메뉴 건너뛰기




Volumn 20, Issue 4, 2009, Pages 350-360

Human α-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: Vector diffusion and biodistribution

Author keywords

[No Author keywords available]

Indexed keywords

GENOMIC DNA; LEVO IDURONIDASE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PARVOVIRUS VECTOR;

EID: 64049083111     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2008.155     Document Type: Article
Times cited : (56)

References (45)
  • 2
    • 0034690714 scopus 로고    scopus 로고
    • Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer
    • Bosch, A., Perret, E., Desmaris, N., Trono, D., and Heard, J.M. (2000). Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum. Gene Ther. 11, 1139-1150.
    • (2000) Hum. Gene Ther , vol.11 , pp. 1139-1150
    • Bosch, A.1    Perret, E.2    Desmaris, N.3    Trono, D.4    Heard, J.M.5
  • 3
    • 0032977833 scopus 로고    scopus 로고
    • Induction of immunity to antigens expressed by recombinant adenoassociated virus depends on the route of administration
    • Brockstedt, D.G., Podsakoff, G.M., Fong, L., Kurtzman, G., Mueller-Ruchholtz, W., and Engleman, E.G. (1999). Induction of immunity to antigens expressed by recombinant adenoassociated virus depends on the route of administration. Clin. Immunol. 92, 67-75.
    • (1999) Clin. Immunol , vol.92 , pp. 67-75
    • Brockstedt, D.G.1    Podsakoff, G.M.2    Fong, L.3    Kurtzman, G.4    Mueller-Ruchholtz, W.5    Engleman, E.G.6
  • 4
    • 4344566457 scopus 로고    scopus 로고
    • Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display 358 differential efficiency and cell tropism after delivery to different regions of the central nervous system
    • Burger, C., Gorbatyuk, O.S., Velardo, M.J., Peden, C.S., Williams, P., Zolotukhin, S., Reier, P.J., Mandel, R.J., and Muzyczka, N. (2004). Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display 358 differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol. Ther. 10, 302-317.
    • (2004) Mol. Ther , vol.10 , pp. 302-317
    • Burger, C.1    Gorbatyuk, O.S.2    Velardo, M.J.3    Peden, C.S.4    Williams, P.5    Zolotukhin, S.6    Reier, P.J.7    Mandel, R.J.8    Muzyczka, N.9
  • 5
    • 22144477188 scopus 로고    scopus 로고
    • Recombinant adeno-associated viral vectors in the nervous system
    • Burger, C., Nash, K., and Mandel, R.J. (2005). Recombinant adeno-associated viral vectors in the nervous system. Hum. Gene Ther. 16, 781-791.
    • (2005) Hum. Gene Ther , vol.16 , pp. 781-791
    • Burger, C.1    Nash, K.2    Mandel, R.J.3
  • 6
    • 34548610909 scopus 로고    scopus 로고
    • A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease
    • Cearley, C.N., and Wolfe, J.H. (2007). A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease. J. Neurosci. 27, 9928-9940.
    • (2007) J. Neurosci , vol.27 , pp. 9928-9940
    • Cearley, C.N.1    Wolfe, J.H.2
  • 9
    • 8544258806 scopus 로고    scopus 로고
    • Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum
    • Cressant, A., Desmaris, N., Verot, L., Brejot, T., Froissart, R., Vanier, M.T., Maire, I., and Heard, J.M. (2004). Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum. J. Neurosci. 24, 10229-10239.
    • (2004) J. Neurosci , vol.24 , pp. 10229-10239
    • Cressant, A.1    Desmaris, N.2    Verot, L.3    Brejot, T.4    Froissart, R.5    Vanier, M.T.6    Maire, I.7    Heard, J.M.8
  • 11
    • 0034724209 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
    • Davidson, B.L., Stein, C.S., Heth, J.A., Martins, I., Kotin, R.M., Derksen, T.A., Zabner, J., Ghodsi, A., and Chiorini, J.A. (2000). 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. 97, 3428-3432.
    • (2000) Proc. Natl. Acad. Sci. U.S.A , vol.97 , pp. 3428-3432
    • Davidson, B.L.1    Stein, C.S.2    Heth, J.A.3    Martins, I.4    Kotin, R.M.5    Derksen, T.A.6    Zabner, J.7    Ghodsi, A.8    Chiorini, J.A.9
  • 13
    • 0035667722 scopus 로고    scopus 로고
    • Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle
    • Favre, D., Provost, N., Blouin, V., Blancho, G., Cherel, Y., Salvetti, A., and Moullier, P. (2001). Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle. Mol. Ther. 4, 559-566.
    • (2001) Mol. Ther , vol.4 , pp. 559-566
    • Favre, D.1    Provost, N.2    Blouin, V.3    Blancho, G.4    Cherel, Y.5    Salvetti, A.6    Moullier, P.7
  • 14
    • 34247270295 scopus 로고    scopus 로고
    • Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites
    • Flotte, T.R., Conlon, T.J., Poirier, A., Campbell-Thompson, M., and Byrne, B.J. (2007). Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites. Hum. Gene Ther. 18, 245-256.
    • (2007) Hum. Gene Ther , vol.18 , pp. 245-256
    • Flotte, T.R.1    Conlon, T.J.2    Poirier, A.3    Campbell-Thompson, M.4    Byrne, B.J.5
  • 16
    • 0038150506 scopus 로고    scopus 로고
    • Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6
    • Grimm, D., Kay, M.A., and Kleinschmidt, J.A. (2003). Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Mol. Ther. 7, 839-850.
    • (2003) Mol. Ther , vol.7 , pp. 839-850
    • Grimm, D.1    Kay, M.A.2    Kleinschmidt, J.A.3
  • 17
    • 33645452310 scopus 로고    scopus 로고
    • Convectionenhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain
    • Hadaczek, P., Kohutnicka, M., Krauze, M.T., Bringas, J., Pivirotto, P., Cunningham, J., and Bankiewicz, K. (2006). Convectionenhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum. Gene Ther. 17, 291-302.
    • (2006) Hum. Gene Ther , vol.17 , pp. 291-302
    • Hadaczek, P.1    Kohutnicka, M.2    Krauze, M.T.3    Bringas, J.4    Pivirotto, P.5    Cunningham, J.6    Bankiewicz, K.7
  • 21
  • 22
    • 37549061385 scopus 로고    scopus 로고
    • AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: Effects of serotype, promoter and purification method
    • Klein, R.L., Dayton, R.D., Tatom, J.B., Henderson, K.M., and Henning, P.P. (2008). AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: Effects of serotype, promoter and purification method. Mol. Ther. 16, 89-96.
    • (2008) Mol. Ther , vol.16 , pp. 89-96
    • Klein, R.L.1    Dayton, R.D.2    Tatom, J.B.3    Henderson, K.M.4    Henning, P.P.5
  • 23
    • 26844431562 scopus 로고    scopus 로고
    • Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors
    • Liu, G., Martins, I., Wemmie, J.A., Chiorini, J.A., and Davidson, B.L. (2005). Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. J. Neurosci. 25, 9321-9327.
    • (2005) J. Neurosci , vol.25 , pp. 9321-9327
    • Liu, G.1    Martins, I.2    Wemmie, J.A.3    Chiorini, J.A.4    Davidson, B.L.5
  • 24
    • 6344283047 scopus 로고    scopus 로고
    • Clinical trials in neurological disorders using AAV vectors: Promises and challenges
    • Mandel, R.J., and Burger, C. (2004). Clinical trials in neurological disorders using AAV vectors: Promises and challenges. Curr. Opin. Mol. Ther. 6, 482-490.
    • (2004) Curr. Opin. Mol. Ther , vol.6 , pp. 482-490
    • Mandel, R.J.1    Burger, C.2
  • 25
    • 33644820684 scopus 로고    scopus 로고
    • Manno, C.S., Pierce, G.F., Arruda, V.R., Glader, B., Ragni, M., Rasko, J.J., Ozelo, M.C., Hoots, K., Blatt, P., Konkle, B., Dake, M., Kaye, R., Razavi, M., Zajko, A., Zehnder, J., Rustagi, P.K., Nakai, H., Chew, A., Leonard, D., Wright, J.F., Lessard, R.R., Sommer, J.M., Tigges, M., Sabatino, D., Luk, A., Jiang, H., Mingozzi, F., Couto, L., Ertl, H.C., High, K.A., and Kay, M.A. (2006). Successful transduction of liver in hemophilia by AAVFactor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347.
    • Manno, C.S., Pierce, G.F., Arruda, V.R., Glader, B., Ragni, M., Rasko, J.J., Ozelo, M.C., Hoots, K., Blatt, P., Konkle, B., Dake, M., Kaye, R., Razavi, M., Zajko, A., Zehnder, J., Rustagi, P.K., Nakai, H., Chew, A., Leonard, D., Wright, J.F., Lessard, R.R., Sommer, J.M., Tigges, M., Sabatino, D., Luk, A., Jiang, H., Mingozzi, F., Couto, L., Ertl, H.C., High, K.A., and Kay, M.A. (2006). Successful transduction of liver in hemophilia by AAVFactor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347.
  • 26
    • 0034980351 scopus 로고    scopus 로고
    • Combined injection of rAAV with mannitol enhances gene expression in the rat brain
    • Mastakov, M.Y., Baer, K., Xu, R., Fitzsimons, H., and During, M.J. (2001). Combined injection of rAAV with mannitol enhances gene expression in the rat brain. Mol. Ther. 3, 225-232.
    • (2001) Mol. Ther , vol.3 , pp. 225-232
    • Mastakov, M.Y.1    Baer, K.2    Xu, R.3    Fitzsimons, H.4    During, M.J.5
  • 27
    • 21244490473 scopus 로고    scopus 로고
    • Adeno-associated virus (AAV) vectors in the CNS
    • McCown, T.J. (2005). Adeno-associated virus (AAV) vectors in the CNS. Curr. Gene Ther. 5, 333-338.
    • (2005) Curr. Gene Ther , vol.5 , pp. 333-338
    • McCown, T.J.1
  • 29
    • 0035800208 scopus 로고    scopus 로고
    • Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain
    • Nguyen, J.B., Sanchez-Pernaute, R., Cunningham, J., and Bankiewicz, K.S. (2001). Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain. Neuroreport 12, 1961-1964.
    • (2001) Neuroreport , vol.12 , pp. 1961-1964
    • Nguyen, J.B.1    Sanchez-Pernaute, R.2    Cunningham, J.3    Bankiewicz, K.S.4
  • 30
    • 19944430177 scopus 로고    scopus 로고
    • Biodistribution of rAAV vectors following intraocular administration: Evidence for the presence and persistence of vector DNA in the optic nerve and in the brain
    • Provost, N., Le Meur, G., Weber, M., Mendes-Madeira, A., Podevin, G., Cherel, Y., Colle, M.A., Deschamps, J.Y., Moullier, P., and Rolling, F. (2005). Biodistribution of rAAV vectors following intraocular administration: Evidence for the presence and persistence of vector DNA in the optic nerve and in the brain. Mol. Ther. 11, 275-283.
    • (2005) Mol. Ther , vol.11 , pp. 275-283
    • Provost, N.1    Le Meur, G.2    Weber, M.3    Mendes-Madeira, A.4    Podevin, G.5    Cherel, Y.6    Colle, M.A.7    Deschamps, J.Y.8    Moullier, P.9    Rolling, F.10
  • 31
    • 0036140216 scopus 로고    scopus 로고
    • Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity
    • Rabinowitz, J.E., Rolling, F., Li, C., Conrath, H., Xiao, W., Xiao, X., and Samulski, R.J. (2002). Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity. J. Virol. 76, 791-801.
    • (2002) J. Virol , vol.76 , pp. 791-801
    • Rabinowitz, J.E.1    Rolling, F.2    Li, C.3    Conrath, H.4    Xiao, W.5    Xiao, X.6    Samulski, R.J.7
  • 34
    • 0032758906 scopus 로고    scopus 로고
    • Sustained production of β-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice
    • Skorupa, A.F., Fisher, K.J., Wilson, J.M., Parente, M.K., and Wolfe, J.H. (1999). Sustained production of β-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice. Exp. Neurol. 160, 17-27.
    • (1999) Exp. Neurol , vol.160 , pp. 17-27
    • Skorupa, A.F.1    Fisher, K.J.2    Wilson, J.M.3    Parente, M.K.4    Wolfe, J.H.5
  • 35
    • 27944437854 scopus 로고    scopus 로고
    • Sondhi, D., Peterson, D.A., Giannaris, E.L., Sanders, C.T., Mendez, B.S., De, B., Rostkowski, A.B., Blanchard, B., Bjugstad, K., Sladek, J.R., Jr., Redmond, D.E., Jr., Leopold, P.L., Kaminsky, S.M., Hackett, N.R., and Crystal, R.G. (2005). AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL. Gene Ther. 12, 1618-1632.
    • Sondhi, D., Peterson, D.A., Giannaris, E.L., Sanders, C.T., Mendez, B.S., De, B., Rostkowski, A.B., Blanchard, B., Bjugstad, K., Sladek, J.R., Jr., Redmond, D.E., Jr., Leopold, P.L., Kaminsky, S.M., Hackett, N.R., and Crystal, R.G. (2005). AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL. Gene Ther. 12, 1618-1632.
  • 36
    • 33847211798 scopus 로고    scopus 로고
    • Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector
    • Sondhi, D., Hackett, N.R., Peterson, D.A., Stratton, J., Baad, M., Travis, K.M., Wilson, J.M., and Crystal, R.G. (2007). Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector. Mol. Ther. 15, 481-491.
    • (2007) Mol. Ther , vol.15 , pp. 481-491
    • Sondhi, D.1    Hackett, N.R.2    Peterson, D.A.3    Stratton, J.4    Baad, M.5    Travis, K.M.6    Wilson, J.M.7    Crystal, R.G.8
  • 40
    • 0141996932 scopus 로고    scopus 로고
    • Adeno-associated virus vector-mediated transduction in the cat brain
    • Vite, C.H., Passini, M.A., Haskins, M.E., and Wolfe, J.H. (2003). Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther. 10, 1874-1881.
    • (2003) Gene Ther , vol.10 , pp. 1874-1881
    • Vite, C.H.1    Passini, M.A.2    Haskins, M.E.3    Wolfe, J.H.4
  • 42
    • 10744229842 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery
    • Weber, M., Rabinowitz, J., Provost, N., Conrath, H., Folliot, S., Briot, D., Cherel, Y., Chenuaud, P., Samulski, J., Moullier, P., and Rolling, F. (2003). Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery. Mol. Ther. 7, 774-781.
    • (2003) Mol. Ther , vol.7 , pp. 774-781
    • Weber, M.1    Rabinowitz, J.2    Provost, N.3    Conrath, H.4    Folliot, S.5    Briot, D.6    Cherel, Y.7    Chenuaud, P.8    Samulski, J.9    Moullier, P.10    Rolling, F.11
  • 43
    • 0031779697 scopus 로고    scopus 로고
    • Adenoassociated virus vector-mediated transgene integration into neurons and other nondividing cell targets
    • Wu, P., Phillips, M.I., Bui, J., and Terwilliger, E.F. (1998). Adenoassociated virus vector-mediated transgene integration into neurons and other nondividing cell targets. J. Virol. 72, 5919-5926.
    • (1998) J. Virol , vol.72 , pp. 5919-5926
    • Wu, P.1    Phillips, M.I.2    Bui, J.3    Terwilliger, E.F.4
  • 44
    • 0031104792 scopus 로고    scopus 로고
    • Gene transfer by adeno-associated virus vectors into the central nervous system
    • Xiao, X., Li, J., McCown, T.J., and Samulski, R.J. (1997). Gene transfer by adeno-associated virus vectors into the central nervous system. Exp. Neurol. 144, 113-124.
    • (1997) Exp. Neurol , vol.144 , pp. 113-124
    • Xiao, X.1    Li, J.2    McCown, T.J.3    Samulski, R.J.4
  • 45
    • 0038618928 scopus 로고    scopus 로고
    • Efficiencies of transgene expression in nociceptive neurons through different routes of delivery of adeno-associated viral vectors
    • Xu, Y., Gu, Y., Wu, P., Li, G.W., and Huang, L.Y. (2003). Efficiencies of transgene expression in nociceptive neurons through different routes of delivery of adeno-associated viral vectors. Hum. Gene Ther. 14, 897-906.
    • (2003) Hum. Gene Ther , vol.14 , pp. 897-906
    • Xu, Y.1    Gu, Y.2    Wu, P.3    Li, G.W.4    Huang, L.Y.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.