A commentary on glial cell line-derived neurotrophic factor (GDNF): From a glial secreted molecule to gene therapy

Biochemical Pharmacology

Volumn 57, Issue 2, 1999, Pages 135-142

  Bohn, Martha C ^a  

^a NORTHWESTERN UNIVERSITY   (United States)

Author keywords

Adenovirus; Amyotrophic lateral sclerosis; Dopamine; Motor neurons; Neuronal cell death; Neurotrophic factor; Parkinson's disease

Indexed keywords

GLIAL CELL LINE DERIVED NEUROTROPHIC FACTOR;

AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL EXPERIMENT; ANIMAL MODEL; CELL DEATH; DEGENERATIVE DISEASE; DOPAMINERGIC SYSTEM; GENE THERAPY; GLIA CELL; HUMAN; MOTOR NEURON DISEASE; NONHUMAN; PARKINSON DISEASE; PRIORITY JOURNAL; RAT; REVIEW;

EID: 0032927678     PISSN: 00062952     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0006-2952(98)00280-9     Document Type: Article

References (72)

1. Schubert D., Brass B., Duman J.-P. Protein complexity of central nervous system cell lines. J Neurosci. 6:1986;2829-2836.
2. Greene L.A., Tischler A.S. Establishment of a noradrenergic clonal cell line of rat adrenal pheochromocytoma cells which respond to nerve growth factor. Proc Natl Acad Sci USA. 73:1973;2424-2428.
3. Engele J., Schubert D., Bohn M.C. Conditioned media derived from glial cell lines promote survival and differentiation of dopaminergic neurons in vitro Role of mesencephalic glia . J Neurosci Res. 30:1991;359-371.
4. Lin L.-F.H., Doherty D.H., Lile J.D., Bektesh S., Collins F. GDNF A glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons . Science. 260:1993;1130-1132.
5. Stratford-Perricaudet L.D., Makeh I., Perricaudet M., Briand P. Widespread long-term gene transfer to mouse skeletal muscles and heart. J Clin Invest. 90:1992;626-630.
6. Strömberg I., Björklund L., Johansson M., Tomac A., Collins F., Olson L., Hoffer B., Humpel C. Glial cell line-derived neurotrophic factor is expressed in the developing but not adult striatum and stimulates developing dopamine neurons in vivo. Exp Neurol. 124:1993;401-412.
7. Schaar D.G., Sieber B.-A., Dreyfus C.F., Black I.B. Regional and cell-specific expression of GDNF in rat brain. Exp Neurol. 124:1993;368-371.
8. Trupp M., Ryden M., Jörnvall H., Funakoshi H., Timmusk T., Arenas E., Ibanez C.F. Peripheral expression and biological activities of GDNF, a new neurotrophic factor for avian and mammalian peripheral neurons. J Cell Biol. 130:1995;137-148.
9. Choi-Lundberg D.L., Bohn M.C. Ontogeny and distribution of glial cell line-derived neurotrophic factor (GDNF) mRNA in rat. Brain Res Dev Brain Res. 85:1995;80-88.
10. Tomac A., Widenflak J., Lin L.-F., Kohno T., Ebendal R., Hoffer B.J., Olson L. Retrograde axonal transport of glial cell line-derived neurotrophic factor in the adult nigrostriatal system suggests a trophic role in the adult. Proc Natl Acad Sci USA. 92:1995;8274-8278.
11. Henderson C.E., Phillips H.S., Pollock R.A., Davies A.M., Lemeulle C., Armanini M., Simmons L., Moffet B., Vandlen R.A., Koliatsos V.E., Rosenthal A. GDNF A potent survival factor for motoneurons present in perhiperhal nerve and muscle . Science. 266:1994;1062-1064.
12. Arenas E., Trupp M., Akerud P., Ibanez C.F. GDNF prevents degeneration and promotes the phenotype of brain noradrenergic neurons in vivo. Neuron. 15:1995;1465-1473.
13. Maxwell G.D., Reid K., Elefanty A., Bartlett P.F., Murphy M. Glial cell line-derived neurotrophic factor promotes the development of adrenergic neurons in mouse neural crest cultures. Proc Natl Acad Sci USA. 93:1996;13274-13279.
14. Mount H.T.J., Dean D.O., Alberch J., Dreyfus C.F., Black I.B. Glial cell line-derived neurotrophic factor promotes the survival and morphologic differentiation of Purkinje cells. Proc Natl Acad Sci USA. 92:1995;9092-9096.
15. Williams L.R., Inouye G., Cummins V., Pelleymounter M.A. Glial cell line-derived neurotrophic factor sustains axotomized basal forebrain cholinergic neurons in vivo Dose-response comparison to nerve growth factor brain-derived neurotrophic factor . J Pharmacol Exp Ther. 277:1996;1140-1151.
16. Beck K.D., Irwin I., Valverde J., Brennan T.J., Langston J.W., Hefti F. GDNF induces a dystonia-like state in neonatal rats and stimulates dopamine and serotonin synthesis. Neuron. 16:1996;665-673.
17. Ebendal T., Tomac A., Hoffer B.J., Olson L. Glial cell line-derived neurotrophic factor stimulates fiber formation and survival in cultured neurons from peripheral autonomic ganglia. J Neurosci Res. 40:1995;276-284.
18. Matheson C., Carnahan J., Urich J., Bocangel D., Zhang T., Yan Q. Glial cell line-derived neurotrophic factor (GDNF) is a neurotrophic factor for sensory neurons Comparison with the effects of the neurotrophins . J Neurobiol. 32:1997;22-32.
19. Moore M.W., Klein R.D., Fariñas I., Sauer H., Armanini M., Phillips H., Reichardt L.F., Ryan A.M., Carver-Moore K., Rosenthal A. Renal and neuronal abnormalities in mice lacking GDNF. Nature. 382:1996;76-79.
20. Pichel J.G., Shen L., Shenq H.Z., Granholm A.-C., Drago J., Grinberg A., Lee E.J., Huang S.P., Saarma M., Hoffer B.J., Sariola H., Westphal H. Defects in enteric innervation and kidney development in mice lacking GDNF. Nature. 382:1996;73-76.
21. Sánchez M.P., Silos-Santiago I., Frisén J., He B., Lira S.A., Barbacid M. Renal agenesis and the absence of enteric neurons in mice lacking GDNF. Nature. 382:1996;70-73.
22. Tomac A., Lindqvist E., Lin L.-F.H., Ögren S.O., Young D., Hoffer B.J., Olson L. Protection and repair of the nigrostriatal dopaminergic system by GDNF in vivo. Nature. 373:1995;335-339.
23. Beck K.D., Valverde J., Alexi T., Poulsen K., Moffat B., Vandlen R.A., Rosenthal A., Hefti F. Mesencephalic dopaminergic neurons protected by GDNF from axotomy-induced degeneration in the adult brain. Nature. 373:1995;339-341.
24. Kearns C.M., Gash D.M. GDNF protects nigral dopamine neurons against 6-hydroxydopamine in vivo. Brain Res. 672:1995;104-111.
25. Gash D.M., Zhang Z., Ovadia A., Cass W.A., Yi A., Simmerman L., Russell D., Martin D., Lapchak P.A., Collins F., Hoffer B.J., Gerhardt G.A. Functional recovery in parkinsonian monkeys treated with GDNF. Nature. 380:1996;252-255.
26. Oppenheim R.W., Housenou L.J., Johnson J.E., Lin L.-F.H., Li L., Lo A.C., Newsome A.L., Prevette D.M., Wang S. Developing motor neurons rescued from programmed and axotomy-induced cell death by GDNF. Nature. 373:1995;344-346.
27. Nomenclature of GPI-linked receptors for the GDNF ligand family. Neuron. 19:1997;485.
28. Jing S., Wen D., Yu Y., Holst P.L., Luo Y., Fang M., Tamir R., Antonio L., Hu Z., Cupples R., Louis J.-C., Hu S., Altrock B.W., Fox G.M. GDNF-induced activation of the ret protein tyrosine kinase is mediated by GDNFR-α, a novel receptor for GDNF. Cell. 85:1996;1113-1124.
29. Trupp M., Arenas E., Fainzilber M., Nilsson A.-S., Sieber B.-A., Grigoriou M., Kilkenny C., Salazar-Grueso E., Pachnis V., Arumae U., Sariola H., Saarma M., Ibanez C.F. Functional receptor for GDNF encoded by the c-ret proto-oncogene. Nature. 381:1996;785-789.
30. Durbec P., Marcos-Gutierrez C.V., Kilkenny C., Grigoriou M., Wartiowaara K., Suvanto P., Smith D., Ponder B., Costantini F., Saarma M., Sariola H., Pachnis V. GDNF signalling through the ret receptor tyrosine kinase. Nature. 381:1996;789-793.
31. Engele J., Bohn M.C. The neurotrophic effects of fibroblast growth factors on dopaminergic neurons in vitro are mediated by mesencephalic glia. J Neurosci. 11:1991;3070-3078.
32. Kotzbauer P., Lampe P., Heuckeroth R., Golden J., Creedon D., Johnson E. Jr. Neurturin, a relative of glial-cell-like-derived neurotrophic factor. Nature. 384:1996;467-470.
33. Rousselet A., Felter L., Chamak B., Prochiantz A. Rat mesencephalic neurons in culture exhibit different morphological traits in the presence of media conditioned on mesencephalic or striatal astroglia. Dev Biol. 129:1988;495-504.
34. Hoffman P.C., Hemmendinger L.M., Kotake C., Heller A. Enhanced dopamine cell survival in reaggregates containing telencephalic target cells. Brain Res. 274:1983;275-281.
35. Bohn M.C., Marciano F., Cupit L., Gash D.M. Adrenal medulla grafts enhance recovery of striatal dopaminergic fibers. Science. 237:1987;913-916.
36. Hudson J., Granholm A.-C., Gerhardt G.A., Henry M.A., Hoffman A., Biddle P., Leela N.S., Mackerlova L., Lile J.D., Collins F., Hoffer B.J. Glial cell line-derived neurotrophic factor augments midbrain dopaminergic circuits in vivo. Brain Res Bull. 36:1995;425-432.
37. Sauer H., Rosenblad C., Björklund A. Glial cell line-derived neurotrophic factor but not transforming growth factor β3 prevents delayed degeneration of nigral dopaminergic neurons following striatal 6-hydroxydopamine lesion. Proc Natl Acad Sci USA. 92:1995;8935-8939.
38. Bilang-Bleuel A., Revah F., Colin P., Locquet I., Robert J.-J., Mallet J., Horellou P. Intrastriatal injection of an adenoviral vector expressing glial-cell-line-derived neurotrophic factor prevents dopaminergic neuron degeneration and behavioral impairment in a rat model of Parkinson disease. Proc Natl Acad Sci, USA. 94:1997;8818-8823.
39. Choi-Lundberg DL, Lin Q, Bardwayj L, Qian J, Davidson BL, Schallert T, Crippens D, Chang Y-N, Chiang YL and Bohn MC, Behavioral and cellular protection of rat dopaminergic (DA) neurons elicited by a GDNF adenoviral vector (Ad). Exp Neurol154: in press, 1998.
40. Bowenkamp K.E., Hoffman A.F., Gerhardt G.A., Henry M.A., Biddle P.T., Hoffer B.J., Granholm A.-C.E. Glial cell line-derived neurotrophic factor supports survival of injured midbrain dopaminergic neurons. J Comp Neurol. 355:1995;479-489.
41. Gash D.M., Zhang Z., Cass W.A., Ovadia A., Simmerman L., Martin D., Russell D., Collins F., Hoffer B.J., Gerhardt G.A. Morphological and functional effects of intranigrally administered GDNF in normal rhesus monkeys. J Comp Neurol. 363:1995;345-358.
42. Lindner M.D., Winn S.R., Baetge E.E., Hammang J.P., Gentile F.T., Doherty E., McDermott P.E., Frydel B., Ullman M.D., Schallert T., Emerich D.F. Implantation of encapsulated catecholamine and GDNF producing cells in rats with unilateral dopamine depletions and Parkinsonian symptoms. Exp Neurol. 132:1995;62-76.
43. Rosenblad C., Martinez-Serrano A., Björklund A. 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. 82:1998;129-137.
44. Tseng J.L., Baetge E.E., Zurn A.D., Aebischer P. GDNF reduces drug-induced rotational behavior after medial forebrain bundle transection by a mechanism not involving striatal dopamine. J Neurosci. 17:1997;325-333.
45. Perez-Navarro E., Arenas E., Reiriz J., Calvo N., Alberch J. Glial cell line-derived neurotrophic factor protects striatal calbindin-immunoreactive neurons from excitotoxic damage. Neuroscience. 75:1996;345-352.
46. Gage F.H., Wictorin K., Rischer W., Williams L.R., Varon S., Bjorklund A. Retrograde cell changes in medial septum and diagonal band following fimbria-fornix transection Quantitative temporal analysis . Neuroscience. 19:1986;241-255.
47. Azmitia E.C., Liao B. Dexamethasone reverses adrenalectomy-induced neuronal de-differentiation in midbrain raphe-hippocampus axis. Ann NY Acad Sci. 746:1994;180-193.
48. Wang T., Lin S., Chiou A., Williams L., Hoffer B. Glial cell line-derived neurotrophic factor protects against ischemia-induced injury in the cerebral cortex. J Neurosci. 17:1997;4341-4348.
49. Treanor J.J.S., Goodman L., de Sauvage F., Stone D.M., Poulsen K.T., Beck C.D., Gray C., Armanini M.P., Pollock R.A., Hefti F., Phillips H.S., Goddard A., Moore M.W., Buj-Bello A., Davies A.M., Asai N., Takahaski M., Vandlen R., Henderson C.E., Rosenthal A. Characterization of a multicomponent receptor for GDNF. Nature. 382:1996;80-83.
50. Creedon D.J., Tansey M.G., Baloh R.H., Osborne P.A., Lampe P.A., Fahrner T.J., Heuckeroth R.O., Milbrandt J., Johnson E.M. Jr. Neurturin share receptors and signal transduction pathways with glial cell line-derived neurotrophic factor in sympathetic neurons. Proc Natl Acad Sci USA. 94:1997;7018-7023.
51. 11C-nicotine binding, blood flow, EEG, and verbal episodic memory in an Alzheimer patient (case report). J Neural Transm Park Dis Dement Sect. 4:1992;79-95.
52. Olson L, Bäckman L, Ebendal T, Eriksdotter-Jönhagen M, Hoffer B, Humpel C, Freedman R, Giacobini M, Meyerson B, Nordberg A, Seiger A, Strömberg I, Sydow O, Tomac A, Trok K and Winblad B, Role of growth factors in degeneration and regeneration in the central nervous system; clinical experiences with NGF in Parkinson's and Alzheimer's diseases. J Neurol 242(Suppl 1): S12-S15, 1994.
53. A double-blind placebo-controlled clinical trial of subcutaneous recombinant human ciliary neurotrophic factor (rHCNTF) in amyotrophic lateral sclerosis. Neurology. 46:1996;1244-1249.
54. 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., Stromatt S.C. A placebo-controlled trial of recombinant human ciliary neurotrophic (rhCNTF) factor in amyotrophic lateral sclerosis. Ann Neurol. 39:1996;256-260.
55. Zhang Z., Miyoshi Y., Lapchak P.A., Collins F., Hilt D., Lebel C., Kryscio R., Gash D.M. Dose response to intraventricular glial cell line-derived neurotrophic factor administration in parkinsonian monkeys. J Pharmacol Exp Ther. 282:1997;1396-1401.
56. Martin D., Miller G., Fischer N., Diz D., Cullen T., Russell D. Glial cell line-derived neurotrophic factor The lateral cerebral ventricle as a site of administration for stimulation of the substantia nigra dopamine system in rats . Eur J Neurosci. 8:1996;1249-1255.
57. Davidson B.L., Bohn M.C. Recombinant adenovirus. A gene transfer vector for study and treatment of CNS diseases. Exp Neurol. 144:1997;125-130.
58. Choi-Lundberg D.L., Lin Q., Chang Y.-N., Chiang Y.L., Hay C.M., Mohajeri H., Davidson B.L., Bohn M.C. Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science. 275:1997;838-841.
59. Choi-Lundberg DL and Bohn MC, Applications of gene therapy to neurological diseases and injuries. In: Stem Cell Biology and Gene Therapy (Eds. Quesenberry PJ, Stein GS, Forget B and Weissman S), pp. 503-553, John Wiley, New York, NY, 1998.
60. Bohn MC and Choi-Lundberg DL, Gene therapies for Parkinson's disease. In: Gene Therapy for Neurological Disorders and Brain Tumors (Eds. Chiocca EA and Breakefield XO), pp. 377-395, Humana Press, Totowa, NJ, 1998.
61. Forno L.S., DeLanney L.E., Irwin I., Langston J.W. Similarities and differences between MPTP-induced parkinsonism and Parkinson's disease. Neuropathologic considerations. Adv Neurol. 60:1993;600-608.
62. Langston JW, The etiology of Parkinson's disease with emphasis on the MPTP story. Neurology 47(Suppl 3): S153-S160, 1996.
63. Davidson B.L., Doran S.E., Shewach D.S., Latta J.M., Hartman J.W., Roessler B.J. Expression of Escherichia coli β-galactosidase and rat HPRT in the CNS of Macaca mulatta following adenoviral mediated gene transfer. Exp Neurol. 125:1994;258-267.
64. Bohn M.C., Choi-Lundberg D.L., Lawrence M.S., Leranth C., Smith J.C., Davidson B.L., O'Banion M.K., Redmond D.E. Jr. Prolonged transgene expression from an adenoviral vector injected into primate striatum. Soc Neurosci Abstr. 23:1997;295.
65. Bankiewicz K.S., Leff S.E., Nagy D., Jungles S., Rokovich J., Spratt K., Cohen L., Libonati M., Snyder R.O., Mandel R.J. Practical aspects of the development of ex vivo and in vivo gene therapy for Parkinson's disease. Exp Neurol. 144:1997;147-156.
66. Houenou L.J., Oppenheim R.W., Li L., Lo A.C., Prevette D. Regulation of spinal motoneuron survival by GDNF during development and following injury. Cell Tissue Res. 286:1996;219-223.
67. Yan Q., Matheson C., Lopez O.T. In vivo neurotrophic effects of GDNF on neonatal and adult facial motor neurons. Nature. 373:1995;341-344.
68. Sagot Y., Tan S.A., Hammang J.P., Aebischer P., Kato A.C. GDNF slows loss of motoneurons but not axonal degeneration or premature death of pmn/pmn mice. J Neurosci. 16:1996;2335-2341.
69. Munson J.B., McMahon S.B. Effects of GDNF on axotomized sensory and motor neurons in adult rats. Eur J Neurosci. 9:1997;1126-1129.
70. Matheson C.R., Wang J., Collins F.D., Yan Q. Long-term survival effects of GDNF on neonatal facial motoneurons after axotomy. Neuroreport. 8:1997;1739-1742.
71. Verma I.M., Somia N. Gene therapy - Promises, problems and prospects. Nature. 389:1997;239-242.
72. Anderson W.F., McGarrity G.J., Moen R.C. Report to the NIH Recombinant DNA Advisory Committee on murine replication-competent retrovirus (RCR) assays. Hum Gene Ther. 4:1993;311-321.