Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectors

Human Gene Therapy

Volumn 16, Issue 1, 2005, Pages 49-56

  Watson, Deborah J ^b   Passini, Marco A ^b   Wolfe, John H ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR; VIRUS GLYCOPROTEIN;

ADENO ASSOCIATED VIRUS; ANIMAL TISSUE; ARTICLE; BRAIN DEVELOPMENT; BRAIN LATERAL VENTRICLE; CENTRAL NERVOUS SYSTEM; CHOROID PLEXUS; CONTROLLED STUDY; CYTOMEGALOVIRUS; EBOLA VIRUS; EPENDYMA; GENE TRANSFER; GENETIC TRANSDUCTION; LENTIVIRINAE; LONG TERMINAL REPEAT; MOUSE; NEWBORN; NONHUMAN; PROMOTER REGION; VESICULAR STOMATITIS VIRUS;

ANIMALS; ANIMALS, NEWBORN; BETA-GALACTOSIDASE; CHOROID PLEXUS; DEPENDOVIRUS; EPENDYMA; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; INJECTIONS, INTRAVENTRICULAR; LENTIVIRUS; MEMBRANE GLYCOPROTEINS; MICE; MICE, INBRED C3H; PLASMIDS; SEROTYPING; TRANSDUCTION, GENETIC; VESICULAR STOMATITIS-INDIANA VIRUS; VIRAL ENVELOPE PROTEINS; VIRAL VACCINES;

EID: 13844269142     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2005.16.49     Document Type: Article

References (39)

1. ALTMAN, J. (1969). Autoradiographic and histological studies of postnatal neurogenesis. IV. Cell proliferation and migration in the anterior forebrain, with special reference to persisting neurogenesis in the olfactory bulb. J. Comp. Neurol. 137, 433-453.
2. ALUR, R.K.P., FOLEY, B., PARENTE, M.K., TOBIN, D.K., HEUER, G.G., AVADHANI, A.N., PONGUBALA, J., and WOLFE, J.H. (2002). Modification of multiple transcriptional regulatory elements in a Moloney murine leukemia virus gene transfer vector circumvents silencing in fibroblast grafts and increases levels of expression of the transferred enzyme. Gene Ther. 9, 1146-1154.
3. BAEKELANDT, V., CLAEYS, A., EGGERMONT, K., LAUWERS, E., DE STROOPER, B., NUTTIN, B., and DEBYSER, Z. (2002). Characterization of lentiviral vector-mediated gene transfer in adult mouse brain. Hum. Gene Ther. 13, 841-853.
4. BAJOCCHI, G., FELDMAN, S.H., CRYSTAL, R.G., and MASTRANGELI, A. (1993). Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors. Nat. Genet. 3, 229-234.
5. BOSCH, A., PERRET, E., DESMARIS, N., TRONO, D., and HEARD, J. (2000). Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum. Gene Ther. 11, 1139-1150.
6. DAVIDSON, B.L., STEIN, C.S., HETH, J.A., MARTINS, I., KOTIN, R.M., DERKSEN, T.A., ZABNER, J., GHODSI, A., and CHIORINI, J.A. (2000). Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system. Proc. Natl. Acad. Sci. U.S.A. 97, 4430-4433.
7. DEL BIGIO, M.R. (1995). The ependyma: A protective barrier between brain and cerebrospinal fluid. Glia 14, 1-13.
8. DESMARIS, N., BOSCH, A., SALAUN, C., PETIT, C., PREVOST, M.C., TORDO, N., PERRIN, P., SCHWARTZ, O., DE ROCQUIGNY, H., and HEARD, J.M. (2001). Production and neurotropism of lentivirus vectors pseudotyped with lyssavirus envelope glycoproteins. Mol. Ther. 4, 149-156.
9. DI PASQUALE, G., DAVIDSON, B.L., STEIN, C.S., MARTINS, I., SCUDIERO, D., MONKS, A., and CHIORINI, J. (2003). Identification of PDGFR as a receptor for AAV-5 transduction. Nat. Med. 9, 1306-1312.
10. DOETSCH, F., GARCIA-VERDUGO, J.M., and ALVAREZ-BUYLLA, A. (1997). Cellular composition and three-dimensional organization of the subventricular germinal zone in the adult mammalian brain. J. Neurosci. 17, 5046-5061.
11. DRIESSE, M.J., KROS, J.M., AVEZAAT, C.J., VALERIO, D., VECHT, C.J., BOUT, A., and SMITT, P.A. (1999). Distribution of recombinant adenovirus in the cerebrospinal fluid of nonhuman primates. Hum. Gene Ther. 10, 2347-2354.
12. DRIESSE, M.J., ESANDI, M.C., KROS, J.M., AVEZAAT, C.J., VECHT, C., ZURCHER, C., VAN DER VELDE, I., VALERIO, D., BOUT, A., and SILLEVIS SMITT, P.A. (2000). Intra-CSF administered recombinant adenovirus causes an immune response-mediated toxicity. Gene Ther. 7, 1401-1409.
13. ELLIGER, S.S., ELLIGER, C.A., AGUILAR, C.P., RAJU, N.R., and WATSON, G.L. (1999). Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector. Gene Ther. 6, 1175-1178.
14. ELLIGER, S.S., ELLIGER, C.A., LANG, C., and WATSON, G.L. (2002). Enhanced secretion and uptake of β-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice. Mol. Ther. 5, 617-626.
15. FURLAN, R., POLIANI, P.L., GALBIATI, F., BERGAMI, A., GRIMALDI, L.M., COMI, G., ADORINI, L., and MARTINO, G. (1998). Central nervous system delivery of interleukin 4 by a non-replicative herpes simplex type 1 viral vector ameliorates autoimmune demyelination. Hum. Gene Ther. 9, 2605-2617.
16. FURLAN, R., POLIANI, P.L., BRAMBILLA, E., MARCONI, P.C., BERGAMI, A., DESINA, G., GLORIOSO, J.C., COMI, G., and MARTINO, G. (2001). Delivery to the central nervous system of a nonreplicative herpes simplex type 1 vector engineered with the interleukin 4 gene protects rhesus monkeys from hyperacute autoimmune encephalomyelitis. Gene Ther. 8, 1207-1213.
17. HEUER, G.G., PASSINI, M.A., JIANG, K., PARENTE, M.K., LEE, V.M., TROJANOWSKI, J.Q., and WOLFE, J.H. (2002). Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible. Ann. Neurol. 52, 762-770.
18. HILDINGER, M., AURICCHIO, A., GAO, G., WANG, L., CHIRMULE, N., and WILSON, J.M. (2001). Hybrid vectors based on adeno-associated virus serotypes 2 and 5 for muscle-directed gene transfer. J. Virol. 75, 6199-6203.
19. KOBINGER, G.P., WEINER, D.J., YU, Q.C., and WILSON, J.M. (2001). Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat. Biotechnol. 19, 225-230.
20. LUSKIN, M.B. (1993). Restricted proliferation and migration of postnatally generated neurons derived from the forebrain subventricular zone. Neuron. 11, 173-189.
21. MAZARAKIS, N.D., AZZOUZ, M., ROHLL, J.B., ELLARD, F.M., WILKES, F.J., OLSEN, A.L., CARTER, E.E., BARBER, R.D., BABAN, D.F., KINGSMAN, S.M., KINGSMAN, A.J., O'MALLEY, K., and MITROPHANOUS, K.A. (2001). Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery. Hum. Mol. Genet. 10, 2109-2121.
22. MOCHIZUKI, H., SCHWARTZ, J.P., TANAKA, K., BRADY, R.O., and REISER, J. (1998). High-liter human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J. Virol. 72, 8873-8883.
23. NALDINI, L., BLOMER, U., GAGE, F.H., TRONO, D., and VERMA, I.M. (1996a). Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. U.S.A. 93, 11382-11388.
24. NALDINI, L., BLOMER, U., GALLAY, P., ORY, D., MULLIGAN, R., GAGE, F.H., VERMA, I.M., and TRONO, D. (1996b). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267.
25. OHASHI, T., WATABE, K., UEHARA, K., SLY, W.S., VOGLER, C., and ETO, Y. (1997). Adenovirus-mediated gene transfer and expression of human β-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice. Proc. Natl. Acad. Sci. U.S.A. 94, 1287-1292.
26. OUMESMAR, B.N., VIGNAIS, L., and BARON-VAN EVERCOOREN, A. (1997). Developmental expression of platelet-derived growth factor alpha-receptor in neurons and glial cells of the mouse CNS. J. Neurosci. 17, 125-139.
27. PASSINI, M.A., and WOLFE, J.H. (2001). Widespread gene delivery and structure-specific patterns of expression from an adeno-associated virus vector following intraventricular injections of neonatal mice. J. Virol. 75, 12382-12392.
28. PASSINI, M.A., LEE, E.B., HEUER, G.G., and WOLFE, J.H. (2002). Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream. J. Neurosci. 22, 6437-6446.
29. PASSINI, M., WATSON, D.J., VITE, C.H., LANDSBURG, D., FEIGENBAUM, A., and WOLFE, J.H. (2003). Intraventricular brain injection of AAV1 in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of β-glucuronidase- deficient mice. J. Virol. 77, 7034-7040.
30. POLIANI, P.L., BROK, H., FURLAN, R., RUFFINI, F., BERGAMI, A., DESINA, G., MARCONI, P.C., ROVARIS, M., UCCELLI, A., GLORIOSO, J.C., PENNA, G., ADORINI, L., COMI, G., 't HART, B., and MARTINO, G. (2001). Delivery to the central nervous system of a nonreplicative herpes simplex type 1 vector engineered with the interleukin 4 gene protects rhesus monkeys from hyperacute autoimmune encephalomyelitis. Hum. Gene Ther. 12, 905-920.
31. SANCHEZ-RAMOS, J., SONG, S., DAILEY, D., CARDOZOPELAEZ, F., HAZZI, C., STEDEFORD, T., WILLING, A., FREEMAN, T., SAPORTA, S., ZIGOVA, T., SANBERG, P., and SNYDER, E. (2000). The X-Gal caution in neural transplantation studies. Cell Transplant. 9, 657-667.
32. SARNAT, H.B. (1995). Ependymal reactions to injury: A review. J. Neuropathol. Exp. Neurol. 54, 1-15.
33. SKORUPA, A.F., FISHER, K.J., WILSON, J.M., PARENTE, M.K., and WOLFE, J.H. (1999). Sustained production of β-glucuronidase from localized sites after AAV vector gene transfer results in wide-spread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice. Exp. Neurol. 160, 17-27.
34. SNYDER, E.Y., TAYLOR, R.M., and WOLFE, J.H. (1995). Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature 374, 367-370.
35. SPRINGER, S.L., VITE, C.H., POLESKY, A.C., KESARI, S., FRASER, N.W., and WOLFE, J.H. (2001). Infection and establishment of latency in the dog brain after direct inoculation of a non-pathogenic strain of herpes simplex virus-1. J. Neurovirol. 7, 149-154.
36. VITE, C.H., PASSINI, M.A., HASKINS, M.E., and WOLFE, J.H. (2003). Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther. 10, 1874-1881.
37. WATSON, D., and WOLFE, J. (2002). Lentiviral vectors for gene transfer to the central nervous system: Applications in lysosomal storage disease animal models. In: Viral Vectors for Gene Therapy: Methods and Protocols. C. Machida, ed. (Humana Press, Totowa, NJ) pp. 383-403.
38. WATSON, D.J., KOBINGER, G.P., PASSINI, M.A., WILSON, J.M., and WOLFE, J.H. (2002). Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV or MuLV envelope proteins. Mol. Ther. 5, 528-537.
39. WOOL-LEWIS, R.J., and BATES, P. (1998). Characterization of Ebola virus entry by using pseudotyped viruses: identification of receptor-deficient cell lines. J. Virol. 72, 3155-3160.