Gene expression along the cerebral-spinal axis after regional gene delivery

Human Gene Therapy

Volumn 10, Issue 16, 1999, Pages 2689-2700

  Meuli Simmen, Claudia ^a   Liu, Yong ^b   Yeo, Tracy T ^c   Liggitt, Denny ^d   Tu, Guanghuan ^b   Yang, Tao ^c   Meuli, Martin ^e   Knauer, Sharon ^b   Heath, Timothy D ^f   Longo, Frank M ^c   Debs, Robert J ^b  

^a UNIVERSITY HOSPITAL ZURICH   (Switzerland)

^b CALIFORNIA PACIFIC MEDICAL CENTER   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^e UNIVERSITY CHILDREN S HOSPITAL   (Switzerland)

^f UNIVERSITY OF WISCONSIN MADISON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; CHLORAMPHENICOL ACETYLTRANSFERASE; CHOLINE ACETYLTRANSFERASE; GRANULOCYTE COLONY STIMULATING FACTOR; LIPOSOME; NERVE GROWTH FACTOR; PLASMID DNA;

ARTICLE; BRAIN; BRAIN CELL; CONTROLLED STUDY; FEMALE; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; GENETIC TRANSFECTION; HIPPOCAMPUS; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; INTRATHECAL DRUG ADMINISTRATION; MOUSE; NONHUMAN; REPORTER GENE; SPINAL CORD;

ANIMALS; BETA-GALACTOSIDASE; BRAIN CHEMISTRY; CENTRAL NERVOUS SYSTEM; CHLORAMPHENICOL O-ACETYLTRANSFERASE; DNA; DOSAGE FORMS; FEMALE; GENE EXPRESSION REGULATION; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GRANULOCYTE COLONY-STIMULATING FACTOR; HUMANS; INJECTIONS, INTRAVENTRICULAR; INJECTIONS, SPINAL; LIPOSOMES; MICE; MICE, INBRED ICR; NERVE GROWTH FACTOR; PLASMIDS; SPINAL CORD; TISSUE DISTRIBUTION;

FELIS CATUS; MURINAE;

EID: 0033231104     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950016735     Document Type: Article

References (33)

1. BARINAGA, M. (1994). Neurotrophic factors enter the clinic. Science 264, 772-774.
2. BUELER, H., AGUZZEI, A., SAILER, A., GREINER, R.A., AUTENRIED, P., and WEISSMANN, C. (1993). Mice devoid of PrP are resistant to scrapie. Cell 73, 1339-1347.
3. FISCHER, W., WICTORIN, K., BJORKLUND, A., WILLIAMAS, L.R., VARON, S., and GAGE, F.H. (1987). Amelioration of cholinergic neuron atrophy and spatial memory impairment in aged rats by nerve growth factor. Nature (London) 329, 65-68.
4. FOOKS, A.R., JENNINGS, A.D., DOYLE, A., CLEGG, J.C., and STAGEY, G.N. (1997). Neurological gene therapy. Sci. Prog. 80(Pt. 2), 107-124.
5. FRANKLIN, K.B.J., and PAXINOS, G. (1997). The Mouse Brain in Stereotaxic Coordinates. (Academic Press, San Diego, CA).
6. GNAHN, H., HEFTI, F., HEUMANN, R., CHWAB, M.E., and THOENEN, H. (1983). NGF-medicated increase of choline acetyltransferase (ChAT) in the neonatal rat forebrain: Evidence for a physiological role of NGF in the brain. Dev. Brain Res. 9, 45-52.
7. GOULA, D., REMY, J.S., ERBACHER, P., WASCOWICZ, M., LEVI, G., ABDALLAH, B., and DEMENEIX, B.A. (1998). Size, diffusibility and transfection performance of linear PEI/DNA complexes in the mouse central nervous system. Gene Ther. 5, 712-717.
8. HAASE, G., PETTMANN, B., BORDER, T., VILLA, P., VIGNE, E., SCHMALBRUCH, H., and KAHN, A. (1999). Therapeutic benefit of ciliary neurotrophic factor in progressive motor neuronopathy depends on the route of delivery. Ann. Neurol. 45, 296-304.
9. HAGG, T., PASS-HOLMES, B., VAHLSING, H.L., MANTHORPE, M., CONNER, J.M., and VARON, S. (1989). Nerve growth factor (NGF) reverses axotomy-induced decreases in choline acetyltransferase, NGF receptor and size of medial septum cholinergic neurons. Brain Res. 505, 29-38.
10. HEFTI, F. (1986). Nerve growth factor promotes survival of septal cholinergic neurons after fimbrial transections. J. Neurosci. 8, 2155-2162.
11. HEFTI, F. (1997). Pharmacology of neurotrophic factors. Annu. Rev. Pharmacol. Toxicol. 37, 239-267.
12. JOHNSTON, M.V., RUTKOWSKI, J.L., WAINER, B.H., LONG, J.B., and MOBLEY, W.C. (1987). NGF effects on developing forebrain cholinergic neurons are regionally specific. Neurochem. Res. 12, 985-994.
13. KENNEDY, P.G. (1997). Potential use of herpes simplex virus (HSV) vectors for gene therapy of neurological disorders. Brain 120 (Pt. 7), 1245-1259.
14. KOLB, B., COTE, S., RIBEIRO-DA-SILVA, A., and CUELLO, A.C. (1996). Nerve growth factor treatment prevents dendtritic atrophy and promotes recovery of function after cortical injury. Neuroscience 76, 1139-1151.
15. LEWIN, G.R., and MENDELL, L.M. (1993). Nerve growth factor and nociception. Trends Neurosci. 16, 353-359.
16. LIU, Y., LIGGITT, D., ZHONG, W., TU, G., GAENSLER, K., and DEBS, R. (1995). Cationic liposome-mediated intravenous gene delivery. J. Biol. Chem. 270, 24864-24870.
17. LIU, Y., MOUNKES, L.C., LIGGITT, H.D., BROWN, C.S., SOLODIN, I., HEATH, T.D., and DEBS, R.J. (1997). Factors influencing the efficiency of cationic liposome mediated intravenous gene delivery. Nature Biotechnol. 15, 167-173.
18. LONGO, F.M., HOLTZMAN, D.M., GRIMES, M.L., and MOBLEY, W.C. (1993). Nerve growth factor actions in the PNS and CNS. In Neurotrophic Factors. S.E. Loughlin and J.H. Fallon, eds. (Academic Press. San Diego, CA) pp. 209-256.
19. MILLER, R.G., and SUFIT, R. (1997). New approaches to the treatment of ALS. Neurology 48, S28-S32.
20. MOBLEY, W.C., RUTKOWSKI, J.L., TENNEKOON, G.I., GEMSKI, J., BUCHANAN, K., and JOHNSTON, M.V. (1986). Nerve growth factor increase choline acetyl-transferase activity in developing basal forebrain neurons. Mol. Brain Res. 1, 53-62.
21. OPPENHEIM, R.W., HOUENOU, L.J., JOHNSON, J.E., LIN, L.F., LI, L., LO, A.C., NEWSOME, A.L., PREVETTE, D.M., and WANG, S. (1995). Developing motor neurons rescues from programmed and axotomy-induced cell death by GDNF. Nature (London) 373, 344-346.
22. 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. Mol. Brain Res. 36, 280-286.
23. SENTNER, M., SCHMALBRUCH, H., STOECKLI, 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 (London) 358, 502-504.
24. SNYDER, E.Y., and FISHER, L.J. (1996). Gene therapy in neurology. Curr. Opin. Pediatr. 8, 558-568.
25. SOFRONIEW, M.V., and COOPER, J.D. (1993). Neurotrophic mechanisms and neuronal degeneration. Semin. Neurosci. 5, 285-294.
26. SOLODIN, I., BROWN, C.S., BRUNO, M.S., CHOW, C.Y., JANG, E.H., DEBS, R.J., and HEATH, T.D. (1995). A novel series of amphiphilic imidazolium compounds for in vitro and in vivo gene delivery. Biochemistry 34, 13537-13544.
27. TUSZYNSKI, M.H., and GAGE, F.H. (1996). Somatic gene therapy for nervous system disease. CIBA Found. Symp. 196, 85-94.
28. YANG, K., CLIFTON, G.L., and HAYES, R.L. (1997). Gene therapy for central nervous system injury: The use of cationic liposomes. An invited review. J. Neurotrauma 14, 281-297.
29. YEO, T.T., CHUA-COUZENS, J., BUTCHER, L.L., BREDESEN, D.E., COOPER, J.D., VALETTA, J.S., MOBLEY, W.C., and LONGO, P.M. (1997). Absence of p75NTR causes increased basal forebrain cholinergic neuron size, choline acetyltransferase activity, and target innervation. J. Neurosci. 17, 7594-7605.
30. YUEN, E.C., and MOBLEY, W.C. (1996). Therapeutic potential of neurotrophic factors for neurological disorders. Ann. Neurol. 40, 346-354.
31. ZELPHATI, O., LIANG, X., HOBART, P., and FELGNER, P.L. (1999). Gene chemistry: Functionally and conformationally intact fluorescent plasmid DNA. Hum. Gene Ther. 10, 15-24.
32. ZHU, N., LIGGITT, D., LIU, Y., and DEBS, R. (1993). Systemic gene expression after intravenous DNA delivery into adult mice. Science 261, 209-211.
33. ZLOKOVIC, B.V., and APUZZO, M.L. (1997). Cellular and molecular neurosurgery: Pathways from concept to reality. II. Vector systems and delivery methodologies for gene therapy of the central nervous system. Neurosurgery 40, 805-812.