. Ann Neurol 44:S121-S125
50. Gash DM, Zhang Z, Ovadia A, Cass WA, Yi A, Simmerman L, Russell D, Martin D, Lapchak PA, Collins F, Hoffer BJ, Gerhardt GA (1996) Functional recovery in parkinsonian monkeys treated with GDNF. Nature 380:252-255
51. 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
52. Goetz CG, Leurgans S, Raman R, Stebbins GT (2000) Objective changes in motor function during placebo treatment in PD. Neurology 54:710-714
53. Grimes ML, Zhou J, Beattie EC, Yuen EC, Hall DE, Valletta JS, Topp KS, La Vail JH, Bunnett NW, Mobley WC (1996) Endocytosis of activated TrkA: evidence that nerve growth factor induces formation of signaling endosomes. J Neurosci 16:7950-7964
54. Grondin R, Cass WA, Zhang Z, Stanford JA, Gash DM, Gerhardt GA (2003) Glial cell line-derived neurotrophic factor increases stimulus-evoked dopamine release and motor speed in aged rhesus monkeys. J Neurosci 23:1974-1980
55. Grondin R, Gash DM (1998) Glial cell line-derived neurotrophic factor (GDNF): a drug candidate for the treatment of Parkinson's disease. J Neurol 245:P35-P42
56. Grondin R, Zhang Z, Yi A, Cass WA, Maswood N, Andersen AH, Elsberry DD, Klein MC, Gerhardt GA, Gash DM (2002) Chronic, controlled GDNF infusion promotes structural and functional recovery in advanced parkinsonian monkeys. Brain 125:2191-2201
57. Haller MF, Saltzman WM (1998) Localized delivery of proteins in the brain: can transport be customized? Pharm Res 15:377-385
58. Hassani OK, Francois C, Yelnik J, Feger J (1997) Evidence for a dopaminergic innervation of the subthalamic nucleus in the rat. Brain Res 749:88-94
59. Hebert MA, Gerhardt GA (1997) Behavioral and neurochemical effects of intranigral administration of glial cell line-derived neurotrophic factor on aged Fischer 344 rats. J Pharmacol Exp Ther 282:760-768
60. Hefti F (1983) Is Alzheimer disease caused by lack of nerve growth factor? Ann Neurol 13:109-110
61. Hoane MR, Gulwadi AG, Morrison S, Hovanesian G, Lindner MD, Tao W (1999) Differential in vivo effects of neurturin and glial cell-line-derived neurotrophic factor. Exp Neurol 160:235-243
62. Hoffman AF, van Horne CG, Eken S, Hoffer BJ, Gerhardt GA (1997) In vivo microdialysis studies on somatodendritic dopamine release in the rat substantia nigra: effects of unilateral 6-OHDA lesions and GDNF. Exp Neurol 147:130-141
63. Hotton GR, Patel NK, Gill SS, Heywood P, Svendson SN, Brooks DJ (2004) The long term effect of glial derived neurotrophic factor infusions and 18F-dopa uptake in Parkinson's disease. Neurology 62:S38.001, A345
64. Hughes PE, Alexi T, Walton M, Williams CE, Druganow M, Clark RG, Gluckman PD (1999) Activity and injury-dependant expression of inducible trophic factors, growth factors, and apoptosisrelated genes within the central nervous system. Progr Neurobiol 57:421-450
65. Hyman C, Hofer M, Barde YA, Juhasz M, Yancopoulos GD, Squinto SP, Lindsay RM (1991) BDNF is a neurotrophic factor for dopaminergic neurons of the substantia nigra. Nature 350:230-232
66. Hyman C, Juhasz M, Jackson C, Wright P, Ip NY, Lindsay RM (1994) Overlapping and distinct actions of the neurotrophins BDNF, NT-3, and NT-4/5 on cultured dopaminergic and GABAergic neurons of the ventral mesencephalon. J Neurosci 14:335-347
67. Hynes MA, Poulsen K, Armanini M, Berkemeier L, Phillips H, Rosenthal A (1994) Neurotrophin-4/5 is a survival factor for embryonic midbrain dopaminergic neurons in enriched cultures. J Neurosci Res 37:144-154
68. Isacson O, Deacon T (1997) Neural transplantation studies reveal the brain's capacity for continuous reconstruction. Trends Neurosci 20:477-482
69. Jellinger K, Riederer P, Tomonaga M (1980) Progressive supranuclear palsy: clinico-pathological and biochemical studies. J Neural Transm Suppl: 111-128
70. Jellinger KA (2002) Recent developments in the pathology of Parkinson's disease. J Neural Transm Suppl: 347-376
71. Jing S, Wen D, Yu Y, Holst PL, Luo Y, Fang M, Tamir R, Antonio L, Hu Z, Cupples R, Louis JC, Hu S, Altrock BW, Fox GM (1996) GDNF-induced activation of the ret protein tyrosine kinase is mediated by GDNFR-alpha, a novel receptor for GDNF. Cell 85:1113-1124
72. Jollivet C, Aubert-Pouessel A, Clavreul A, Venier-Julienne MC, Remy S, Montero-Menei CN, Benoit JP, Menei P (2004) Striatal implantation of GDNF releasing biodegradable microspheres promotes recovery of motor function in a partial model of Parkinson's disease. Biomaterials 25:933-942
73. Kafri T, van Praag H, Gage FH, Verma IM (2000) Lentiviral vectors: regulated gene expression. Mol Ther 1:516-521
74. Kawamoto Y, Nakamura S, Matsuo A, Akiguchi I, Shibasaki H (2000) Immunohistochemical localization of glial cell linederived neurotrophic factor in the human central nervous system. Neuroscience 100:701-712
75. Kirik D, Georgievska B, Bjorklund A (2004) Localized striatal delivery of GDNF as a treatment for Parkinson disease. Nat Neurosci 7:105-110
76. Knusel B, Hefti F (1991) Trophic actions of IGF-I, IGF-II and insulin on cholinergic and dopaminergic brain neurons. Adv Exp Med Biol 293:351-360
77. Kokaia Z, Bengzon J, Metsis M, Kokaia M, Persson H, Lindvall O (1993) Coexpression of neurotrophins and their receptors in neurons of the central nervous system. Proc Natl Acad Sci USA 90:6711-6715
78. Kolmac C, Mitrofanis J (1998) Distribution of various neurochemicals within the zona incerta: an immunocytochemical and histochemical study. Anat Embryol (Berl) 199:265-280
79. Kordower JH (2003) In vivo gene delivery of glial cell line-derived neurotrophic factor for Parkinson's disease. Ann Neurol 53:S120-S134
80. Kordower JH, Emborg ME, Bloch J, Ma SY, Chu Y, Leventhal L, McBride J, Chen EY, Palfi S, Roitberg BZ, Brown WD, Holden JE, Pyzalski R, Taylor MD, Carvey P, Ling Z, Trono D, Hantraye P, Deglon N, Aebischer P (2000) Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science 290:767-773
81. Kordower JH, Isacson O, Emerich DF (1999a) Cellular delivery of trophic factors for the treatment of Huntington's disease: is neuroprotection possible? Exp Neurol 159:4-20
82. Kordower JH, Palfi S, Chen EY, Ma SY, Sendera T, Cochran EJ, Mufson EJ, Penn R, Goetz CG, Comella CD (1999b) Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease. Ann Neurol 46:419-424
83. Korsching S (1993) The neurotrophic factor concept: a reexamination. J Neurosci 13:2739-2748
84. Krieglstein K, Unsicker K (1994) Transforming growth factorbeta promotes survival of midbrain dopaminergic neurons and protects them against N-methyl-4-phenylpyridinium ion toxicity. Neuroscience 63:1189-1196
85. Lachyankar MB, Condon PJ, Quesenberry PJ, Litofsky NS, Recht LD, Ross AH (1997) Embryonic precursor cells that express Trk receptors: induction of different cell fates by NGF, BDNF, NT-3, and CNTF. Exp Neurol 144:350-360
86. Lang AE, Gill SS, Patel NK, Lozano AM, Nutt JG, Penn R (2006) Randomized controlled trial of intraputamenal GDNF infusion in Parkinson disease. Ann Neurol 59:459-466
87. Lang AE, Lozano AM (1998) Parkinson's disease. First of two parts. N Engl J Med 339:1044-1053
88. Lapchak PA (1996) Therapeutic potentials for glial cell linederived neurotrophic factor (GDNF) based upon pharmacological activities in the CNS. Rev Neurosci 7:165-176
89. Lapchak PA, Beck KD, Araujo DM, Irwin I, Langston JW, Hefti F (1993) Chronic intranigral administration of brain-derived neurotrophic factor produces striatal dopaminergic hypofunction in unlesioned adult rats and fails to attenuate the decline of striatal dopaminergic function following medial forebrain bundle transection. Neuroscience 53:639-650
90. Levi-Montalcini R (1987) The nerve growth factor: thirty-five years later. Embo J 6:1145-1154
91. Li Duan M, Bordet T, Mezzina M, Kahn A, Ulfendahl M (2002) Adenoviral and adeno-associated viral vector mediated gene transfer in the guinea pig cochlea. Neuroreport 13:1295-1299
92. Lieberman DM, Laske DW, Morrison PF, Bankiewicz KS, Oldfield EH (1995) Convection-enhanced distribution of large molecules in gray matter during interstitial drug infusion. J Neurosurg 82:1021-1029
93. 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
94. Lin LF, Zhang TJ, Collins F, Armes LG (1994) Purification and initial characterization of rat B49 glial cell line-derived neurotrophic factor. J Neurochem 63:758-768
95. Lindvall O, Hagell P (2000) Clinical observations after neural transplantation in Parkinson's disease. Prog Brain Res 127:299-320
96. Lindvall O, Rehncrona S, Brundin P, Gustavii B, Astedt B, Widner H, Lindholm T, Bjorklund A, Leenders KL, Rothwell JC et al (1989) Human fetal dopamine neurons grafted into the striatum in two patients with severe Parkinson's disease. A detailed account of methodology and a 6-month follow-up. Arch Neurol 46:615-631
97. Louis ED, Marder K, Cote L, Tang M, Mayeux R (1997) Mortality from Parkinson disease. Arch Neurol 54:260-264
98. Love S, Plaha P, Patel NK, Hotton GR, Brooks DJ, Gill SS (2005) Glial cell line-derived neurotrophic factor induces neuronal sprouting in human brain. Nat Med 11:703-704
99. Lu X, Hagg T (1997) Glial cell line-derived neurotrophic factor prevents death, but not reductions in tyrosine hydroxylase, ofinjured nigrostriatal neurons in adult rats. J Comp Neurol 388:484-494
100. Maisonpierre PC, Belluscio L, Friedman B, Alderson RF, Wiegand SJ, Furth ME, Lindsay RM, Yancopoulos GD (1990) NT-3, BDNF, and NGF in the developing rat nervous system: parallel as well as reciprocal patterns of expression. Neuron 5:501-509
101. Marek K, Innis R, van Dyck C, Fussell B, Early M, Eberly S, Oakes D, Seibyl J (2001) [123I]beta-CIT SPECT imaging assessment of the rate of Parkinson's disease progression. Neurology 57:2089-2094
102. Martin D, Miller G, Cullen T, Fischer N, Dix D, Russell D (1996) Intranigral or intrastriatal injections of GDNF: effects on monoamine levels and behavior in rats. Eur J Pharmacol 317:247-256
103. Martin D, Miller G, Rosendahl M, Russell DA (1995) Potent inhibitory effects of glial derived neurotrophic factor against kainic acid mediated seizures in the rat. Brain Res 683:172-178
104. Mattson MP (1998) Neuroprotective strategies based on targeting of postreceptor signalling events. Neuroprotective signal transduction, Humana Press, Totown, pp 301-335
105. Meng X, Lindahl M, Hyvonen ME, Parvinen M, de Rooij DG, Hess M, Raatikainen-Ahokas A, Sainio K, Rauvala H, Lakso M, Pichel JG, Westphal H, Saarma M, Sariola H (2000) Regulation of cell fate decision of undifferentiated spermatogonia by GDNF. Science 287:1489-1493
106. Miranda RC, Sohrabji F, Toran-Allerand CD (1993) Neuronal colocalization of mRNAs for neurotrophins and their receptors in the developing central nervous system suggests a potential for autocrine interactions. Proc Natl Acad Sci USA 90:6439-6443
107. Miyoshi Y, Zhang Z, Ovadia A, Lapchak PA, Collins F, Hilt D, Lebel C, Kryscio R, Gash DM (1997) Glial cell line-derived neurotrophic factor-levodopa interactions and reduction of side effects in parkinsonian monkeys. Ann Neurol 42:208-214
108. Moore MW, Klein RD, Farinas I, Sauer H, Armanini M, Phillips H, Reichardt LF, Ryan AM, Carver-Moore K, Rosenthal A (1996) Renal and neuronal abnormalities in mice lacking GDNF. Nature 382:76-79
109. Morens DM, Davis JW, Grandinetti A, Ross GW, Popper JS, White LR (1996) Epidemiologic observations on Parkinson's disease: incidence and mortality in a prospective study of middle-aged men. Neurology 46:1044-1050
110. Morrish PK, Sawle GV, Brooks DJ (1996) An [18F]dopa-PET and clinical study of the rate of progression in Parkinson's disease. Brain 119(Pt 2):585-591
111. Morrison PF, Laske DW, Bobo H, Oldfield EH, Dedrick RL (1994) High-flow microinfusion: tissue penetration and pharmacodynamics. Am J Physiol 266:R292-R305
112. Morse JK, Wiegand SJ, Anderson K, You Y, Cai N, Carnahan J, Miller J, Di Stefano PS, Altar CA, Lindsay RM et al (1993) Brainderived neurotrophic factor (BDNF) prevents the degeneration of medial septal cholinergic neurons following fimbria transection. J Neurosci 13:4146-4156
113. Mount HT, Dean DO, Alberch J, Dreyfus CF, Black IB (1995) Glial cell line-derived neurotrophic factor promotes the survival and morphologic differentiation of Purkinje cells. Proc Natl Acad Sci USA 92:9092-9096
114. Muldoon LL, Nilaver G, Kroll RA, Pagel MA, Breakefield XO, Chiocca EA, Davidson BL, Weissleder R, Neuwelt EA (1995) Comparison of intracerebral inoculation and osmotic blood-brain barrier disruption for delivery of adenovirus, herpesvirus, and iron oxide particles to normal rat brain. Am J Pathol 147:1840-1851
115. Nakao N, Yokote H, Nakai K, Itakura T (2000) Promotion of survival and regeneration of nigral dopamine neurons in a rat model of Parkinson's disease after implantation of embryonal carcinoma-derived neurons genetically engineered to produce glial cell line-derived neurotrophic factor. J Neurosurg 92:659-670
116. Nosrat CA, Tomac A, Lindqvist E, Lindskog S, Humpel C, Stromberg I, Ebendal T, Hoffer BJ, Olson L (1996) Cellular expression of GDNF mRNA suggests multiple functions inside and outside the nervous system. Cell Tissue Res 286:191-207
117. 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
118. Olanow CW, Goetz CG, Kordower JH, Stoessl AJ, Sossi V, Brin MF, Shannon KM, Nauert GM, Perl DP, Godbold J, Freeman TB (2003) A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann Neurol 54:403-414
119. Olson L (1996) Toward trophic treatment in parkinsonism: a primate step. Nat Med 2:400-401
120. Olson L, Nordberg A, von Holst H, Backman L, Ebendal T, Alafuzoff I, Amberla K, Hartvig P, Herlitz A, Lilja A et al (1992) Nerve growth factor affects 11C-nicotine binding, blood flow, EEG, and verbal episodic memory in an Alzheimer patient (case report). J Neural Transm Park Dis Dement Sect 4:79-95
121. Opacka-Juffry J, Ashworth S, Hume SP, Martin D, Brooks DJ, Blunt SB (1995) GDNF protects against 6-OHDA nigrostriatal lesion: in vivo study with microdialysis and PET. Neuroreport 7:348-352
122. Oppenheim RW (1989) The neurotrophic theory and naturally occurring motoneuron death. Trends Neurosci 12:252-255
123. Oppenheim RW, Houenou LJ, Parsadanian AS, Prevette D, Snider WD, Shen L (2000) Glial cell line-derived neurotrophic factor and developing mammalian motoneurons: regulation of programmed cell death among motoneuron subtypes. J Neurosci 20:5001-5011
124. Patel NK, Bunnage M, Plaha P, Svendsen CN, Heywood P, Gill SS (2005) Intraputamenal infusion of glial cell line-derived neurotrophic factor in PD: a two-year outcome study. Ann Neurol 57:298-302
125. Pichel JG, Shen L, Sheng HZ, Granholm AC, Drago J, Grinberg A, Lee EJ, Huang SP, Saarma M, Hoffer BJ, Sariola H, Westphal H (1996) Defects in enteric innervation and kidney development in mice lacking GDNF. Nature 382:73-76
126. Pochon NA, Menoud A, Tseng JL, Zurn AD, Aebischer P (1997) Neuronal GDNF expression in the adult rat nervous system identified by in situ hybridization. Eur J Neurosci 9:463-471
127. Pothos EN, Davila V, Sulzer D (1998) Presynaptic recording of quanta from midbrain dopamine neurons and modulation of the quantal size. J Neurosci 18:4106-4118
128. Ridet JL, Deglon N, Aebischer P (2000) Gene transfer techniques for the delivery of GDNF in Parkinson's disease. Novartis Found Symp 231:202-215; discussion 215-219, 302-306
129. Rosenblad C, Martinez-Serrano A, Bjorklund A (1998) Intrastriatal glial cell line-derived neurotrophic factor promotes sprouting of spared nigrostriatal dopaminergic afferents and induces recovery of function in a rat model of Parkinson's disease. Neuroscience 82:129-137
130. Sanchez MP, Silos-Santiago I, Frisen J, He B, Lira SA, Barbacid M (1996) Renal agenesis and the absence of enteric neurons in mice lacking GDNF. Nature 382:70-73
131. Schecterson LC, Bothwell M (1992) Novel roles for neurotrophins are suggested by BDNF and NT-3 mRNA expression in developing neurons. Neuron 9:449-463
132. Schuchardt A, D'Agati V, Larsson-Blomberg L, Costantini F, Pachnis V (1994) Defects in the kidney and enteric nervous system of mice lacking the tyrosine kinase receptor ret. Nature 367:380-383
133. 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
134. Smith Y, Kieval JZ (2000) Anatomy of the dopamine system in the basal ganglia. Trends Neurosci 23:S28-S33
135. Sofroniew MV, Howe CL, Mobley WC (2001) Nerve growth factor signaling, neuroprotection, and neural repair. Annu Rev Neurosci 24:1217-1281
136. Takahashi M (2001) The GDNF/RET signaling pathway and human diseases. Cytokine Growth Factor Rev 12:361-373
137. Tansey MG, Baloh RH, Milbrandt J, Johnson EM Jr (2000) GFRalpha-mediated localization of RET to lipid rafts is required for effective downstream signaling, differentiation, and neuronal survival. Neuron 25:611-623
138. Thoenen H, Barde YA, Davies AM, Johnson JE (1987) Neurotrophic factors and neuronal death. Ciba Found Symp 126:82-95
139. Thorne RG, Frey WH 2nd (2001) Delivery of neurotrophic factors to the central nervous system: pharmacokinetic considerations. Clin Pharmacokinet 40:907-946
140. 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
141. Tornqvist N, Bjorklund L, Almqvist P, Wahlberg L, Stromberg I (2000) Implantation of bioactive growth factor-secreting rods enhances fetal dopaminergic graft survival, outgrowth density, and functional recovery in a rat model of Parkinson's disease. Exp Neurol 164:130-138
142. Treanor JJ, Goodman L, de Sauvage F, Stone DM, Poulsen KT, Beck CD, Gray C, Armanini MP, Pollock RA, Hefti F, Phillips HS, Goddard A, Moore MW, Buj-Bello A, Davies AM, Asai N, Takahashi M, Vandlen R, Henderson CE, Rosenthal A (1996) Characterization of a multicomponent receptor for GDNF. Nature 382:80-83
143. Trupp M, Arenas E, Fainzilber M, Nilsson AS, Sieber BA, Grigoriou M, Kilkenny C, Salazar-Grueso E, Pachnis V, Arumae U (1996) Functional receptor for GDNF encoded by the c-ret proto-oncogene. Nature 381:785-789
144. Trupp M, Belluardo N, Funakoshi H, Ibanez CF (1997) Complementary and overlapping expression of glial cell linederived neurotrophic factor (GDNF), c-ret proto-oncogene, and GDNF receptor-alpha indicates multiple mechanisms of trophic actions in the adult rat CNS. J Neurosci 17:3554-3567
145. Trupp M, Ryden M, Jornvall H, Funakoshi H, Timmusk T, Arenas E, Ibanez CF (1995) Peripheral expression and biological activities of GDNF, a new neurotrophic factor for avian and mammalian peripheral neurons. J Cell Biol 130:137-148
146. Trupp M, Scott R, Whittemore SR, Ibanez CF (1999) Ret-dependent and -independent mechanisms of glial cell line-derived neurotrophic factor signaling in neuronal cells. J Biol Chem 274:20885-20894
147. Tuszynski MH (2002) Growth-factor gene therapy for neurodegenerative disorders. Lancet Neurol 1:51-57
148. Vigna E, Cavalieri S, Ailles L, Geuna M, Loew R, Bujard H, Naldini L (2002) Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Mol Ther 5:252-261
149. 2+ channels and excitatory synaptic transmission in midbrain dopaminergic neurons. Neurosignals 12:78-88
150. Wang LJ, Lu YY, Muramatsu S, Ikeguchi K, Fujimoto K, Okada T, Mizukami H, Matsushita T, Hanazono Y, Kume A, Nagatsu T, Ozawa K, Nakano I (2002) Neuroprotective effects of glial cell line-derived neurotrophic factor mediated by an adeno-associated virus vector in a transgenic animal model of amyotrophic lateral sclerosis. J Neurosci 22:6920-6928
151. Weis C, Marksteiner J, Humpel C (2001) Nerve growth factor and glial cell line-derived neurotrophic factor restore the cholinergic neuronal phenotype in organotypic brain slices of the basal nucleus of Meynert. Neuroscience 102:29-38
152. Williams LR, Inouye G, Cummins V, Pelleymounter MA (1996) Glial cell line-derived neurotrophic factor sustains axotomized basal forebrain cholinergic neurons in vivo: dose-response comparison to nerve growth factor and brain-derived neurotrophic factor. J Pharmacol Exp Ther 277:1140-1151
153. Xu RY, Pong K, Yu Y, Chang D, Liu S, Lile JD, Treanor J, Beck KD, Louis JC (1998) Characterization of two distinct monoclonal antibodies specific for glial cell line-derived neurotrophic factor. J Neurochem 70:1383-1393
154. Yan Q, Matheson C, Lopez OT (1995) In vivo neurotrophic effects of GDNF on neonatal and adult facial motor neurons. Nature 373:341-344
155. Yoshimoto Y, Lin Q, Collier TJ, Frim DM, Breakefield XO, Bohn MC (1995) Astrocytes retrovirally transduced with BDNF elicit behavioral improvement in a rat model of Parkinson's disease. Brain Res 691:25-36
156. Yuen EC, Howe CL, Li Y, Holtzman DM, Mobley WC (1996) Nerve growth factor and the neurotrophic factor hypothesis. Brain Dev 18:362-368
157. Zurn AD, Baetge EE, Hammang JP, Tan SA, Aebischer P (1994) Glial cell line-derived neurotrophic factor (GDNF), a new neurotrophic factor for motoneurones. Neuroreport 6:113-118