Stability of mRNA/cationic lipid lipoplexes in human and rat cerebrospinal fluid: Methods and evidence for nonviral mrna gene delivery to the central nervous system

Human Gene Therapy

Volumn 14, Issue 3, 2003, Pages 191-202

  Anderson, Dua M ^a   Hall, Leon L ^a   Ayyalapu, Anitha R ^a   Irion, Van R ^a   Nantz, Michael H ^a   Hecker, James G ^b  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CATION; CATIONIC LIPID; DNA; ENZYME INHIBITOR; GREEN FLUORESCENT PROTEIN; HEAT SHOCK PROTEIN 70; LIPID; LIPID MEDIATED MESSENGER RNA; LIPOPLEX; LUCIFERASE; MESSENGER RNA; N [1 (2,3 DIOLEOYLOXY)PROPYL] N [1 (2 HYDROXY)ETHYL] N,N DIMETHYLAMMONIUM IODIDE; RIBONUCLEASE; RIBONUCLEASE INHIBITOR; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; BRAIN SLICE; CATIONIC LIPID MEDIATED MESSENGER RNA DELIVERY; CELL POPULATION; CELL SPECIFICITY; CENTRAL NERVOUS SYSTEM DISEASE; CEREBROSPINAL FLUID ANALYSIS; COMPLEX FORMATION; CONTROLLED STUDY; CYTOTOXICITY; DNA VECTOR; ENZYME ACTIVITY; GEL ELECTROPHORESIS; GENE DELIVERY SYSTEM; GENE EXPRESSION REGULATION; GENE SEQUENCE; GENE THERAPY; GENE TRANSFER; GENETIC CODE; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; IN VITRO STUDY; INCUBATION TIME; LATERAL BRAIN VENTRICLE; LIPID MEDIATED MESSENGER RNA DELIVERY; LIPID METABOLISM; MESSENGER RNA CATIONIC COMPLEX; MESSENGER RNA VECTOR; NEUROPROTECTION; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PROTEIN DEGRADATION; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PROTEIN TRANSPORT; RAT; REPORTER GENE; REPRODUCIBILITY; RNA ANALYSIS; RNA BINDING; RNA STABILITY; RNA TRANSLATION; TIME; TRANSCRIPTION REGULATION;

ANIMALS; CENTRAL NERVOUS SYSTEM; CHO CELLS; CRICETINAE; GENE TRANSFER TECHNIQUES; GENES, REPORTER; HUMANS; LIPID METABOLISM; LIPIDS; RATS; RNA, MESSENGER; TRANSFECTION;

ANIMALIA; CRICETINAE; CRICETULUS GRISEUS;

EID: 0037429077     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430340360535751     Document Type: Article

References (39)

1. ALLINQUANT, B., MUSENGER, C., and SCHULLER, E. (1984). Intrathecal origin of CSF ribonuclease: Differences between infectious processes of the nervous system and multiple sclerosis. Acta Neurol. Scand. 69, 12-19.
2. BALASUBRAMANIAM R.P., BENNETT M.J., ABERLE A.M., MALONE J.G., NANTZ M.H., and MALONE R.W. (1996). Structural and functional analysis of cationic transfection lipids: The hydrophobic domain. Gene Ther. 3, 163-172.
3. BENNETT M.J., ABERLE A.M., BALASUBRAMANIAM R.P., MALONE J.G., NANTZ M.H., and MALONE R.W. (1996). Considerations for the design of improved cationic amphiphile-based transfection reagents. J. Liposome Res. 6, 545-565.
4. BENNETT M.J., MALONE R.W., and NANTZ M.H. (1995). A flexible approach to synthetic lipid ammonium salts for polynucleotide transfection. Tetrahedron Lett. 36, 2207-2210.
5. BENNETT, M.J., ABERLE, A.M., BALASUBRAMANIAM, R.P., MALONE, J.G., MALONE, R.W., and NANTZ, M.H. (1997). Cationic lipid-mediated gene delivery to murine lung: correlation of lipid hydration with in vivo transfection activity. J. Med. Chem. 40, 4069-4078.
6. CAO Y.-J., SHIBATA, T., and RAINOV, N.G. (2002). Liposome-mediated transfer of the bcl-2 gene results in neuroprotection after in vivo transient focal cerebral ischemia in an animal model. Gene Ther. 9, 415-419.
7. CHESNOY, S., and HUANG, L. (2000). Structure and function of lipid-DNA complexes for gene delivery. Annu. Rev. Biophys. Biomol. Struct. 29, 27-47.
8. DRIESSE, M.J., ESANDI, M.C., KROS, J.M., AVEZAAT, C.J.J., VECHT, CH.J., ZURCHER, V., VAN DER VELDE, I., VALERIO, D., BOUT, A., and SILLEVIS SMITT, P.A.E. (2000). Intra-CSF administered recombinant adenovirus causes an immune response-mediated toxicity. Gene Ther. 7, 1401-1409.
9. ENZMANN, D.R., and PELC, N.J. (1993). Cerebrospinal fluid flow measured by phase-contrast cine MR. Am. J. Neuroradiol. 14, 1301-1310.
10. FELGNER, P.L., and RINGOLD, G.M. (1989). Cationic liposome-mediated transfection. Nature 337, 387-388.
11. FELGNER, J.H., KUMAR, R., SRIDHAR, C.N., WHEELER, C.J., TSAI, Y.J., BORDER, R., RAMSEY, P., MARTIN, M., and FELGNER, P.L. (1994). Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. 269, 2550-2561.
12. FUJII, S.K., NANTZ, M.H., and HECKER, J.G. (2001). A comparison of mRNA transfection efficacy and cytotoxicity of five lipofectins in five mammalian cell lines. Mol. Ther. 3(5 Part 2):582.
13. GALLIE, D.R., FEDER, J.N., SCHIMKE, R.T., and WALBOT, V. (1991). Functional analysis of the tobacco mosaic virus tRNA-like structure in cytoplasmic gene regulation. Nucleic Acids Res. 19, 5031-5036.
14. GALLIE, D.R., and WALBOT, V. (1990). RNA pseudoknot domain of tobacco mosaic virus can functionally substitute for a poly(A) tail in plant and animal cells. Genes Dev. 4, 1149-1157.
15. GREITZ, D., and HANNERZ, J. (1996). A proposed model of cerebrospinal fluid circulation: Observations with radionuclide cisternography. Am. J. Neuroradiol. 17, 431-438.
16. HAGIHARA, Y., SAITOH, Y., KANEDA, Y., KOHMURA, E., and YOSHIMINE, T. (2000). Widespread gene transfection into the central nervous system of primates. Gene Ther. 7, 759-763.
17. HECKER, J.G., and IRION, V.R. (1998). Advances in self-limited gene expression of protective intracellular proteins in vivo in rat brain using mRNA/cationic lipid complexes. Anesth. Analg. 86, S346.
18. HECKER, J.G., HALL, L.L., and IRION, V.R. (2001). Nonviral gene delivery to the lateral ventricles in rat brain: Initial evidence for widespread distribution and expression in the central nervous system. Mol. Ther. 3, 375-384.
19. KARIKO, K., KELLER, J.M., HARRIS, V.A., LANGER, D.J., and WELSH, F.A. (2001). In vivo protein expression from mRNA delivered into adultrat brain. J. Neurosci. Methods 105, 77-86.
20. KOFLER, P., WIESENHOFER, B., REHRL, C., BAIER, G., STOCK-HAMMER, G., and HUMPEL, C. (1998). Liposome-mediated gene transfer into established CNS cell lines, primary glial cells, and in vivo. Cell Transplant. 7, 175-185.
21. KRIEG P.A., and MELTON, D.A. (1984). Functional messenger RNAs are produced by SP6 in vitro transcription of cloned cDNAs. Nucleic Acids Res. 12, 7057-7070.
22. LEONE, P., JANSON, C.G., BILIANUK, L., WANG, Z., SORGI, F., HUANG, L., MATALON, R., KAUL, R., ZENG, Z., and FREESE, A. (2000). Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan Disease. Ann. Neurol. 48, 27-38.
23. LEVENTIS, R., and SILVIUS, JR. (1990). Interactions of mammalian cells with lipid dispersions containing novel metabolizable cationic amphiphiles. Biochim. Biophys. Acta 1023, 124-132.
24. LIN, M.T.S., PULKKINEN, L., UITTO, J., and YOON, K. (2000). The gene gun: Current applications in cutaneous gene therapy. Int. J. Dermatol. 39, 161-170.
25. LU, D., BENJAMIN, R. KIM, M., CONRY, R.M., and CURIEL, D.T. (1994). Optimization of methods to achieve mRNA-mediated transfection of tumor cells in vitro and in vivo employing cationic liposome vectors. Cancer Gene Ther. 1, 245-252.
26. MALONE, R.W., FELGNER, P.L., and VERMA, I.M. (1989). Cationic liposome-mediated RNA transfection. Proc. Natl. Acad. Sci. U.S.A. 86, 6077-6081.
27. MEULI-SIMMEN, C., LIU, Y., YEO, T.T., LIGGITT, D., TU, G., YANG, T., MUELI, M., KNAUER, S., HEATH, T.D., LONGO, F.M., and DEBS, R.J. (1999). Gene expression along the cerebralspinal axis after regional gene delivery. Hum. Gene Ther. 10, 2689-2700.
28. NANTZ, M.H., BENNETT, M.J., and BALASUBRAMANIAM, R.P. (1999). Polyfunctional cationic cytofectins. U.S. Patent No. 5,869,715.
29. NANTZ, M.H., BENNETT, M.J., and MALONE, R.W. (1996) Cationic Transport Reagents. U.S. Patent No. 5,527,928.
30. NISHIKAWA, M., and HUANG, L. (2001). Nonviral vectors in the new millennium: Delivery barriers in gene transfer. Hum. Gene Ther. 12, 861-870.
31. PAXINOS G., and WATSON, C. (1986). The Rat Brain in Stereotaxic Coordinates. (Academic Press, San Diego, CA).
32. ROSS, J. (1988). Messenger RNA turnover in eukaryotic cells. Mol. Biol. Med. 5, 1-14.
33. ROSS, J. (1989). The turnover of messenger RNA. Sci. Am. 260, 48-55.
34. ROTBART, H.A., LEVIN, M.J., MURPHY, N.L., and ABZUG, M.J. (1987). RNA target loss during solid phase hybridization of body fluids: A quantitative study. Mol. Cell. Probes 1, 347-358.
35. SCHIEVEN, G.L., BLANK, A., and DEKKER, C.A. (1982). Ribonucleases of human cerebrospinal fluid: Detection of altered glycosylation relative to their serum counterparts. Biochemistry 21, 5148-5155.
36. STRONG, T.V., HAMPTON, T.A., LOURO, I., BILBAO, G., CONRY, R.M., and CURIEL, D.T. (1997). Incorporation of β-globin untranslated regions into a Sindbis virus vector for augmentation of heterologous mRNA expression. Gene Ther. 4, 624-627.
37. TANGUAY, R.L., and GALLIE, D.R. (1996). Isolation and characterization of the 102-kilodalton RNA-binding protein that binds to the 5′ and 3′ translational enhancers of tobacco mosaic virus RNA. J. Biol. Chem. 271, 14316-14322.
38. WOLFF, J.A., MALONE, R.W., WILLIAMS, P., CHONG, W., ACSADI, G., JANI, A., and FELGNER, P.L. (1990). Direct gene transfer into mouse muscle in vivo. Science 247, 1465-1468.
39. YANG, J.P., and HUANG, L. (1998). Time-dependent maturation of cationic liposome-DNA complex for serum resistance. Gene Ther. 5, 380-387.