Local and retrograde gene transfer into primate neuronal pathways via adeno-associated virus serotype 8 and 9

Neuroscience

Volumn 193, Issue , 2011, Pages 249-258

  Masamizu, Y ^a   Okada, T ^a   Kawasaki, K ^b   Ishibashi, H ^a   Yuasa, S ^a   Takeda, S ^a   Hasegawa, I ^b   Nakahara, K ^a  

^a NATIONAL INSTITUTE OF NEUROSCIENCE   (Japan)

^b NIIGATA UNIVERSITY   (Japan)

Author keywords

AAV8; AAV9; Common marmoset; Gene transfer; Macaque monkey; Neuronal pathway

Indexed keywords

ENHANCED GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS SEROTYPE 8; ADENO ASSOCIATED VIRUS SEROTYPE 9; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CALLITRICHINAE; CONTROLLED STUDY; DOPAMINERGIC NERVE CELL; FRONTAL CORTEX; IMMUNOHISTOCHEMISTRY; ION EXCHANGE CHROMATOGRAPHY; MALE; MEDICAL RESEARCH; NIGRONEOSTRIATAL SYSTEM; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN LOCALIZATION; STEREOTAXIS; SUBSTANTIA NIGRA; SUBSTANTIA NIGRA PARS COMPACTA; SUBSTANTIA NIGRA PARS RETICULATA; THALAMUS; TRANSGENE; TROPISM; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BRAIN; BRAIN MAPPING; CALLITHRIX; DEPENDOVIRUS; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; MALE; NERVE TISSUE PROTEINS; NEURAL PATHWAYS; NEURONS;

EID: 80052506951     PISSN: 03064522     EISSN: 18737544     Source Type: Journal    
DOI: 10.1016/j.neuroscience.2011.06.080     Document Type: Article

References (36)

1. Albin R.L., Young A.B., Penney J.B. The functional anatomy of basal ganglia disorders. Trends Neurosci 1989, 12:366-375.
2. Alexander G.E., Crutcher M.D. Functional architecture of basal ganglia circuits: neural substrates of parallel processing. Trends Neurosci 1990, 13:266-271.
3. Bostan A.C., Dum R.P., Strick P.L. The basal ganglia communicate with the cerebellum. Proc Natl Acad Sci U S A 2010, 107:8452-8456.
4. Broekman M.L., Comer L.A., Hyman B.T., Sena-Esteves M. Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain. Neuroscience 2006, 138:501-510.
5. Chan A.W., Chong K.Y., Martinovich C., Simerly C., Schatten G. Transgenic monkeys produced by retroviral gene transfer into mature oocytes. Science 2001, 291:309-312.
6. Dodiya H.B., Bjorklund T., Stansell J., Mandel R.J., Kirik D., Kordower J.H. Differential transduction following basal ganglia administration of distinct pseudotyped AAV capsid serotypes in nonhuman primates. Mol Ther 2010, 18:579-587.
7. Eslamboli A., Georgievska B., Ridley R.M., Baker H.F., Muzyczka N., Burger C., Mandel R.J., Annett L., Kirik D. Continuous low-level glial cell line-derived neurotrophic factor delivery using recombinant adeno-associated viral vectors provides neuroprotection and induces behavioral recovery in a primate model of Parkinson's disease. J Neurosci 2005, 25:769-777.
8. Felleman D.J., Van Essen D.C. Distributed hierarchical processing in the primate cerebral cortex. Cereb Cortex 1991, 1:1-47.
9. Foust K.D., Nurre E., Montgomery C.L., Hernandez A., Chan C.M., Kaspar B.K. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 2009, 27:59-65.
10. Foust K.D., Wang X., McGovern V.L., Braun L., Bevan A.K., Haidet A.M., Le T.T., Morales P.R., Rich MM Burghes A.H., Kaspar B.K. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 2010, 28:271-274.
11. Fritsches K.A., Rosa M.G. Visuotopic organisation of striate cortex in the marmoset monkey (Callithrix jacchus). J Comp Neurol 1996, 372:264-282.
12. Gao G., Vandenberghe L.H., Alvira M.R., Lu Y., Calcedo R., Zhou X., Wilson J.M. Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol 2004, 78:6381-6388.
13. Gao G.P., Alvira M.R., Wang L., Calcedo R., Johnston J., Wilson J.M. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A 2002, 99:11854-11859.
14. Han X., Qian X., Bernstein J.G., Zhou H.H., Franzesi G.T., Stern P., Bronson R.T., Graybiel A.M., Desimone R., Boyden E.S. Millisecond-timescale optical control of neural dynamics in the nonhuman primate brain. Neuron 2009, 62:191-198.
15. Hollis E.R., Kadoya K., Hirsch M., Samulski R.J., Tuszynski M.H. Efficient retrograde neuronal transduction utilizing self-complementary AAV1. Mol Ther 2008, 16:296-301.
16. Kaplitt M.G., Leone P., Samulski R.J., Xiao X., Pfaff D.W., O'Malley K.L., During M.J. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 1994, 8:148-154.
17. Kaspar B.K., Erickson D., Schaffer D., Hinh L., Gage F.H., Peterson D.A. Targeted retrograde gene delivery for neuronal protection. Mol Ther 2002, 5:50-56.
18. Masamizu Y., Okada T., Ishibashi H., Takeda S., Yuasa S., Nakahara K. Efficient gene transfer into neurons in monkey brain by adeno-associated virus 8. Neuroreport 2010, 21:447-451.
19. Matsushita T., Elliger S., Elliger C., Podsakoff G., Villarreal L., Kurtzman G.J., Iwaki Y., Colosi P. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther 1998, 5:938-945.
20. Minamimoto T., Hori Y., Kimura M. Roles of the thalamic CM-PF complex-Basal ganglia circuit in externally driven rebias of action. Brain Res Bull 2009, 78:75-79.
21. Murphy P.C., Sillito A.M. Functional morphology of the feedback pathway from area 17 of the cat visual cortex to the lateral geniculate nucleus. J Neurosci 1996, 16:1180-1192.
22. Nakahara K., Adachi Y., Osada T., Miyashita Y. Exploring the neural basis of cognition: multi-modal links between human fMRI and macaque neurophysiology. Trends Cogn Sci 2007, 11:84-92.
23. Nakahira E., Yuasa S. Neuronal generation, migration, and differentiation in the mouse hippocampal primoridium as revealed by enhanced green fluorescent protein gene transfer by means of in utero electroporation. J Comp Neurol 2005, 483:329-340.
24. Nishimura Y., Onoe H., Morichika Y., Perfiliev S., Tsukada H., Isa T. Time-dependent central compensatory mechanisms of finger dexterity after spinal cord injury. Science 2007, 318:1150-1155.
25. Okada T., Nomoto T., Yoshioka T., Nonaka-Sarukawa M., Ito T., Ogura T., Iwata-Okada M., Uchibori R., Shimazaki K., Mizukami H., Kume A., Ozawa K. Large-scale production of recombinant viruses by use of a large culture vessel with active gassing. Hum Gene Ther 2005, 16:1212-1218.
26. Okada T., Nonaka-Sarukawa M., Uchibori R., Kinoshita K., Hayashita-Kinoh H., Nitahara-Kasahara Y., Takeda S., Ozawa K. Scalable purification of adeno-associated virus serotype 1 (AAV1) and AAV8 vectors, using dual ion-exchange adsorptive membranes. Hum Gene Ther 2009, 20:1013-1021.
27. Okada T., Shimazaki K., Nomoto T., Matsushita T., Mizukami H., Urabe M., Hanazono Y., Kume A., Tobita K., Ozawa K., Kawai N. Adeno-associated viral vector-mediated gene therapy of ischemia-induced neuronal death. Methods Enzymol 2002, 346:378-393.
28. Passingham R. How good is the macaque monkey model of the human brain?. Curr Opin Neurobiol 2009, 19:6-11.
29. Sasaki E., Suemizu H., Shimada A., Hanazawa K., Oiwa R., Kamioka M., Tomioka I., Sotomaru Y., Hirakawa R., Eto T., Shiozawa S., Maeda T., Ito M., Ito R., Kito C., Yagihashi C., Kawai K., Miyoshi H., Tanioka Y., Tamaoki N., Habu S., Okano H., Nomura T. Generation of transgenic non-human primates with germline transmission. Nature 2009, 459:523-527.
30. Tan E.M., Yamaguchi Y., Horwitz G.D., Gosgnach S., Lein E.S., Goulding M., Albright T.D., Callaway E.M. Selective and quickly reversible inactivation of mammalian neurons in vivo using the Drosophila allatostatin receptor. Neuron 2006, 51:157-170.
31. Taymans J.M., Vandenberghe L.H., Haute C.V., Thiry I., Deroose C.M., Mortelmans L., Wilson J.M., Debyser Z., Baekelandt V. Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain. Hum Gene Ther 2007, 18:195-206.
32. Towne C., Schneider B.L., Kieran D., Redmond D.E., Aebischer P. Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6. Gene Ther 2010, 17:141-146.
33. White A.J., Wilder H.D., Goodchild A.K., Sefton A.J., Martin P.R. Segregation of receptive field properties in the lateral geniculate nucleus of a New-World monkey, the marmoset Callithrix jacchus. J Neurophysiol 1998, 80:2063-2076.
34. Yasuda T., Miyachi S., Kitagawa R., Wada K., Nihira T., Ren Y.R., Hirai Y., Ageyama N., Terao K., Shimada T., Takada M., Mizuno Y., Mochizuki H. Neuronal specificity of alpha-synuclein toxicity and effect of Parkin co-expression in primates. Neuroscience 2007, 144:743-753.
35. Yuasa S., Nakamura K., Kohsaka S. Stereotaxic atlas of the marmoset brain with immunohistochemical architecture and MRI Images 2010, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
36. Zhang F., Wang L.P., Brauner M., Liewald J.F., Kay K., Watzke N., Wood P.G., Bamberg E., Nagel G., Gottschalk A., Deisseroth K. Multimodal fast optical interrogation of neural circuitry. Nature 2007, 446:633-639.