- About this Journal ·
- Abstracting and Indexing ·
- Advance Access ·
- Aims and Scope ·
- Annual Issues ·
- Article Processing Charges ·
- Articles in Press ·
- Author Guidelines ·
- Bibliographic Information ·
- Citations to this Journal ·
- Contact Information ·
- Editorial Board ·
- Editorial Workflow ·
- Free eTOC Alerts ·
- Publication Ethics ·
- Reviewers Acknowledgment ·
- Submit a Manuscript ·
- Subscription Information ·
- Table of Contents
BioMed Research International
Volume 2013 (2013), Article ID 974819, 8 pages
Systemic Delivery of Tyrosine-Mutant AAV Vectors Results in Robust Transduction of Neurons in Adult Mice
1Division of Neurology, Department of Medicine, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan
2Division of Neurosurgery, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke, Tochigi 329-0498, Japan
Received 6 February 2013; Revised 19 April 2013; Accepted 21 April 2013
Academic Editor: Akhtar Jamal Khan
Copyright © 2013 Asako Iida et al. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
- W. J. Bowers, X. O. Breakefield, and M. Sena-Esteves, “Genetic therapy for the nervous system,” Human Molecular Genetics, vol. 20, no. R1, pp. R28–R41, 2011.
- K. A. High and P. Aubourg, “rAAV human trial experience,” Methods in Molecular Biology, vol. 807, pp. 429–457, 2011.
- M. S. Weinberg, R. J. Samulski, and T. J. McCown, “Adeno-associated virus (AAV) gene therapy for neurological disease,” Neuropharmacology, vol. 69, pp. 82-–88, 2013.
- P. Hadaczek, J. L. Eberling, P. Pivirotto, J. Bringas, J. Forsayeth, and K. S. Bankiewicz, “Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC,” Molecular Therapy, vol. 18, no. 8, pp. 1458–1461, 2010.
- S. Muramatsu, K. Fujimoto, S. Kato et al., “A phase I study of aromatic L-amino acid decarboxylase gene therapy for parkinson's disease,” Molecular Therapy, vol. 18, no. 9, pp. 1731–1735, 2010.
- R. T. Bartus, T. L. Baumann, L. Brown, et al., “Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating, “clinical proof-of-concept” for AAV-neurturin (CERE-120) in Parkinson's disease,” Neurobiology of Aging, vol. 34, no. 1, pp. 35–61, 2012.
- C. W. Christine, P. A. Starr, P. S. Larson et al., “Safety and tolerability of putaminal AADC gene therapy for Parkinson disease,” Neurology, vol. 73, no. 20, pp. 1662–1669, 2009.
- P. A. LeWitt, A. R. Rezai, M. A. Leehey et al., “AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial,” The Lancet Neurology, vol. 10, no. 4, pp. 309–319, 2011.
- A. K. Bevan, S. Duque, K. D. Foust, et al., “Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders,” Molecular Therapy, vol. 19, no. 11, pp. 1971–1980, 2011.
- S. Duque, B. Joussemet, C. Riviere et al., “Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons,” Molecular Therapy, vol. 17, no. 7, pp. 1187–1196, 2009.
- T. Federici, J. S. Taub, G. R. Baum, et al., “Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs,” Gene Therapy, vol. 19, no. 8, pp. 852–859, 2012.
- K. D. Foust, E. Nurre, C. L. Montgomery, A. Hernandez, C. M. Chan, and B. K. Kaspar, “Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes,” Nature Biotechnology, vol. 27, no. 1, pp. 59–65, 2009.
- A. A. Rahim, A. M. Wong, K. Hoefer, et al., “Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system,” The FASEB Journal, vol. 25, no. 10, pp. 3505–3518, 2011.
- L. Samaranch, E. A. Salegio, W. San Sebastian, et al., “Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates,” Human Gene Therapy, vol. 23, no. 4, pp. 382–389, 2012.
- J. J. Glascock, E. Y. Osman, M. J. Wetz, et al., “Decreasing disease severity in symptomatic, Smn(−/−);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery,” Human Gene Therapy, vol. 23, no. 3, pp. 330–335, 2012.
- K. D. Foust, X. Wang, V. L. McGovern et al., “Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN,” Nature Biotechnology, vol. 28, no. 3, pp. 271–274, 2010.
- D. M. McCarty, “Self-complementary AAV vectors; advances and applications,” Molecular Therapy, vol. 16, no. 10, pp. 1648–1656, 2008.
- S. J. Gray, V. Matagne, L. Bachaboina, S. Yadav, S. R. Ojeda, and R. J. Samulski, “Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates,” Molecular Therapy, vol. 19, no. 6, pp. 1058–1069, 2011.
- A. Ciesielska, P. Hadaczek, G. Mittermeyer, et al., “Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses,” Molecular Therapy, vol. 21, no. 1, pp. 158–166, 2012.
- A. Delzor, N. Dufour, F. Petit, et al., “Restricted transgene expression in the brain with cell-type specific neuronal promoters,” Human Gene Therapy Methods, vol. 23, no. 4, pp. 242–254, 2012.
- C. A. Ku, V. A. Chiodo, S. L. Boye, et al., “Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis,” Human Molecular Genetics, vol. 20, no. 23, pp. 4569–4581, 2011.
- M. Li, G. R. Jayandharan, B. Li et al., “High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy,” Human Gene Therapy, vol. 21, no. 11, pp. 1527–1543, 2010.
- H. Petrs-Silva, A. Dinculescu, Q. Li et al., “Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina,” Molecular Therapy, vol. 19, no. 2, pp. 293–301, 2011.
- C. Qiao, W. Zhang, Z. Yuan et al., “Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle,” Human Gene Therapy, vol. 21, no. 10, pp. 1343–1348, 2010.
- B. Cheng, C. Ling, Y. Dai et al., “Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells,” Gene Therapy, vol. 19, no. 4, pp. 375–384, 2012.
- D. Dalkara, L. C. Byrne, T. Lee, et al., “Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9,” Gene Therapy, vol. 19, no. 2, pp. 176–181, 2012.
- Y. Zhang and D. Duan, “Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma,” Human Gene Therapy, vol. 23, no. 1, pp. 98–103, 2012.
- T. Dittgen, A. Nimmerjahn, S. Komai et al., “Lentivirus-based genetic manipulations of cortical neurons and their optical and electrophysiological monitoring in vivo,” Proceedings of the National Academy of Sciences of the United States of America, vol. 101, no. 52, pp. 18206–18211, 2004.
- O. Meyuhas and A. Klein, “The mouse ribosomal protein L7 gene. Its primary structure and functional analysis of the promoter region,” Journal of Biological Chemistry, vol. 265, no. 20, pp. 11465–11473, 1990.
- G. Gao, L. H. Vandenberghe, M. R. Alvira et al., “Clades of adeno-associated viruses are widely disseminated in human tissues,” Journal of Virology, vol. 78, no. 12, pp. 6381–6388, 2004.
- X. G. Li, T. Okada, M. Kodera et al., “Viral-mediated temporally controlled dopamine production in a rat model of Parkinson disease,” Molecular Therapy, vol. 13, no. 1, pp. 160–166, 2006.
- W. Wagner, S. McCroskery, and J. A. Hammer III, “An efficient method for the long-term and specific expression of exogenous cDNAs in cultured Purkinje neurons,” Journal of Neuroscience Methods, vol. 200, no. 2, pp. 95–105, 2011.
- H. Nakai, S. Fuess, T. A. Storm, S. I. Muramatsu, Y. Nara, and M. A. Kay, “Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice,” Journal of Virology, vol. 79, no. 1, pp. 214–224, 2005.
- Y. Wang, C. Ling, L. Song, et al., “Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation,” Human Gene Therapy Methods, vol. 23, no. 4, pp. 225–233, 2012.
- H. Zhang, B. Yang, X. Mu et al., “Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system,” Molecular Therapy, vol. 19, no. 8, pp. 1440–1448, 2011.
- T. Philips and W. Robberecht, “Neuroinflammation in amyotrophic lateral sclerosis: role of glial activation in motor neuron disease,” The Lancet Neurology, vol. 10, no. 3, pp. 253–263, 2011.
- P. M. Rappold and K. Tieu, “Astrocytes and therapeutics for Parkinson's disease,” Neurotherapeutics, vol. 7, no. 4, pp. 413–423, 2010.
- E. D. Horowitz, M. G. Finn, and A. Asokan, “Tyrosine cross-linking reveals interfacial dynamics in adeno-associated viral capsids during infection,” ACS Chemical Biology, vol. 7, no. 6, pp. 1059–1066, 2012.
- L. Zhong, B. Li, C. S. Mah et al., “Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses,” Proceedings of the National Academy of Sciences of the United States of America, vol. 105, no. 22, pp. 7827–7832, 2008.
- H. Petrs-Silva, A. Dinculescu, Q. Li et al., “High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors,” Molecular Therapy, vol. 17, no. 3, pp. 463–471, 2009.
- Y. Miyazaki, H. Adachi, M. Katsuno, et al., “Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2,” Nature Medicine, vol. 18, no. 7, pp. 1136–1141, 2012.
- D. M. Markusic, R. W. Herzog, G. V. Aslanidi et al., “High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines,” Molecular Therapy, vol. 18, no. 12, pp. 2048–2056, 2010.
- M. Naumer, F. Sonntag, K. Schmidt, et al., “Properties of the adeno-associated virus assembly activating protein,” Journal of Virology, vol. 86, no. 23, pp. 13038–13048, 2012.