Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
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Pardridge, W.M. Drug and gene targeting to the brain with molecular Trojan horses. Nat. Rev. Drug Discov. 1, 131–139 (2002).
Kaspar, B.K., Llado, J., Sherkat, N., Rothstein, J.D. & Gage, F.H. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 301, 839–842 (2003).
Azzouz, M. et al. Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. J. Clin. Invest. 114, 1726–1731 (2004).
Azzouz, M. et al. VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature 429, 413–417 (2004).
Ralph, G.S. et al. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat. Med. 11, 429–433 (2005).
Kaplitt, M.G. et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369, 2097–2105 (2007).
Marks, W.J. Jr. et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 7, 400–408 (2008).
Worgall, S. et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum. Gene Ther. 19, 463–474 (2008).
Blankinship, M.J. et al. Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol. Ther. 10, 671–678 (2004).
Wang, Z. et al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat. Biotechnol. 23, 321–328 (2005).
Inagaki, K. et al. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol. Ther. 14, 45–53 (2006).
Nakai, H. et al. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J. Virol. 79, 214–224 (2005).
Pacak, C.A. et al. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circ. Res. 99, e3–e9 (2006).
Towne, C., Raoul, C., Schneider, B.L. & Aebischer, P. Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice. Mol. Ther. 16, 1018–1025 (2008).
Foust, K.D., Poirier, A., Pacak, C.A., Mandel, R.J. & Flotte, T.R. Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons. Hum. Gene Ther. 19, 61–70 (2008).
Gao, G. et al. Clades of Adeno-associated viruses are widely disseminated in human tissues. J. Virol. 78, 6381–6388 (2004).
Cearley, C.N. & Wolfe, J.H. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol. Ther. 13, 528–537 (2006).
Akache, B. et al. The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9. J. Virol. 80, 9831–9836 (2006).
McCarty, D.M. et al. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 10, 2112–2118 (2003).
Klein, R.L., Dayton, R.D., Tatom, J.B., Henderson, K.M. & Henning, P.P. AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method. Mol. Ther. 16, 89–96 (2008).
Cearley, C.N. et al. Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol. Ther. 16, 1710–1718 (2008).
Kaspar, B.K. et al. Targeted retrograde gene delivery for neuronal protection. Mol. Ther. 5, 50–56 (2002).
Miller, T.M. et al. Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis. Proc. Natl. Acad. Sci. USA 103, 19546–19551 (2006).
Hollis, E.R., II, Kadoya, K., Hirsch, M., Samulski, R.J. & Tuszynski, M.H. Efficient retrograde neuronal transduction utilizing self-complementary AAV1. Mol. Ther. 16, 296–301 (2008).
Abbott, N.J., Ronnback, L. & Hansson, E. Astrocyte-endothelial interactions at the blood-brain barrier. Nat. Rev. Neurosci. 7, 41–53 (2006).
Abbott, N.J. Dynamics of CNS barriers: evolution, differentiation, and modulation. Cell. Mol. Neurobiol. 25, 5–23 (2005).
Baughan, T. et al. Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol. Ther. 14, 54–62 (2006).
Siegel, R.M. & Callaway, E.M. Francis Crick's legacy for neuroscience: between the alpha and the Omega. PLoS Biol. 2, e419 (2004).
Yamanaka, K. et al. Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat. Neurosci. 11, 251–253 (2008).
Dodge, J.C. et al. Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity. Mol. Ther. 16, 1056–1064 (2008).
Harper, S.Q. et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc. Natl. Acad. Sci. USA 102, 5820–5825 (2005).
Rodriguez-Lebron, E., Denovan-Wright, E.M., Nash, K., Lewin, A.S. & Mandel, R.J. Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol. Ther. 12, 618–633 (2005).
Maheshri, N., Koerber, J.T., Kaspar, B.K. & Schaffer, D.V. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat. Biotechnol. 24, 198–204 (2006).
Li, W. et al. Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol. Ther. 16, 1252–1260 (2008).
Koerber, J.T., Jang, J.H. & Schaffer, D.V. DNA shuffling of adeno-associated virus yields functionally diverse viral progeny. Mol. Ther. 16, 1703–1709 (2008).
Haidet, A.M. et al. Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proc. Natl. Acad. Sci. USA 105, 4318–4322 (2008).