Central nervous system delivery of recombinant ciliary neurotrophic factor by polymer encapsulated differentiated C2C12 myoblasts

Human Gene Therapy

Volumn 7, Issue 17, 1996, Pages 2135-2146

  Deglon N ^a   Heyd, B ^b   Tan, S A ^c   Joseph, J M ^a   Zurn, A D ^a   Aebischer, P ^a,d  

^a University of Lausanne Medical Center   (Switzerland)

^b UNIVERSITY HOSPITAL JEAN MINJOZ   (France)

^c Georgia Institute of Technology   (United States)

^d LAUSANNE UNIVERSITY HOSPITAL   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

BROXURIDINE; CHOLINE ACETYLTRANSFERASE; CILIARY NEUROTROPHIC FACTOR; GANCICLOVIR; MESSENGER RNA; RECOMBINANT PROTEIN; THYMIDINE KINASE;

ANIMAL CELL; ARTICLE; BLOOD BRAIN BARRIER; CELL PROLIFERATION; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DNA TRANSFECTION; DRUG DELIVERY SYSTEM; ENCAPSULATION; ENZYME ACTIVITY; ENZYME LINKED IMMUNOSORBENT ASSAY; EXPRESSION VECTOR; GENE EXPRESSION; GENE TARGETING; HERPES SIMPLEX VIRUS; HUMAN; MOTONEURON; MOUSE; MYOBLAST; NERVE CELL CULTURE; NERVE DEGENERATION; NERVE FIBER TRANSECTION; NONHUMAN; NORTHERN BLOTTING; PROTEIN SECRETION; SPINAL CORD;

ANIMALIA; HERPES; SIMPLEXVIRUS;

EID: 0029846012     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.17-2135     Document Type: Article

References (61)

1. ADLER, R., LANDA, K.B., MANTHORPE, M., and VARON, S. (1979). Cholinergic neuronotrophic factors: Intraocular distribution of soluble trophic activity for ciliary neurons. Science 204, 1434-1436.
2. AEBISCHER, P., TRESCO, P.A., WINN, S.R., GREENE, L.A., and JAEGER, C.B. (1991). Long-term cross-species brain transplantation of a polymer-encapsulated dopamine-secreting cell line. Exp. Neurol. 111, 269-275.
3. AEBISCHER, P., BUCHSER, E., JOSEPH, J.M., FAVRE, J., DE TRIBOLET, N., LYSAGHT, M., RUDNICK, S., and GODDARD, M. (1994a). Tranplantation in humans of encapsulated xenogenic cells without immunosuppression. Transplantation 58, 1275-1277.
4. AEBISCHER, P., GODDARD, M., SIGNORE, A.P., and TIMPSON, R.L. (1994b). Functional recovery in hermiparkinsonian primates transplanted with polymer-encapsulated PC12 cells. Exp. Neurol. 126, 151-158.
5. AEBISCHER, P., POCHON, N.A.-M., HEYD, B., DÉGLON, N., JOSEPH, J.-M., ZURN, A.D., BAETGE, E.E., HAMMANG, J.P., GODDARD, M., LYSAGHT, M., KAPLAN, F., KATO, A.C., SCHLUEP, M., HIRT, L., REGLI, F., PORCHET, F., and DE TRIBOLET, N. (1996a). Gene therapy for amyotrophic lateral sclerosis (ALS) using a polymer encapsulated xenogenic cell line engineered to secrete hCNTF. Hum. Gene Ther. 7, 851-860.
6. AEBISCHER, P., SCHLUEP, M., DÉGLON, N., JOSEPH, J.-M., HIRT, L., HEYD, B., GODDARD, M., HAMMANG, J., ZURN, A.D., KATO, A., REGLI, F., and BAETGE, E.E. (1996b). Intrathecal delivery of CNTF using encapsulated genetically modified xenogeneic cells in amyotrophic lateral sclerosispatients. Nature Med. 2, 696-699.
7. AL-HENDY, A., HORTELANO, G., TANNENBAUM, G.S., and CHANG, P.L. (1995). Correction of the growth defect in dwarf mice with nonautologous microencapsulated myoblasts: an alternate approach to somatic gene therapy. Hum. Gene Ther. 6, 165-175.
8. 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.
9. BAQUÉ, S., NEWGARD, C.B., GERARD, R.D., GUINOVARD, J.J., and GOMEZ-FOIX, A.M. (1994). Adenovirus-mediated delivery into myocytes of muscle glycogen phosphorylase, the enzyme deficient in patients with glycogen-sorage disease type V. Biochem. J. 304, 1009-1014.
10. BARBIN, G., MANTHORPE, M., and VARON, S. (1984). Purification of the chick eye ciliary neurotrophic factor. J. Neurochem. 43, 1468-1478.
11. BARR, E., and LEIDEN, J.M. (1991). Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509.
12. BERETTA, C., and ZURN, A.D. (1991). The neuropeptide VIP modulates the neurotransmitter phenotype of cultured chick sympathetic neurons. Dev. Biol. 148, 87-94.
13. BLAU, H.M., PAVLATH, G.K., HARDEMAN, E.C., CHIU, C.-P., SILBERSTEIN, L., WEBSTER, S.G., MILLER, S.C., and WEBSTER, C. (1985). Plasticity of the differentiated state. Science 230, 758-766.
14. CEDARBAUM, J.M., CHAPMAN, C., CHARATAN, M., STAMBLER, N., ANDREWS, L., ZHAN, C., RADKA, S., MORRISEY, D., LAKINGS, D., BROOKS, B.R., SANJAK, M., PESTRONK, A., FLORENCE, J., MITSUMOTO, H., SZIRONY, K., BITTLE, L., NEVILLE, H., RINGEL, S., BRINKMANN, J., and WITTES, J. (1995a). The phamacokinetics of subcutaneously administered recombinant human ciliary neurotrophic factor (rHCNTF) in patients with amyotrophic lateral sclerosis: relation to parameters of the acute-phase response. Clin. Neuropharmacol. 18, 500-514.
15. CEDARBAUM, J.M., CHAPMAN, C., CHARATAN, M., STAMBLER, N., ANDREWS, L., ZHAN, C., RADKA, S., MORRISEY, D., LAKINGS, D., BROOKS, B.R., SANJAK, M., PESTRONK, A., FLORENCE, J., MITSUMOTO, H., SZIRONY, K., BITTLE, L., NEVILLE, H., RINGEL, S., BRINKMANN, J., and WITTES, J. (1995b). A phase I sutdy of recombinant human ciliary neurotrophic factor (rHCNTF) in patients with amyotrophic lateral sclerosis. Clin. Neuropharmacol. 18, 515-532.
16. CHANG, P.L., SHEN, N., and WESTCOTT, A.J. (1993). Delivery of recombinant gene products with microencapsulated cells in vivo. Hum. Gene Ther. 4, 433-440.
17. DAHLER, A., WADE, R.P., MUSCAT, G.E.O., and WATERS, M.J. (1994). Expression vectors encoding human growth hormone (hGH) controlled by human muscle-specific promoters: prospects for regulated production of hGH delivered by myoblast transfer or intravenous injection. Gene 145, 305-310.
18. DAI, Y., ROMAN, M., NAVIAUX, R.K., and VERMA, I.M. (1992). Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo. Proc. Natl. Acad. Sci. USA 89, 10892-10895.
19. DAVIES, A.M. (1994). The role of neurotrophins in the developing nervous system. J. Neurobiol. 25, 1334-1348.
20. DHAWAN, J., PAN, L.C., PAVLATH, G.K., TRAVIS, M.A., LANCTOT, A.M., and BLAU, H.M. (1991). Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254, 1509-1512.
21. DITTRICH, F., THOENEN, H., and SENDTNER, M. (1994). Ciliary neurotrophic factor: pharmacokinetics and acute-phase response in rat. Ann. Neurol. 35, 151-163.
22. DÉGLON, N., WILSON, A., DESPONDS, C., LAURENT, P., BRON, C., and FASEL, N. (1995). Fatty acids regulate Thy-1 antigen mRNA stability in T lymphocyte precursors. Eur. J. Biochem. 231, 687-696.
23. EMERICH, D.F., HAMMANG, J.P., BAETGE, E.E., and WINN, S.R. (1994). Implantation of polymer-encapsulated human nerve growth factor-secreting fibroblasts attenuates the behavioral and neuropathological consequences of quinolinic acid injections into rodent striatum. Exp. Neurol. 130, 141-150.
24. ERNFORS, P., LEE, K.-F., KUCERA, J., and JAENISCH, R. (1994). Lack of neurotrophin-3 leads to deficiencies in the peripheral nervous system and loss of limb proprioceptive afferents. Cell 77, 503-512.
25. FRIM, D.M., UHLER, T.A., GALPERN, W.R., BEAL, M.F., BREAKEFIELD, X.O., and 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.
26. FURMAN, P.A., MCGUJIRT, P.V., KELLER, P.M., FYFE, J.A., and ELION, G.B. (1980). Inhibition of acyclovir cell growth and DNA synthesis of cells biochemically transformed with herpesvirus genetic information. Virology 102, 420-430.
27. HOFFMAN, D., BREAKEFIELD, X.O., SHORT, M.P., and AEBISCHER, P. (1993). Transplantation of a polymer-encapsulated cell line genetically engineered to release NGF. Exp. Neurol. 122, 100-106.
28. IKEDA, K., WONG, V., HOLMLUND, T.H., GREENE, T., CEDARBAUM, J.M., LINDSAY, R.M., and MITSUMOTO, H. (1995). Histometric effects of ciliary neurotrophic factor in wobbler mouse motor neuron disease. Ann. Neurol. 37, 47-54.
29. IP, N.Y., NYE, S.H., BOULTON, T.G., DAVIS, S., TAGA, T., LI, Y., BIRREN, S.J., YASUKAWA, K., KISHIMOTO, T., ANDERSON, D.J., STAHL, N., and YANCOPOULOS, G.D. (1992). CNTF and LIF act on neuronal cells via shared signaling pathways that involve the IL-6 signal transducing receptor component gp130. Cell 69, 1121-1132.
30. JIN, P., RAHM, M., CLAESSON-WELSH, L., HELDIN, C.-H., and SEJERSEN, T. (1990). Expression of PDGF A-chain and b-receptor genes during rat myoblast differentiation. J. Cell Biol. 110, 1665-1672.
31. JONES, K.R., FARIÑAS, I., BACKUS, C., and REICHARDT, L.F. (1994). Targeted disruption of the BDNF gene perturbs brain and sensory neuron development but not motor neuron development. Cell 76, 989-999.
32. JONGENEEL, C.V., NEDOSPASOV, S.A., PLAETINCK, G., NAQUET, P., and CEROTTINI, J.-C. (1988). Expression of the tumor necrosis factor locus is not necessary for the cytolytic activity of T lymphocytes. J. Immunol. 140, 1916-1922.
33. JOSEPH, J.-M., GODDARD, M.B., MILLS, J., PADRUN, V., ZURN, A., ZIELINSKI, B., FAVRE, J., GARDAZ, J.P., MOSIMANN, F., SAGEN, J., CHRISTENSON, L., and AEBISCHER, P. (1994). Transplantation of encapsulated bovine chromaffin cells in the sheep subarachnoid space: a preclinical study for the treatment of cancer pain. Cell Transpl. 3, 355-364.
34. KAUFMAN, R.J. (1990). Selection and coamplification of heterologous genes in mammalian cells. Methods Enzymol. 185, 537-566.
35. KEMBALL-COOK, G., GARNER, I., IMANAKA, Y., NISHIMURA, T., O'BRIEN, D.P., TUDDENHAM, E.G.D., and MCVEY, J.H. (1994). High-level production of human blood coagulation factors VII and XI using a new mammalian expression vector. Gene 139, 275-279.
36. KLEIN, R., SMEYNE, R.J., WURST, W., LONG, L.K., AUERBACH, B.A., JOYNER, A.L., and BARBACID, M. (1993). Targeted disruption of the trkB neurotrophin receptor gene results in nervous system lesions and neonatal death. Cell 75, 113-122.
37. KLEIN, R., SILOS-SANTIAGO, I., SMEYNE, R.J., LIRA, S.A., BRAMBILLA, R., BRYANT, S., ZHANG, L., SNIDER, W.D., and BARBACID, M. (1994). Disruption of the neurotrophin-3 receptor gene trkC eliminates Ia muscle afferents and results in abnormal movements. Nature 368, 249-251.
38. LEE, K.-F., HUBER, J., LANDIS, S.C., SHARPE, A.H., CHAO, M.V., and JAENISCH, R. (1992). Targeted mutation of the gene encoding the low affinity NGF receptor p75 leads to deficits in the peripheral sensory nervous system. Cell 69, 737-749.
39. LINDSAY, R.M., WIEGAND, S.J., ALTAR, C.A., and DISTEFANO, P.S. (1994). Neurotrophic factors: from molecule to man. Trends Neural Sci. 17, 182-190.
40. LIU, H.W., OFOSU, F.A., and CHANG, P.L. (1993). Expression of human factor IX by microencapsulated recombinant fibroblasts. Hum. Gene Ther. 4, 291-301.
41. MAGAL, E., BRUNHAM, P., and VARON, S. (1991). Effects of ciliary neurotrophic factor on rat spinal cord neuron in vitro: survival and expression of choline acetyltransferase and low-affinity nerve growth factor receptors. Dev. Brain Res. 63, 141-150.
42. MARTINOU, J.-C., MARTINOU, I., and KATO, A.C. (1992). Cholinergic differentiation factor (CDF/LIF) promotes survival of isolated rat embryonic motoneurons in vitro. Neuron 8, 737-744.
43. MASU, Y., WOLF, E., HOLTMANN, B., SENDTNER, M., BREM, G., and THOENEN, H. (1993). Disruption of the CNTF gene results in motor neuron degeneration. Nature 365, 27-32.
44. MELTON, D.A., KRIEG, P.A., REBAGLIATI, M.R., MANIATIS, T., ZINN, K., and GREEN, M.R. (1984). Efficient in vitro synthesis of biologically active RNA and hybridization probes from plasmids containing a bacteriophage promoter. Nucleic Acids Res. 12, 7035-7056.
45. 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.
46. MULLEN, C.A. (1994). Metabolic suicide genes in gene therapy. Pharmacol. Ther. 63, 199-207.
47. PLAUTZ, G., NABEL, E.G., and NABEL, G.J. (1991). Selective elimination of recombinant genes in vivo with a suicide retroviral vector. New Biol. 3, 709-715.
48. 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.
49. SAHENK, Z., SEHARASEYON, J., and MENDELL, J.R. (1994). CNTF potentiates peripheral nerve regeneration. Brain Res. 655, 246-250.
50. SENDTNER, M., KREUTZBERG, G.W., and THOENEN, H. (1990). Ciliary neurotrophic factor prevents the degeneration of motor neurons after axotomy. Nature 345, 440-441.
51. SENDTNER, M., SCHMALBRUCH, H., STÖCKLI, 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, 502-504.
52. SMEYNE, R.J., KLEIN, R., SCHNAPP, A., LONG, L.K., BRYANT, S., LEWIN, A., LIRA, S.A., and BARBACID, M. (1994). Severe sensory and sympathetic neuropathies in mice carrying a doisrupted trk/NGF receptor gene. Nature 368, 246-249.
53. TAN, S.A., DÉGLON, N., ZURN, A.D., BAETGE, E.E., BAMBER, B., KATO, A.C., and AEBISCHER, P. (1996). Rescue of motoneurons from axotomy induced cell death by polymer encapsulated cells genetically engineered to release CNTF. Cell Transpl. 5, 577-587.
54. TOJO, H., KAISHO, Y., NAKATA, M., MATSUOKA, K., KITAGAWA, M., ABE, T., TAKAMI, K., YAMAMOTO, M., SHINO, A., IGARASHI, K., AIZAWA, S., and SHIHO, O. (1995). Targeted disruption of the neurotrophin-3 gene with lacZ induces loss of trkC-positive neurons in sensory ganglia but not in spinal cords. Brain Res. 669, 163-175.
55. TRESCO, P.A., WINN, S.R., TAN, S., JAEGER, C.B., GREENE, L.A., and AEBISCHER, P. (1992). Polymer-encapsulated PC12 cells: long-term survival and associated reduction in lesion-induced rotational behavior. Cell Transpl. 1, 255-264.
56. WONG, V., ARRIAGA, R., IP, N.Y., and LINDSAY, R.M. (1993). The neurotrophins BDNF, NT-3 and NT-4/5, but not NGF, up-regulate the cholinergic phenotype of developing motoneurons. Eur. J. Neurosci. 5, 466-474.
57. YAO, S.-N., and KURACHI, K. (1992). Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc. Natl. Acad. Sci. USA 89, 3357-3361.
58. YAFFE, D. (1968). Retention of differentiation potentialities during prolonged cultivation of myogenic cells. Proc. Natl. Acad. Sci. USA 61, 477-483.
59. YAFFE, D., and FELDMAN, M. (1965). The formation of hybrid multinucleated muscle fibers from myoblasts of different genetic origin. Dev. Biol. 11, 300-307.
60. YAFFE, D., and SAXEL, O. (1977). Serial passaging and differentiation of myogenic cells isolated from dystrophic mouse muscle. Nature 270, 725-727.
61. ZURN, A.D., BAETGE, E.E., HAMMANG, J.P., TAN, S.A., and AEBISCHER, P. (1994). Glial cell line-derived neurotrophic factor (GDNF), a new neurotrophic for motoneurons. NeuroReport 6, 113-118.