SpringerProtocols: Abstract: 7. Gene Transfer to Brain and Spinal Cord Using Recombinant Adenoviral Vectors

7. Gene Transfer to Brain and Spinal Cord Using Recombinant Adenoviral Vectors

By: Joseph M. Alisky², Beverly L. Davidson³

Abstract

Full Text

Download PDF (201K)

Recombinant adenoviral (Ad) vectors are derived from human adenoviruses: nonenveloped, encapsidated linear, double-stranded DNA viruses that commonly cause respiratory and gastrointestinal infections. Forty-three different human adenovirus serotypes have been characterized (1). Details about production of recombinant Ad vectors are given in Chapter 1. Ad vectors in widespread use are derived from human Ad serotypes 2 and 5 (Ad2 and Ad5), Ad5 being more common for applications in the central nervous system (CNS). Ad5 replication-impaired vectors most often contain deletions in the E1 and E3 regions, with transgenes driven by a variety of promoters including viral promoters, and those that are neuron-specific (2). Recently fiber-modified and “gutless” Ad vectors, and those based on canine adenovirus serotype 2, have been developed for use in brain (3–9).

Affiliation(s): (2) Marshfield Clinic Research Foundation, Marshfield, WI
(3) Program in Gene Therapy, Departments of Internal Medicine, Neurology, Physiology & Biophysics, University of Iowa College of Medicine, Iowa City, IA

Book Title: Gene Delivery to Mammalian Cells: Volume 2: Viral Gene Transfer Techniques

Series: Methods in Molecular Biology | Volume: 246 | Pub. Date: Oct-28-2003 | Page Range: 91-120 | DOI: 10.1385/1-59259-650-9:91

Subject: Genetics/Genomics

References

Download References

1.	Horwitz, M. S. (1990) Adenoviridae and their replication, in Virology, 2nd ed., (Fields, B. N., and Knipe, D. M., eds.), Raven Press, Ltd, New York, NY, pp. 1679–1721.

2.	Millecamps, S., Kiefer, H., Navarro, V., Geoffroy, M. C., Robert, J. J., Finiels, F., et al. (1999) Neuron-restrictive silencer elements mediate neuron specificity of adenoviral gene expression. Nat. Biotechnol. 17, 865–869.

3.	Chillon, M., Bosch, A., Zabner, J., Law, L., Armentano, D., Welsh, M. J., and Davidson, B. L. (1999) Group D adenoviruses infect primary central nervous system cells more efficiently than those from Group C. J. Virol. 73, 2537–2540.

4.	Xia, H., Anderson, B., Mao, Q., and Davidson, B. L. (2000) Recombinant human adenovirus: targeting to the human transferrin receptor improves gene transfer to brain microcapillary endothelium. J. Virol. 74, 11359–11366.

5.	Davidson, B. L. and Bohn, M. C. (1997) Recombinant adenovirus: a gene transfer vector for study and treatment of CNS diseases. Exp. Neurol. 144, 125–130.

6.	Heistad, D. D. and Faraci, F. M. (1996) Gene therapy for cerebral vascular disease. Stroke 27, 1688–1693.

7.	Soudais, C., Laplace-Builhe, C., Kissa, K., and Kremer, E. J. (2001) Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo. FASEB J. 15, 2283–2285.

8.	Chillon, M. and Kremer, E. J. (2001) Trafficking and propagation of canine aden-ovirus vectors lacking a known integrin-interacting motif. Hum. Gene Ther. 12, 1815–1823.

9.	Umana, P., Gerdes, C. A., Stone, D., Davis, J. R., Ward, D., Castro, M. G., and Lowenstein, P. R. (2001) Efficient FLPe recombinase enables scalable production helper-dependent adenoviral vectors with negligible helper-virus contamination. Nat. Biotechnol. 19, 582–585.

10.	Byrnes, A. P., Rusby, J. E., Wood, M. J. A., and Charlton, H. M. (1995) Adenovirus gene transfer causes inflammation in the brain. Neuroscience 66, 1015–1024.

11.	Kajiwara, K., Byrnes, A. P., Charlton, H. M., Wood, M. J., and Wood, K. J. (1997) Immune responses to adenoviral vectors during gene transfer in the brain. Hum. Gene Ther. 8, 253–265.

12.	Liu, Y., Himes, B. T., Moul, J., Huang, W., Chow, S. Y., Tessler, A., and Fischer, I. (1997) Application of recombinant adenovirus for in vivo gene delivery to spinal cord. Brain Res. 768, 19–29.

13.	Davidson, B. L., Allen, E. D., Kozarsky, K. F., Wilson, J. M. and Roessler, B. J. (1993) A model system for in vivo gene transfer into the central nervous system using an adenoviral vector. Nat. Genet. 3, 219–223.

14.	Anderson, R. D., Haskell, R. E., Xia, H., Roessler, B. J., and Davidson, B. L. (2000) A simple method for the rapid generation of recombinant adenovirus vectors. Gene Ther. 7, 1034–1038.

15.	Aoki, K., Barker, C., Danthinne, X., Imperiale, M. J., and Nabel, G. J. (1999) Efficient generation of recombinant adenoviral vectors by Cre-lox recombination in vitro. Mol. Med. 5, 224–231.

16.	Alisky, J. M. and Davidson, B. L. (2000) Gene therapy for amyotrophic lateral sclerosis and other motor neuron diseases. Hum. Gene Ther. 11, 2315–2329.

17.	Ridoux, V., Robert, J. J., Zhang, X., Perricaudet, M., Mallet, J., and La Salle, G. L. G. (1994) Adenoviral vectors as functional retrograde neuronal tracers. Brain Res. 648, 171–175.

18.	Kuo, H., Ingram, D. K., Crystal, R. G., and Mastrangeli, A. (1995) Retrograde transfer of replication deficient recombinant adenovirus vector in the central nervous system for tracing studies. Brain Res. 705, 31–38.

19.	Ghadge, G. D., Roos, R. P., Kang, U. J., Wollmann, R., Fishman, P. S., Kalynych, A. M., et al. (1995) CNS gene delivery by retrograde transport of recombinant replication-defective adenoviruses. Gene Ther. 2, 132–137.

20.	Millecamps, S., Nicholle, D., Ceballos-Picot, I., Mallet, J. and Barkats, M. (2001) Synaptic sprouting increases the uptake capacities of motoneurons in amyotrophic lateral sclerosis mice. Proc. Natl. Acad. Sci. USA 98, 7582–7587.

21.	Terashima, T., Miwa, A., Kanegae, Y., Saito, I., and Okado, H. (1997) Retrograde and anterograde labeling of cerebellar afferent projection by the injection of recombinant adenoviral vectors into the mouse cerebellar cortex. Anat. Embryol. (Berl) 196, 363–382.

22.	Draghia, R., Caillaud, C., Manicom, R., Pavirani, A., Kohn, A., and Poenaru, L. (1995) Gene delivery into the central nervous system by nasal instillation in rats. Gene Ther. 2, 418–423.

23.	Sandmair, A. M., Loimas, S., Puranen, P., Immonen, A., Kossila, M., Puranen, M., et al. (2000) Thymidine kinase gene therapy for human malignant glioma, using replication-deficient retroviruses or adenoviruses. Hum. Gene Ther. 11, 2197–2205.

24.	Eck, S. L., Alavi, J. B., Judy, K., Phillips, P., Alavi, A., Hackney, D., et al. (2001) Treatment of recurrent or progressive malignant glioma with a recombinant adenovirus expressing human interferon-beta (H5.010CMVhIFN-b): a phase I trial. Hum. Gene Ther. 12, 97–113.

25.	Thomas, C. E., Birkett, D., Anozie, I., Castro, M. G., and Lowenstein, P. R. (2001) Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain. Mol. Ther. 3, 36–46.

26.	Choi-Lundberg, D. L., Lin, Q., Chang, Y.-N., Chiang, Y. L., Hay, C. M., Mohajeri, H., et al. (1997) Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science 275, 838–841.

27.	Ghodsi, A., Stein, C., Derksen, T., Yang, G., Anderson, R. D., and Davidson, B.L. (1998) Extensive μ-glucuronidase activity in murine CNS after adenovirus mediated gene transfer to brain. Hum. Gene Ther. 9, 2331–2340.

28.	Davidson, B. L., Stein, C. S., Heth, J. A., Martins, I., Kotin, R. M., Derksen, T.A., et al. (2000) Recombinant A AV type 2, 4 and 5 vectors: transduction of variant cell types and regions in the mammalian CNS. Proc. Natl. Acad. Sci. USA 97, 3428–3432.

29.	Brooks, A. I., Stein, C. S., Hughes, S. M., Heth, J., McCray, P. B. Jr., Sauter, S.L., et al. (2002) Functional correction of established CNS deficits in an animal model of lysosomal storage disease using feline immunodeficiency virus-based vectors. Proc. Natl. Acad. Sci. USA 99, 6216–6221.

30.	DiPasquale, G., Davidson, B.L., Stein, C.S., Martins, I., Scudiero, D., Monks, A., et al. (2003). Identification of PDGFR as a receptor for AAV5 transduction. Nat. Med. In press.

31.	Franklin, K. B. J. and Paxinos, G. (1997) The Mouse Brain in Stereotaxic Coordinates, Academic Press, San Diego, CA, pp. 1–190.

32.	Paxinos, G. and Watson, C. (1986) The Rat brain in Stereotaxic Coordinates, Academic Press Inc., New York, NY.

33.	Christenson, S. D., Lake, K. D., Ooboshi, H., Faraci, F. M., Davidson, B. L., and Heistad, D. D. (1998) Adenovirus-mediated gene transfer in vivo to cerebral blood vessels and perivascular tissue in mice. Stroke 29, 1411–1415.

34.	Finegold, A. A., Mannes, A. J., and Iadarola, M. J. (1999) A paracrine paradigm for in vivo gene therapy in the central nervous system: treatment of chronic pain. Hum. Gene Ther. 10, 1251–1257.

35.	Ooboshi, H., Welsh, M. J., Rios, C. D., Davidson, B. L., and Heistad, D. D. (1995) Adenovirus-mediated gene transfer in vivo to cerebral blood vessels and perivas-cular tissue. Circ. Res. 77, 7–13.

36.	Nilaver, G., Muldoon, L. L., Kroll, R. A., Pagel, M. A., Breakefield, X. O., Davidson, B. L., and Neuwelt, E. A. (1995) Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood-brain barrier disruption. Proc. Natl. Acad. Sci. USA 92, 9829–9833.

37.	Colak, A., Goodman, J. C., Chen, S. H., Woo, S. L. C., Grossman, R. G., and Shine, H. D. (1995) Adenovirus-mediated gene therapy for experimental spinal cord tumors: tumoricidal efficacy and functional outcome. Brain Res. 691, 76–82.

38.	Ross, B. D., Kim, B., and Davidson, B. L. (1995) Assessment of ganciclovir toxicity to experimental intracranial gliomas following recombinant adenoviral-mediated transfer of the herpes simplex virus thymidine kinase gene by magnetic resonance imaging and proton magnetic resonance spectroscopy. Clin. Cancer Res. 1, 651–657.

39.	Boulis, N. M., Bhatia, V., Brindle, T. I., Holman, H. T., Krauss, D. J., Blaivas, M., and Hoff, J.T. (1999) Adenoviral nerve growth factor and beta-galactosidase transfer to spinal cord: a behavioral and histological analysis. J. Neurosurg. 90(Suppl 1), 99–108.

40.	Mannes, A. J., Caudle, R. M., O’Connell, B. C., and Iadarola, M. J. (1998) Adenoviral gene transfer to spinal cord neurons: intrathecal vs. intraparenchymal administration. Brain Res. 793, 1–6.

41.	Alisky, J. M. and Tolbert, D. L. (1997) Quantitative analysis of converging spinal and cuneate mossy fiber afferent projections to the rat cerebellar anterior lobe. Neuroscience 80, 373–388.

42.	Finiels, F., Gimenez, Y., Ribotta, M., Barkats, M., Samolyk, M., Robert, J., et al. (1995) Specific and efficient gene transfer offers new potentialities for the treatment of motor neurone diseases. NeuroReport 6, 2473–2478.

43.	Haase, G., Kennel, P., Pettmann, B., Vigne, E., Akli, S., Revah, F., et al. (1997) Gene therapy of murine motor neuron disease using adenoviral vectors for neurotrophic factors. Nat. Med. 3, 429–436.

44.	Warita, H., Abe, K., Setoguchi, Y. and Itoyama, Y. (1998) Expression of adenovirus-mediated E. coli lacZ gene in skeletal muscles and spinal motor neurons of transgenic mice with a mutant superoxide dismutase gene. Neurosci. Lett. 246, 153–156.

45.	Geddes, B. J., Harding, T. C., Hughes, D. S., Byrnes, A. P., Lightman, S. L., Conde, G. and Uney, J. B. (1996) Persistent transgene expression in the hypothalamus following stereotaxic delivery of a recombinant adenovirus: suppresion of the immune response with cyclosporin. Endocrinology 137, 5166–5169.

46.	Petrof, B. J., Acsadi, G., Jani, A., Massie, B., Bourdon, J., Matusiewicz, N., et al. (1995) Efficiency and functional consequences of adenovirus-mediated in vivo gene transfer to normal and dystrophic (mdx) mouse diaphragm. Am. J. Respir. Cell Mol. Biol. 13, 508–517.

47.	Ooboshi, H., Rios, C. D., and Heistad, D. D. (1997) Novel methods for adenovirus-mediated gene transfer to blood vessels in vivo. Mol. Cell Biochem. 172, 37–46.

48.	Brooks, A. I., Halterman, M. W., Chadwick, C. A., Davidson, B. L., Haak-Fredenscho, M., Radel, C., et al. (1997) Reproducible and efficient murine CNS gene delivery using a microprocessor-controlled injector. J. Neurosci. Methods 80, 137–147.

49.	DiPolo, A., Lerner, L. E., and Farber, D. B. (1997) Transcriptional activation of the human rod cGMP-phosphodiesterase beta-subunit gene is mediated upstream AP-1 element. Nucleic Acids Res. 25, 3863–3867.

50.	DeMatteo, R. P., Chu, G., Ahn, M., Chang, E., Barker, C. F., and Markmann, J. F. (1997) Long-lasting adenovirus transgene expression in mice through neonatal intrathymic tolerance induction without the use of immunosuppression. J. Virol. 71, 5330–5335.

51.	Ghodsi, A., Stein, C., Derksen, T., Martins, I., Anderson, R. D., and Davidson, B. L. (1999) Systemic hyperosmolality improves b-glucuronidase distribution and pathology in murine MPS VII brain following intraventricular gene transfer. Exp. Neurol. 160, 109–116.

52.	Vasquez, E. C., Beltz, T. G., Haskell, R. E., Johnson, R. F., Meyrelles, S. S., Davidson, B. L., and Johnson, A. K. (2001) Adenovirus-mediated gene delivery to cells of the magnocellular hypothalamo-neurohypophyseal system. Exp. Neurol. 167, 260–271.

53.	Masumura, M., Hata, R., Nishimura, I., Uetsuki, T., Sawada, T., and Yoshikawa, K. (2000) Caspase-3 activation and inflammatory responses in rat hippocampus inoculated with a recombinant adenovirus expressing the Alzheimer amyloid precursor protein. Mol. Brain Res. 80, 219–227.

54.	Nishimura, I., Uetsuki, T., Dani, S. U., Ohsawa, Y., Saito, I., Okamura, H., et al. (1998) Degeneration in vivo of rat hippocampal neurons by wild-type Alzheimer amyloid precursor protein overexpressed by adenovirus-mediated gene transfer. J. Neurosci. 18, 2387–2398.

55.	Abe, K., Setoguchi, Y., Hayashi, T., and Itoyama, Y. (1997) In vivo adenovirus-mediated gene transfer and the expression in ischemic and reperfused rat brain. Brain Res. 763, 191–201.

56.	Kitagawa, H., Sasaki, C., Sakai, K., Mori, A., Mitsumoto, Y., Mori, T., et al (1999) Adenovirus-mediated gene transfer of glial cell line-derived neurotrophic factor prevents ischemic brain injury after transient middle cerebral artery occlusion in rats. J. Cereb. Blood Flow Metab. 19, 1336–1344.

57.	Vasquez, E. C., Beltz, T. G., Meyrelles, S. S., and Johnson, A. K. (1999) Aden-ovirus-mediated gene delivery to hypothalamic magnocellular neurons in mice. Hypertension 34, 756–761.

Comments (Loading...)

Post comment/View all comments