메뉴 건너뛰기




Volumn 24, Issue 2, 2016, Pages 276-286

A comprehensive map of CNS transduction by eight recombinant adeno-associated virus serotypes upon cerebrospinal fluid administration in pigs

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; GREEN FLUORESCENT PROTEIN;

EID: 84959503797     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.212     Document Type: Article
Times cited : (14)

References (30)
  • 2
    • 44349136847 scopus 로고    scopus 로고
    • Treatment of late infantile neuronal ceroid lipofuscinosis by cns administration of a serotype 2 adeno-associated virus expressing cln2 cdna
    • Worgall, S, Sondhi, D, Hackett, NR, Kosofsky, B, Kekatpure, MV, Neyzi, N, et al. (2008). 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) Hum Gene Ther , vol.19 , pp. 463-474
    • Worgall, S.1    Sondhi, D.2    Hackett, N.R.3    Kosofsky, B.4    Kekatpure, M.V.5    Neyzi, N.6
  • 5
    • 78349247631 scopus 로고    scopus 로고
    • Gene delivery of AAV2-neurturin for Parkinson's disease: A double-blind, randomised, controlled trial
    • Marks, WJ Jr, Bartus, RT, Siffert, J, Davis, CS, Lozano, A, Boulis, N, et al. (2010). Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol 9: 1164-1172.
    • (2010) Lancet Neurol , vol.9 , pp. 1164-1172
    • Marks, W.J.1    Bartus, R.T.2    Siffert, J.3    Davis, C.S.4    Lozano, A.5    Boulis, N.6
  • 6
    • 79952740079 scopus 로고    scopus 로고
    • AAV2-GAD gene therapy for advanced Parkinson's disease: A double-blind, sham-surgery controlled, randomised trial
    • LeWitt, PA, Rezai, AR, Leehey, MA, Ojemann, SG, Flaherty, AW, Eskandar, EN, et al. (2011). AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol 10: 309-319.
    • (2011) Lancet Neurol , vol.10 , pp. 309-319
    • LeWitt, P.A.1    Rezai, A.R.2    Leehey, M.A.3    Ojemann, S.G.4    Flaherty, A.W.5    Eskandar, E.N.6
  • 8
    • 84903760379 scopus 로고    scopus 로고
    • Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10
    • Yang, B, Li, S, Wang, H, Guo, Y, Gessler, DJ, Cao, C, et al. (2014). Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther 22: 1299-1309.
    • (2014) Mol Ther , vol.22 , pp. 1299-1309
    • Yang, B.1    Li, S.2    Wang, H.3    Guo, Y.4    Gessler, D.J.5    Cao, C.6
  • 9
    • 84927081397 scopus 로고    scopus 로고
    • Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice
    • Duncan, FJ, Naughton, BJ, Zaraspe, K, Murrey, DA, Meadows, AS, Clark, KR, et al. (2015). Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice. Mol Ther 23: 638-647.
    • (2015) Mol Ther , vol.23 , pp. 638-647
    • Duncan, F.J.1    Naughton, B.J.2    Zaraspe, K.3    Murrey, D.A.4    Meadows, A.S.5    Clark, K.R.6
  • 10
    • 70350502352 scopus 로고    scopus 로고
    • Molecular signatures of disease brain endothelia provide new sites for CNS-Directed enzyme therapy
    • Chen, YH, Chang, M and Davidson, BL (2009). Molecular signatures of disease brain endothelia provide new sites for CNS-Directed enzyme therapy. Nat Med 15: 1215-1218.
    • (2009) Nat Med , vol.15 , pp. 1215-1218
    • Chen, Y.H.1    Chang, M.2    Davidson, B.L.3
  • 12
    • 84865123660 scopus 로고    scopus 로고
    • A paravascular pathway facilitates CSF flow through the brain parenchyma and the clearance of interstitial solutes, including amyloid
    • 147ra111
    • Iliff, JJ, Wang, M, Liao, Y, Plogg, BA, Peng, W, Gundersen, GA, et al. (2012). A paravascular pathway facilitates CSF flow through the brain parenchyma and the clearance of interstitial solutes, including amyloid. Sci Transl Med 4: 147ra111.
    • (2012) Sci Transl Med , vol.4
    • Iliff, J.J.1    Wang, M.2    Liao, Y.3    Plogg, B.A.4    Peng, W.5    Gundersen, G.A.6
  • 13
    • 84907299494 scopus 로고    scopus 로고
    • Biology of adeno-Associated viral vectors in the central nervous system
    • Murlidharan, G, Samulski, RJ and Asokan, A (2014). Biology of adeno-Associated viral vectors in the central nervous system. Front Mol Neurosci 7: 76.
    • (2014) Front Mol Neurosci , vol.7 , pp. 76
    • Murlidharan, G.1    Samulski, R.J.2    Asokan, A.3
  • 14
    • 84927155708 scopus 로고    scopus 로고
    • Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis i
    • Hinderer, C, Bell, P, Gurda, BL, Wang, Q, Louboutin, JP, Zhu, Y, et al. (2014). Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I. Mol Ther 22: 2018-2027.
    • (2014) Mol Ther , vol.22 , pp. 2018-2027
    • Hinderer, C.1    Bell, P.2    Gurda, B.L.3    Wang, Q.4    Louboutin, J.P.5    Zhu, Y.6
  • 15
    • 84881228888 scopus 로고    scopus 로고
    • Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
    • epub ahead of print)
    • Haurigot, V, Marco, S, Ribera, A, Garcia, M, Ruzo, A, Villacampa, P, et al. (2013). Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. J Clin Invest (epub ahead of print
    • (2013) J Clin Invest.
    • Haurigot, V.1    Marco, S.2    Ribera, A.3    Garcia, M.4    Ruzo, A.5    Villacampa, P.6
  • 16
    • 84877847142 scopus 로고    scopus 로고
    • Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates
    • Samaranch, L, Salegio, EA, San Sebastian, W, Kells, AP, Bringas, JR, Forsayeth, J, et al. (2013). Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates. Hum Gene Ther 24: 526-532.
    • (2013) Hum Gene Ther , vol.24 , pp. 526-532
    • Samaranch, L.1    Salegio, E.A.2    San Sebastian, W.3    Kells, A.P.4    Bringas, J.R.5    Forsayeth, J.6
  • 17
    • 84926421140 scopus 로고    scopus 로고
    • Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats
    • Hordeaux, J, Dubreil, L, Deniaud, J, Iacobelli, F, Moreau, S, Ledevin, M, et al. (2015). Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats. Gene Ther 22: 316-324.
    • (2015) Gene Ther , vol.22 , pp. 316-324
    • Hordeaux, J.1    Dubreil, L.2    Deniaud, J.3    Iacobelli, F.4    Moreau, S.5    Ledevin, M.6
  • 18
    • 84875240244 scopus 로고    scopus 로고
    • Adeno-Associated virus (AAV) gene therapy for neurological disease
    • Weinberg, MS, Samulski, RJ and McCown, TJ (2013). Adeno-Associated virus (AAV) gene therapy for neurological disease. Neuropharmacology 69: 82-88.
    • (2013) Neuropharmacology , vol.69 , pp. 82-88
    • Weinberg, M.S.1    Samulski, R.J.2    McCown, T.J.3
  • 20
    • 84927738438 scopus 로고    scopus 로고
    • Access to cerebrospinal fluid in piglets via the cisterna magna: Optimization and description of the technique
    • Romagnoli, N, Ventrella, D, Giunti, M, Dondi, F, Sorrentino, NC, Fraldi, A, et al. (2014). Access to cerebrospinal fluid in piglets via the cisterna magna: optimization and description of the technique. Lab Anim 48: 345-348.
    • (2014) Lab Anim , vol.48 , pp. 345-348
    • Romagnoli, N.1    Ventrella, D.2    Giunti, M.3    Dondi, F.4    Sorrentino, N.C.5    Fraldi, A.6
  • 21
    • 0031604307 scopus 로고    scopus 로고
    • Clinical biochemistry, haematology and body weight in piglets
    • Egeli, AK, Framstad, T and Morberg, H (1998). Clinical biochemistry, haematology and body weight in piglets. Acta Vet Scand 39: 381-393.
    • (1998) Acta Vet Scand , vol.39 , pp. 381-393
    • Egeli, A.K.1    Framstad, T.2    Morberg, H.3
  • 22
    • 79955574241 scopus 로고    scopus 로고
    • Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder
    • Spampanato, C, De Leonibus, E, Dama, P, Gargiulo, A, Fraldi, A, Sorrentino, NC, et al. (2011). Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder. Mol Ther 19: 860-869.
    • (2011) Mol Ther , vol.19 , pp. 860-869
    • Spampanato, C.1    De Leonibus, E.2    Dama, P.3    Gargiulo, A.4    Fraldi, A.5    Sorrentino, N.C.6
  • 23
    • 84875925195 scopus 로고    scopus 로고
    • Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
    • Gray, SJ, Nagabhushan Kalburgi, S, McCown, TJ and Jude Samulski, R (2013). Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates. Gene Ther 20: 450-459.
    • (2013) Gene Ther , vol.20 , pp. 450-459
    • Gray, S.J.1    Nagabhushan Kalburgi, S.2    McCown, T.J.3    Jude Samulski, R.4
  • 24
    • 84864877570 scopus 로고    scopus 로고
    • Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs
    • Federici, T, Taub, JS, Baum, GR, Gray, SJ, Grieger, JC, Matthews, KA, et al. (2012). Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther 19: 852-859.
    • (2012) Gene Ther , vol.19 , pp. 852-859
    • Federici, T.1    Taub, J.S.2    Baum, G.R.3    Gray, S.J.4    Grieger, J.C.5    Matthews, K.A.6
  • 25
    • 84895532287 scopus 로고    scopus 로고
    • Humoral immune response to aav
    • Calcedo, R and Wilson, JM (2013). Humoral Immune Response to AAV. Front Immunol 4: 341.
    • (2013) Front Immunol , vol.4 , pp. 341
    • Calcedo, R.1    Wilson, J.M.2
  • 26
    • 84904412918 scopus 로고    scopus 로고
    • Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
    • Passini, MA, Bu, J, Richards, AM, Treleaven, CM, Sullivan, JA, O'Riordan, CR, et al. (2014). Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Hum Gene Ther 25: 619-630.
    • (2014) Hum Gene Ther , vol.25 , pp. 619-630
    • Passini, M.A.1    Bu, J.2    Richards, A.M.3    Treleaven, C.M.4    Sullivan, J.A.5    O'Riordan, C.R.6
  • 27
    • 84893307092 scopus 로고    scopus 로고
    • AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction
    • Samaranch, L, San Sebastian, W, Kells, AP, Salegio, EA, Heller, G, Bringas, JR, et al. (2014). AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction. Mol Ther 22: 329-337.
    • (2014) Mol Ther , vol.22 , pp. 329-337
    • Samaranch, L.1    San Sebastian, W.2    Kells, A.P.3    Salegio, E.A.4    Heller, G.5    Bringas, J.R.6
  • 28
    • 84890387154 scopus 로고    scopus 로고
    • AAV2/8 vectors purified from culture medium with a simple and rapid protocol transduce murine liver, muscle and retina efficiently
    • Doria, M, Ferrara, A and Auricchio, A (2013). AAV2/8 vectors purified from culture medium with a simple and rapid protocol transduce murine liver, muscle and retina efficiently. Hum Gene Ther Methods 24: 392-398.
    • (2013) Hum Gene Ther Methods , vol.24 , pp. 392-398
    • Doria, M.1    Ferrara, A.2    Auricchio, A.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.