B. Akache, D. Grimm, K. Pandey, S. R. Yant, H. Xu et al., The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9, J. Virol, vol.80, pp.9831-9836, 2006.

R. T. Bartus, T. L. Baumann, L. Brown, B. R. Kruegel, J. M. Ostrove et al., Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating "clinical proof-ofconcept" for AAV-neurturin (CERE-120) in Parkinson's disease, Neurobiol. Ag, vol.34, pp.35-61, 2013.

C. L. Bell, L. H. Vandenberghe, P. Bell, M. P. Limberis, G. P. Gao et al., The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice, J. Clin. Invest, vol.121, pp.2427-2435, 2011.

A. K. Bevan, S. Duque, K. D. Foust, P. R. Morales, L. Braun et al., Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders, Mol. Ther, vol.19, 1971.

A. K. Bevan, K. R. Hutchinson, K. D. Foust, L. Braun, V. L. Mcgovern et al., Early heart failure in the SMN 7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery, Hum. Mol. Genet, vol.19, pp.3895-3905, 2010.

S. Boutin, V. Monteilhet, P. Veron, C. Leborgne, O. Benveniste et al., Prevalence of serum IgG and neutralizing factors against adenoassociated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors, Hum. Gene Ther, vol.21, pp.704-712, 2010.

R. Calcedo, H. Morizono, L. Wang, R. Mccarter, J. He et al., , 2011.

, Adeno-associated virus antibody profiles in newborns, children, and adolescents, Clin. Vaccine Immunol, vol.18, pp.1586-1588

Y. H. Chen, M. Chang, and B. L. Davidson, Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy, Nat. Med, vol.15, pp.1215-1218, 2009.

A. M. Colangelo, L. Alberghinaa, M. Papa, and C. , Astrogliosis as a therapeutic target for neurodegenerative diseases, Neurosci. Lett, vol.565, pp.59-64, 2014.

D. Dalkara, L. C. Byrne, R. R. Klimczak, M. Visel, L. Yin et al., In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous, Sci. Transl. Med, vol.5, pp.189-76, 2013.

D. Dalkara, L. C. Byrne, T. Lee, N. V. Hoffmann, D. V. Schaffer et al., Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9, Gene Ther, vol.19, pp.176-181, 2011.

B. L. Davidson, C. S. Stein, J. A. Heth, I. Martins, R. M. Kotin et al., 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, vol.97, pp.3428-3432, 2000.

R. D. Dayton, D. B. Wang, and R. L. Klein, The advent of AAV9 expands applications for brain and spinal cord gene delivery, Expert Opin. Biol. Ther, vol.12, pp.757-766, 2012.

M. Decressac, B. Mattsson, M. Lundblad, P. Weikop, and A. Björklund, Progressive neurodegenerative and behavioural changes induced by AAV-mediated overexpression of ?-synuclein in midbrain dopamine neurons, Neurobiol. Dis, vol.45, pp.939-953, 2012.

B. Dehay, D. Dalkara, S. Dovero, Q. Li, and E. Bezard, Systemic scAAV9 variant mediates brain transduction in newborn rhesus macaques, Sci. Rep, vol.2, p.253, 2012.
URL : https://hal.archives-ouvertes.fr/hal-01053467

S. Duque, B. Joussemet, C. Riviere, T. Marais, L. Dubreil et al., Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons, Mol. Ther, vol.17, pp.1187-1196, 2009.

, Frontiers in Molecular Neuroscience www.frontiersin.org

M. Engeln, S. Fasano, S. H. Ahmed, M. Cador, V. Baekelandt et al., Levodopa gains psychostimulant-like properties after nigral dopaminergic loss, Ann. Neurol, vol.74, pp.140-144, 2013.
URL : https://hal.archives-ouvertes.fr/hal-01153692

T. Federici, J. S. Taub, G. R. Baum, S. J. Gray, J. C. Grieger et al., Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs, Gene Ther, vol.19, pp.852-859, 2012.

F. K. Ferrari, T. Samulski, T. Shenk, and R. J. Samulski, Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adenoassociated virus vectors, J. Virol, vol.70, pp.3227-3234, 1996.

F. K. Ferrari, X. Xiao, D. Mccarty, and R. J. Samulski, New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors, Nat. Med, vol.3, pp.1295-1297, 1997.

K. D. Foust, E. Nurre, C. L. Montgomery, A. Hernandez, C. M. Chan et al., Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes, Nat. Biotechnol, vol.27, pp.59-65, 2008.

K. D. Foust, X. Wang, V. L. Mcgovern, L. Braun, A. K. Bevan et al., Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN, Nat. Biotechnol, vol.28, pp.271-274, 2010.

H. Fu, J. Dirosario, S. Killedar, K. Zaraspe, and D. M. Mccarty, Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery, Mol. Ther, vol.19, pp.1025-1033, 2011.

G. Gao, L. H. Vandenberghe, M. R. Alvira, Y. Lu, R. Calcedo et al., Clades of adeno-associated viruses are widely disseminated in human tissues, J. Virol, vol.78, pp.6381-6388, 2004.

S. J. Gray, B. L. Blake, H. E. Criswell, S. C. Nicolson, R. J. Samulski et al., Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB), Mol. Ther, vol.18, pp.570-578, 2010.

S. J. Gray, S. B. Foti, J. W. Schwartz, L. Bachaboina, B. Taylor-blake et al., optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors, Hum. Gene Ther, vol.22, pp.1143-1153, 2011.

S. J. Gray, V. Matagne, L. Bachaboina, S. Yadav, S. R. Ojeda et al., Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates, Mol. Ther, vol.19, pp.1058-1069, 2011.

S. J. Gray and R. Samulski, Vector design and considerations for CNS applications, Gene Vector Design and Application to Treat Nervous System Disorders, pp.1-9, 2011.

D. Grimm, A. Kern, K. Rittner, and J. A. Kleinschmidt, Novel tools for production and purification of recombinant adenoassociated virus vectors, Hum. Gene Ther, vol.9, pp.2745-2760, 1998.

D. Grimm, J. S. Lee, L. Wang, T. Desai, B. Akache et al., In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses, J. Virol, vol.82, pp.5887-5911, 2008.

S. Hermening, S. Kügler, M. Bähr, and S. Isenmann, Improved highcapacity adenoviral vectors for high-level neuron-restricted gene transfer to the CNS, J. Virol. Methods, vol.136, pp.30-37, 2006.

P. L. Hermonat and N. Muzyczka, Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells, Proc. Natl. Acad. Sci. U.S.A, vol.81, pp.6466-6470, 1984.

A. Iida, N. Takino, H. Miyauchi, K. Shimazaki, and S. Muramatsu, Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice, BioMed Res. Intl, vol.2013, pp.1-8, 2013.

N. Iwata, K. Kobayashi, T. Sasaoka, H. Hidaka, and T. Nagatsu, Structure of the mouse tyrosine hydroxylase gene, Biochem. Biophys. Res. Commun, vol.182, pp.348-354, 1992.

N. Iwata, M. Sekiguchi, Y. Hattori, A. Takahashi, M. Asai et al., Global brain delivery of neprilysin gene by intravascular administration of AAV vector in mice, Sci. Rep, vol.3, p.1472, 2013.

M. G. Kaplitt, A. Feigin, C. Tang, H. L. Fitzsimons, P. Mattis et al., Safety and tolerability of gene therapy with an adenoassociated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial, Lancet, vol.369, pp.60982-60991, 2007.

B. K. Kaspar, Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model, Science, vol.301, pp.839-842, 2003.

R. L. Klein, R. D. Dayton, J. B. Tatom, C. G. Diaczynsky, S. et al., Tau expression levels from various adeno-associated virus vector serotypes produce graded neurodegenerative disease states, Eur. J. Neurosci, vol.27, pp.1615-1625, 2008.

J. Korecka, M. Schouten, R. Eggers, A. Ulusoy, K. Bossers et al., Comparison of AAV serotypes for gene delivery to dopaminergic neurons in the Substantia Nigra, pp.1-21, 2011.

P. R. Lowenstein, Crossing the rubicon, Nat. Biotechnol, vol.27, pp.42-44, 2009.

N. Maheshri, J. T. Koerber, B. K. Kaspar, and D. V. Schaffer, Directed evolution of adeno-associated virus yields enhanced gene delivery vectors, Nat. Biotechnol, vol.24, pp.198-204, 2006.

W. J. Marks, R. T. Bartus, J. Siffert, C. S. Davis, A. Lozano et al., Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial, Lancet Neurol, vol.9, issue.10, pp.70254-70258, 2010.

C. N. Mattar, S. N. Waddington, A. Biswas, N. Johana, X. W. Ng et al., Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems, Gene Ther, vol.20, pp.69-83, 2012.

D. M. Mccarty, J. Dirosario, K. Gulaid, J. Muenzer, and H. Fu, , 2009.

, Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice, Gene Ther, vol.16, pp.1340-1352

D. M. Mccarty, H. Fu, P. E. Monahan, C. E. Toulson, P. Naik et al., Adeno-associated virus terminal repeat (TR) mutant generates selfcomplementary vectors to overcome the rate-limiting step to transduction in vivo, Gene Ther, vol.10, pp.2112-2118, 2003.

S. K. Mclaughlin, P. Collis, P. L. Hermonat, and N. Muzyczka, Adenoassociated virus general transduction vectors: analysis of proviral structures, J. Virol, vol.62, pp.1963-1973, 1988.

T. M. Miller, B. K. Kaspar, G. J. Kops, K. Yamanaka, L. J. Christian et al., Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis, Ann. Neurol, vol.57, pp.773-776, 2005.

F. Mingozzi, X. M. Anguela, G. Pavani, Y. Chen, R. J. Davidson et al., Overcoming preexisting humoral immunity to AAV using capsid decoys, Sci. Transl. Med, vol.5, pp.194-92, 2013.

N. Miyake, K. Miyake, M. Yamamoto, Y. Hirai, and T. Shimada, Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors, Brain Res, vol.1389, pp.19-26, 2011.

O. J. Müller, F. Kaul, M. D. Weitzman, R. Pasqualini, W. Arap et al., Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors, Nat. Biotechnol, vol.21, pp.1040-1046, 2003.

M. Nagai, D. B. Re, T. Nagata, A. Chalazonitis, T. M. Jessell et al., Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons, Nat. Neurosci, vol.10, pp.615-622, 2007.

D. S. Ojala, D. P. Amara, and D. V. Schaffer, Adeno-associated virus vectors and neurological gene therapy, Neuroscientist, 2014.

W. M. Pardridge, Drug and gene targeting to the brain with molecular Trojan horses, Nat. Rev. Drug Discov, vol.1, pp.131-139, 2002.

H. Petrs-silva, A. Dinculescu, Q. Li, S. H. Min, V. Chiodo et al., High-efficiency Transduction of the Mouse Retina by tyrosine-mutant AAV serotype vectors, Mol. Ther, vol.17, pp.463-471, 2008.

G. Porras, B. Dehay, and E. Bezard, Viral vectors in primate research: examples from Parkinson's disease research, Viral Vector Approaches in Neurobiology and Brain Diseases, pp.331-341, 2014.

, Frontiers in Molecular Neuroscience www.frontiersin.org

N. Pulicherla, S. Shen, S. Yadav, K. Debbink, L. Govindasamy et al., Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer, Mol. Ther, vol.19, pp.1070-1078, 2011.

J. E. Rabinowitz, F. Rolling, C. Li, H. Conrath, W. Xiao et al., Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity, J. Virol, vol.76, pp.791-801, 2002.

J. E. Rabinowitz, W. Xiao, and R. J. Samulski, Insertional mutagenesis of AAV2 capsid and the production of recombinant virus, Virology, vol.265, pp.274-285, 1999.

A. A. Rahim, A. M. Wong, K. Hoefer, S. M. Buckley, C. N. Mattar 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, FASEB J, vol.25, pp.3505-3518, 2011.

L. Samaranch, E. A. Salegio, W. San-sebastian, A. P. Kells, K. D. Foust et al., Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates, Hum. Gene Ther, vol.23, pp.382-389, 2012.

R. J. Samulski, K. I. Berns, M. Tan, and N. Muzyczka, Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells, Proc. Natl. Acad. Sci. U.S.A, vol.79, pp.2077-2081, 1982.

N. R. Saunders, C. Joakim-ek, and K. M. Dziegielewska, The neonatal blood-brain barrier is functionally effective, and immaturity does not explain differential targeting of AAV9, Nat. Biotechnol, vol.27, pp.804-805, 2009.

S. Shen, K. D. Bryant, S. M. Brown, S. H. Randell, A. et al., Terminal N-linked galactose is the primary receptor for adeno-associated virus 9, J. Biol. Chem, vol.286, pp.13532-13540, 2011.

E. Sidransky, M. A. Nalls, J. O. Aasly, J. Aharon-peretz, G. Annesi et al., Multicenter analysis of glucocerebrosidase mutations in Parkinson's disease, N. Engl. J. Med, vol.361, pp.1651-1661, 2009.

C. Spampanato, E. De-leonibus, P. Dama, A. Gargiulo, A. Fraldi et al., Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder, Mol. Ther, vol.19, pp.860-869, 2011.

A. F. Tarantal and C. C. Lee, Long-term luciferase expression monitored by bioluminescence imaging after adeno-associated virus-mediated fetal gene delivery in rhesus monkeys (Macaca mulatta). Hum, Gene Ther, vol.21, pp.143-148, 2010.

J. Taymans, L. H. Vandenberghe, C. V. Haute, I. Thiry, C. M. Deroose et al., Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain, Hum. Gene Ther, vol.18, pp.195-206, 2007.

C. Towne, M. Pertin, A. T. Beggah, P. Aebischer, and I. Decosterd, Recombinant adeno-associated virus serotype 6 (rAAV2/6)-mediated gene transfer to nociceptive neurons through different routes of delivery, Mol. Pain, vol.5, p.52, 2009.

L. H. Vandenberghe, J. M. Wilson, and G. Gao, Tailoring the AAV vector capsid for gene therapy, Gene Ther, vol.16, pp.311-319, 2008.

D. B. Wang, R. D. Dayton, P. P. Henning, C. D. Cain, L. R. Zhao et al., Expansive gene transfer in the rat CNS rapidly produces amyotrophic lateral sclerosis relevant sequelae when TDP-43 is overexpressed, Mol. Ther, vol.18, pp.2064-2074, 2010.

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.

D. A. Wolf, L. R. Hanson, E. L. Aronovich, Z. Nan, W. C. Low et al., Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration, Mol. Genet. Metab, vol.106, pp.131-134, 2012.

Z. Wu, A. Asokan, and R. J. Samulski, Adeno-associated virus serotypes: vector toolkit for human gene therapy, Mol. Ther, vol.14, pp.316-327, 2006.

X. Xiao, J. Li, and R. J. Samulski, Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus, J. Virol, vol.72, pp.2224-2232, 1998.

J. Xie, Q. Xie, H. Zhang, S. L. Ameres, J. H. Hung et al., MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNSrestricted transgene expression, Mol. Ther, vol.19, pp.526-535, 2011.

J. Xu, C. Ma, C. Bass, and E. F. Terwilliger, A combination of mutations enhances the neurotropism of AAV-2, Virology, vol.341, pp.203-214, 2005.

H. Zhang, J. Xie, Q. Xie, J. M. Wilson, and G. Gao, Adenovirusadeno-associated virus hybrid for large-scale recombinant adeno-associated virus production. Hum, Gene Ther, vol.20, pp.922-929, 2009.

H. Zhang, B. Yang, X. Mu, S. S. Ahmed, Q. Su et al., Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system, Mol. Ther, vol.19, pp.1440-1448, 2011.

J. Zhang, X. Wu, C. Qin, J. Qi, S. Ma et al., A novel recombinant adeno-associated virus vaccine reduces behavioral impairment and beta-amyloid plaques in a mouse model of Alzheimer's disease, Neurobiol. Dis, vol.14, pp.365-379, 2003.

L. Zhong, B. Li, C. S. Mah, L. Govindasamy, M. Agbandje-mckenna et al., Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses, Proc. Natl. Acad. Sci. U.S.A, vol.105, pp.7827-7832, 2008.

C. Zincarelli, S. Soltys, G. Rengo, and J. E. Rabinowitz, Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection, Mol. Ther, vol.16, pp.1073-1080, 2008.