{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T12:13:34Z","timestamp":1780316014785,"version":"3.54.1"},"publisher-location":"New York, NY","reference-count":48,"publisher":"Springer New York","isbn-type":[{"value":"9781493932702","type":"print"},{"value":"9781493932719","type":"electronic"}],"license":[{"start":{"date-parts":[[2016,1,1]],"date-time":"2016-01-01T00:00:00Z","timestamp":1451606400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"},{"start":{"date-parts":[[2016,1,1]],"date-time":"2016-01-01T00:00:00Z","timestamp":1451606400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springernature.com\/gp\/researchers\/text-and-data-mining"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2016]]},"DOI":"10.1007\/978-1-4939-3271-9_1","type":"book-chapter","created":{"date-parts":[[2015,11,26]],"date-time":"2015-11-26T06:47:47Z","timestamp":1448520467000},"page":"3-18","source":"Crossref","is-referenced-by-count":9,"title":["Introduction to Viral Vectors and Other Delivery Methods for Gene Therapy of the Nervous System"],"prefix":"10.1007","author":[{"given":"Fredric P.","family":"Manfredsson","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","reference":[{"key":"1_CR1","first-page":"204","volume":"9","author":"FP Manfredsson","year":"2010","unstructured":"Manfredsson FP, Mandel RJ (2010) Development of gene therapy for neurological disorders. Discov Med 9:204\u2013211","journal-title":"Discov Med"},{"key":"1_CR2","doi-asserted-by":"publisher","first-page":"488","DOI":"10.1038\/mt.2015.7","volume":"23","author":"MJ Benskey","year":"2015","unstructured":"Benskey MJ et al (2015) Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants. Mol Ther 23:488\u2013500","journal-title":"Mol Ther"},{"key":"1_CR3","doi-asserted-by":"publisher","first-page":"302","DOI":"10.1016\/j.ymthe.2004.05.024","volume":"10","author":"C Burger","year":"2004","unstructured":"Burger C et al (2004) Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol Ther 10:302\u2013317","journal-title":"Mol Ther"},{"key":"1_CR4","doi-asserted-by":"publisher","first-page":"2331","DOI":"10.1089\/104303400750038444","volume":"11","author":"JT Evans","year":"2000","unstructured":"Evans JT, Garcia JV (2000) Lentivirus vector mobilization and spread by human immunodeficiency virus. Hum Gene Ther 11:2331\u20132339","journal-title":"Hum Gene Ther"},{"key":"1_CR5","doi-asserted-by":"publisher","first-page":"415","DOI":"10.1126\/science.1088547","volume":"302","author":"S Hacein-Bey-Abina","year":"2003","unstructured":"Hacein-Bey-Abina S et al (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302:415\u2013419","journal-title":"Science"},{"key":"1_CR6","doi-asserted-by":"publisher","first-page":"348","DOI":"10.1038\/nm1365","volume":"12","author":"RJ Yanez-Munoz","year":"2006","unstructured":"Yanez-Munoz RJ et al (2006) Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 12:348\u2013353","journal-title":"Nat Med"},{"key":"1_CR7","doi-asserted-by":"publisher","first-page":"41","DOI":"10.1016\/j.expneurol.2010.10.016","volume":"228","author":"JR Cannon","year":"2011","unstructured":"Cannon JR, Sew T, Montero L, Burton EA, Greenamyre JT (2011) Pseudotype-dependent lentiviral transduction of astrocytes or neurons in the rat substantia nigra. Exp Neurol 228:41\u201352","journal-title":"Exp Neurol"},{"key":"1_CR8","doi-asserted-by":"crossref","first-page":"1330","DOI":"10.1038\/sj.gt.3302988","volume":"14","author":"B Liehl","year":"2007","unstructured":"Liehl B et al (2007) Simian immunodeficiency virus vector pseudotypes differ in transduction efficiency and target cell specificity in brain. Gene Ther 14:1330\u20131343","journal-title":"Gene Ther"},{"key":"1_CR9","doi-asserted-by":"publisher","first-page":"528","DOI":"10.1006\/mthe.2002.0584","volume":"5","author":"DJ Watson","year":"2002","unstructured":"Watson DJ, Kobinger GP, Passini MA, Wilson JM, Wolfe JH (2002) Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins. Mol Ther 5:528\u2013537","journal-title":"Mol Ther"},{"key":"1_CR10","doi-asserted-by":"publisher","first-page":"201","DOI":"10.1016\/j.expneurol.2011.03.013","volume":"229","author":"FP Manfredsson","year":"2011","unstructured":"Manfredsson FP, Mandel RJ (2011) The development of flexible lentiviral vectors for gene transfer in the CNS. Exp Neurol 229:201\u2013206","journal-title":"Exp Neurol"},{"issue":"S1","key":"1_CR11","doi-asserted-by":"crossref","first-page":"S18","DOI":"10.1038\/sj.gt.3302612","volume":"12","author":"R Alba","year":"2005","unstructured":"Alba R, Bosch A, Chillon M (2005) Gutless adenovirus: last-generation adenovirus for gene therapy. Gene Ther 12 Suppl 1: S18\u2013S27","journal-title":"Gene Therapy"},{"key":"1_CR12","doi-asserted-by":"publisher","first-page":"13090","DOI":"10.1073\/pnas.202486199","volume":"99","author":"I Semkova","year":"2002","unstructured":"Semkova I et al (2002) Autologous transplantation of genetically modified iris pigment epithelial cells: a promising concept for the treatment of age-related macular degeneration and other disorders of the eye. Proc Natl Acad Sci U S A 99:13090\u201313095","journal-title":"Proc Natl Acad Sci U S A"},{"key":"1_CR13","doi-asserted-by":"crossref","first-page":"391","DOI":"10.1096\/fj.03-0438fje","volume":"18","author":"C Soudais","year":"2004","unstructured":"Soudais C, Skander N, Kremer EJ (2004) Long-term in vivo transduction of neurons throughout the rat CNS using novel helper-dependent CAV-2 vectors. FASEB J 18:391\u2013393","journal-title":"FASEB J"},{"key":"1_CR14","doi-asserted-by":"publisher","first-page":"3293","DOI":"10.3390\/v6083293","volume":"6","author":"TB Lewis","year":"2014","unstructured":"Lewis TB, Glasgow JN, Harms AS, Standaert DG, Curiel DT (2014) Fiber-modified adenovirus for central nervous system Parkinson\u2019s disease gene therapy. Viruses 6:3293\u20133310","journal-title":"Viruses"},{"key":"1_CR15","doi-asserted-by":"publisher","first-page":"448","DOI":"10.1016\/j.biopha.2007.12.009","volume":"62","author":"M Sakae","year":"2008","unstructured":"Sakae M et al (2008) Highly efficient in vivo gene transfection by plasmid\/PEI complexes coated by anionic PEG derivatives bearing carboxyl groups and RGD peptide. Biomed Pharmacother 62:448\u2013453","journal-title":"Biomed Pharmacother"},{"key":"1_CR16","doi-asserted-by":"publisher","first-page":"16589","DOI":"10.1073\/pnas.1009158107","volume":"107","author":"E Portales-Casamar","year":"2010","unstructured":"Portales-Casamar E et al (2010) A regulatory toolbox of MiniPromoters to drive selective expression in the brain. Proc Natl Acad Sci U S A 107:16589\u201316594","journal-title":"Proc Natl Acad Sci U S A"},{"key":"1_CR17","doi-asserted-by":"publisher","first-page":"1770","DOI":"10.1038\/ncomms2801","volume":"4","author":"M Akerblom","year":"2013","unstructured":"Akerblom M et al (2013) Visualization and genetic modification of resident brain microglia using lentiviral vectors regulated by microRNA-9. Nat Commun 4:1770","journal-title":"Nat Commun"},{"key":"1_CR18","doi-asserted-by":"publisher","first-page":"47","DOI":"10.3389\/fncir.2012.00047","volume":"6","author":"A Saunders","year":"2012","unstructured":"Saunders A, Johnson CA, Sabatini BL (2012) Novel recombinant adeno-associated viruses for Cre activated and inactivated transgene expression in neurons. Front Neural Circuits 6:47","journal-title":"Front Neural Circuits"},{"key":"1_CR19","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0067680","volume":"8","author":"P Chakrabarty","year":"2013","unstructured":"Chakrabarty P et al (2013) Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain. PLoS One 8, e67680","journal-title":"PLoS One"},{"key":"1_CR20","doi-asserted-by":"publisher","first-page":"1018","DOI":"10.1038\/mt.2008.73","volume":"16","author":"C Towne","year":"2008","unstructured":"Towne C, Raoul C, Schneider BL, Aebischer P (2008) Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice. Mol Ther 16:1018\u20131025","journal-title":"Mol Ther"},{"key":"1_CR21","doi-asserted-by":"publisher","first-page":"1971","DOI":"10.1038\/mt.2011.157","volume":"19","author":"AK Bevan","year":"2011","unstructured":"Bevan AK et al (2011) Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol Ther 19:1971\u20131980","journal-title":"Mol Ther"},{"key":"1_CR22","doi-asserted-by":"publisher","first-page":"198","DOI":"10.1038\/nbt1182","volume":"24","author":"N Maheshri","year":"2006","unstructured":"Maheshri N, Koerber JT, Kaspar BK, Schaffer DV (2006) Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 24:198\u2013204","journal-title":"Nat Biotechnol"},{"key":"1_CR23","doi-asserted-by":"publisher","first-page":"570","DOI":"10.1038\/mt.2009.292","volume":"18","author":"SJ Gray","year":"2010","unstructured":"Gray SJ et al (2010) Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood\u2013brain barrier (BBB). Mol Ther 18:570\u2013578","journal-title":"Mol Ther"},{"key":"1_CR24","doi-asserted-by":"publisher","first-page":"375","DOI":"10.2174\/156652309789753400","volume":"9","author":"FP Manfredsson","year":"2009","unstructured":"Manfredsson FP, Okun MS, Mandel RJ (2009) Gene therapy for neurological disorders: challenges and future prospects for the use of growth factors for the treatment of Parkinson's disease. Curr Gene Ther 9:375\u2013388","journal-title":"Curr Gene Ther"},{"key":"1_CR25","doi-asserted-by":"publisher","first-page":"1857","DOI":"10.1038\/mt.2009.196","volume":"17","author":"FP Manfredsson","year":"2009","unstructured":"Manfredsson FP et al (2009) Tight Long-term dynamic doxycycline responsive nigrostriatal GDNF using a single rAAV vector. Mol Ther 17:1857\u20131867","journal-title":"Mol Ther"},{"key":"1_CR26","doi-asserted-by":"publisher","first-page":"605","DOI":"10.1038\/33416","volume":"392","author":"T Kitada","year":"1998","unstructured":"Kitada T et al (1998) Mutations in the parkin gene cause autosomal recessive juvenile parkinsonism. Nature 392:605\u2013608","journal-title":"Nature"},{"key":"1_CR27","doi-asserted-by":"publisher","first-page":"289","DOI":"10.1016\/j.expneurol.2007.06.019","volume":"207","author":"FP Manfredsson","year":"2007","unstructured":"Manfredsson FP et al (2007) rAAV-mediated nigral human parkin over-expression partially ameliorates motor deficits via enhanced dopamine neurotransmission in a rat model of Parkinson's disease. Exp Neurol 207:289\u2013301","journal-title":"Exp Neurol"},{"key":"1_CR28","doi-asserted-by":"publisher","first-page":"716","DOI":"10.1016\/j.ymthe.2006.06.009","volume":"14","author":"L Vercammen","year":"2006","unstructured":"Vercammen L et al (2006) Parkin protects against neurotoxicity in the 6-hydroxydopamine rat model for Parkinson's disease. Mol Ther 14:716\u2013723","journal-title":"Mol Ther"},{"key":"1_CR29","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0081426","volume":"8","author":"SE Gombash","year":"2013","unstructured":"Gombash SE et al (2013) Morphological and behavioral impact of AAV2\/5-mediated overexpression of human wildtype alpha-synuclein in the rat nigrostriatal system. PLoS One 8, e81426","journal-title":"PLoS One"},{"key":"1_CR30","doi-asserted-by":"crossref","first-page":"249","DOI":"10.3233\/JPD-012138","volume":"2","author":"NM Kanaan","year":"2012","unstructured":"Kanaan NM, Manfredsson FP (2012) Loss of functional alpha-synuclein: a toxic event in Parkinson's disease? Journal of Parkinson's disease 2:249\u2013267","journal-title":"Journal of Parkinson's disease"},{"key":"1_CR31","doi-asserted-by":"publisher","first-page":"243","DOI":"10.1016\/j.nbd.2011.12.030","volume":"48","author":"S Ramaswamy","year":"2012","unstructured":"Ramaswamy S, Kordower JH (2012) Gene therapy for Huntington's disease. Neurobiol Dis 48:243\u2013254","journal-title":"Neurobiol Dis"},{"key":"1_CR32","doi-asserted-by":"publisher","first-page":"1053","DOI":"10.1038\/mt.2009.17","volume":"17","author":"RL Boudreau","year":"2009","unstructured":"Boudreau RL et al (2009) Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 17:1053\u20131063","journal-title":"Mol Ther"},{"key":"1_CR33","doi-asserted-by":"publisher","first-page":"2152","DOI":"10.1038\/mt.2011.219","volume":"19","author":"JL McBride","year":"2011","unstructured":"McBride JL et al (2011) Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease. Mol Ther 19:2152\u20132162","journal-title":"Mol Ther"},{"key":"1_CR34","doi-asserted-by":"publisher","first-page":"537","DOI":"10.1038\/nature04791","volume":"441","author":"D Grimm","year":"2006","unstructured":"Grimm D et al (2006) Fatality in mice due to oversaturation of cellular microRNA\/short hairpin RNA pathways. Nature 441:537\u2013541","journal-title":"Nature"},{"key":"1_CR35","doi-asserted-by":"publisher","first-page":"1450","DOI":"10.1038\/mt.2010.115","volume":"18","author":"OS Gorbatyuk","year":"2010","unstructured":"Gorbatyuk OS et al (2010) In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration. Mol Ther 18:1450\u20131457","journal-title":"Mol Ther"},{"key":"1_CR36","doi-asserted-by":"publisher","first-page":"5955","DOI":"10.1093\/nar\/gkr196","volume":"39","author":"F Chan","year":"2011","unstructured":"Chan F, Hauswirth WW, Wensel TG, Wilson JH (2011) Efficient mutagenesis of the rhodopsin gene in rod photoreceptor neurons in mice. Nucleic Acids Res 39:5955\u20135966","journal-title":"Nucleic Acids Res"},{"key":"1_CR37","doi-asserted-by":"publisher","first-page":"1258096","DOI":"10.1126\/science.1258096","volume":"346","author":"JA Doudna","year":"2014","unstructured":"Doudna JA, Charpentier E (2014) Genome editing. The new frontier of genome engineering with CRISPR-Cas9. Science 346:1258096","journal-title":"Science"},{"key":"1_CR38","doi-asserted-by":"publisher","first-page":"636","DOI":"10.1038\/nrg2842","volume":"11","author":"FD Urnov","year":"2010","unstructured":"Urnov FD, Rebar EJ, Holmes MC, Zhang HS, Gregory PD (2010) Genome editing with engineered zinc finger nucleases. Nat Rev Genet 11:636\u2013646","journal-title":"Nat Rev Genet"},{"key":"1_CR39","doi-asserted-by":"publisher","first-page":"15","DOI":"10.1042\/BJ20140295","volume":"462","author":"DA Wright","year":"2014","unstructured":"Wright DA, Li T, Yang B, Spalding MH (2014) TALEN-mediated genome editing: prospects and perspectives. Biochem J 462:15\u201324","journal-title":"Biochem J"},{"key":"1_CR40","doi-asserted-by":"publisher","first-page":"589","DOI":"10.1038\/nbt957","volume":"22","author":"AL Szymczak","year":"2004","unstructured":"Szymczak AL et al (2004) Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector. Nat Biotechnol 22:589\u2013594","journal-title":"Nat Biotechnol"},{"key":"1_CR41","doi-asserted-by":"publisher","first-page":"212","DOI":"10.1016\/j.nbd.2012.03.001","volume":"48","author":"FP Manfredsson","year":"2012","unstructured":"Manfredsson FP, Bloom DC, Mandel RJ (2012) Regulated protein expression for in vivo gene therapy for neurological disorders: progress, strategies, and issues. Neurobiol Dis 48:212\u2013221","journal-title":"Neurobiol Dis"},{"key":"1_CR42","doi-asserted-by":"publisher","first-page":"5547","DOI":"10.1073\/pnas.89.12.5547","volume":"89","author":"M Gossen","year":"1992","unstructured":"Gossen M, Bujard H (1992) Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci U S A 89:5547\u20135551","journal-title":"Proc Natl Acad Sci U S A"},{"key":"1_CR43","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1097\/01.jnen.0000218444.53405.f0","volume":"65","author":"FP Wachs","year":"2006","unstructured":"Wachs FP et al (2006) Transforming growth factor-beta1 is a negative modulator of adult neurogenesis. J Neuropathol Exp Neurol 65:358\u2013370","journal-title":"J Neuropathol Exp Neurol"},{"key":"1_CR44","doi-asserted-by":"publisher","first-page":"2169","DOI":"10.1038\/mt.2013.169","volume":"21","author":"L Quintino","year":"2013","unstructured":"Quintino L et al (2013) Functional neuroprotection and efficient regulation of GDNF using destabilizing domains in a rodent model of Parkinson's disease. Mol Ther 21:2169\u20132180","journal-title":"Mol Ther"},{"key":"1_CR45","doi-asserted-by":"publisher","first-page":"695","DOI":"10.1161\/hy0202.103784","volume":"39","author":"Y Tang","year":"2002","unstructured":"Tang Y, Jackson M, Qian K, Phillips MI (2002) Hypoxia inducible double plasmid system for myocardial ischemia gene therapy. Hypertension 39:695\u2013698","journal-title":"Hypertension"},{"key":"1_CR46","doi-asserted-by":"publisher","first-page":"1271","DOI":"10.1038\/gt.2008.75","volume":"15","author":"H Hurttila","year":"2008","unstructured":"Hurttila H et al (2008) Oxidative stress-inducible lentiviral vectors for gene therapy. Gene Ther 15:1271\u20131279","journal-title":"Gene Ther"},{"key":"1_CR47","doi-asserted-by":"publisher","first-page":"517","DOI":"10.1016\/j.ymthe.2005.10.008","volume":"13","author":"RL Klein","year":"2006","unstructured":"Klein RL et al (2006) Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fluorescent proteins. Mol Ther 13:517\u2013527","journal-title":"Mol Ther"},{"key":"1_CR48","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0120120","volume":"10","author":"TM Grabinski","year":"2015","unstructured":"Grabinski TM, Kneynsberg A, Manfredsson FP, Kanaan NM (2015) A Method for Combining RNAscope In Situ Hybridization with Immunohistochemistry in Thick Free-Floating Brain Sections and Primary Neuronal Cultures. PLoS One 10, e0120120","journal-title":"PLoS One"}],"container-title":["Methods in Molecular Biology","Gene Therapy for Neurological Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-4939-3271-9_1","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,2,15]],"date-time":"2023-02-15T18:41:06Z","timestamp":1676486466000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/978-1-4939-3271-9_1"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016]]},"ISBN":["9781493932702","9781493932719"],"references-count":48,"URL":"https:\/\/doi.org\/10.1007\/978-1-4939-3271-9_1","relation":{},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"value":"1064-3745","type":"print"},{"value":"1940-6029","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016]]}}}