-
1
-
-
0033942230
-
Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach
-
Bankiewicz K.S., Eberling J.L., Kohutnicka M., Jagust W., Pivivrotto P., Bringas J., and Cunningham J. Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Exp. Neurol. 164 (2000) 2-14
-
(2000)
Exp. Neurol.
, vol.164
, pp. 2-14
-
-
Bankiewicz, K.S.1
Eberling, J.L.2
Kohutnicka, M.3
Jagust, W.4
Pivivrotto, P.5
Bringas, J.6
Cunningham, J.7
-
2
-
-
0036852208
-
A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5
-
Brument N., Morenweiser R., Blouin V., Toublance E., Raimbaud I., Cherel Y., Folliot S., Gaden F., Boulannnger P., Kroner-Lux G., Moullier P., Rolling F., and Salvetti A. A versatile and scalable two-step ion-exchange chromatography process for the purification of recombinant adeno-associated virus serotypes-2 and -5. Mol. Ther. 6 (2002) 678-686
-
(2002)
Mol. Ther.
, vol.6
, pp. 678-686
-
-
Brument, N.1
Morenweiser, R.2
Blouin, V.3
Toublance, E.4
Raimbaud, I.5
Cherel, Y.6
Folliot, S.7
Gaden, F.8
Boulannnger, P.9
Kroner-Lux, G.10
Moullier, P.11
Rolling, F.12
Salvetti, A.13
-
3
-
-
0033607305
-
Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
-
Burton M., Nakai H., Colosi P., Cunningham J., Mitchell R., and Couto L. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proc. Natl. Acad. Sci. U.S.A. 96 (1999) 12725-12730
-
(1999)
Proc. Natl. Acad. Sci. U.S.A.
, vol.96
, pp. 12725-12730
-
-
Burton, M.1
Nakai, H.2
Colosi, P.3
Cunningham, J.4
Mitchell, R.5
Couto, L.6
-
4
-
-
0034161486
-
Sustained expression of human factor VIII in mice using a parvovirus-based vector
-
Chao H., Mao L., Bruce A.T., and Walsh C.E. Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood 95 (2000) 1594-1599
-
(2000)
Blood
, vol.95
, pp. 1594-1599
-
-
Chao, H.1
Mao, L.2
Bruce, A.T.3
Walsh, C.E.4
-
5
-
-
0033541640
-
Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses
-
Clark K.R., Liu X., Mcgrath J.P., and Johnon P.R. Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum. Gene Ther. 10 (1999) 1031-1039
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 1031-1039
-
-
Clark, K.R.1
Liu, X.2
Mcgrath, J.P.3
Johnon, P.R.4
-
6
-
-
4644321610
-
Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock
-
Davdoff A.M., Ng C.Y., Sleep S., Gray J., Azam S., Zhao Y., Mcintosh J.H., Karimipoor M., and Nathwani A.C. Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock. J. Virol. Methods 121 (2004) 209-215
-
(2004)
J. Virol. Methods
, vol.121
, pp. 209-215
-
-
Davdoff, A.M.1
Ng, C.Y.2
Sleep, S.3
Gray, J.4
Azam, S.5
Zhao, Y.6
Mcintosh, J.H.7
Karimipoor, M.8
Nathwani, A.C.9
-
7
-
-
0035839562
-
Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation
-
During M.J., Kaplitt M.G., Stern M.B., and Eidelberg D. Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation. Hum. Gene Ther. 12 (2001) 1589-1591
-
(2001)
Hum. Gene Ther.
, vol.12
, pp. 1589-1591
-
-
During, M.J.1
Kaplitt, M.G.2
Stern, M.B.3
Eidelberg, D.4
-
8
-
-
0742307402
-
Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults
-
Flotte T.R., Bantly M.L., Spencer L.T., Byrne B.J., Spence C.T., Baker D.J., and Humphries M. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults. Hum. Gene Ther. 15 (2004) 93-128
-
(2004)
Hum. Gene Ther.
, vol.15
, pp. 93-128
-
-
Flotte, T.R.1
Bantly, M.L.2
Spencer, L.T.3
Byrne, B.J.4
Spence, C.T.5
Baker, D.J.6
Humphries, M.7
-
9
-
-
0034633742
-
Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo
-
Gao G., Qu G., Burnham M.S., Huang J., Chirmule N., Joshi B., Yu Q.C., Marsh J.A., Concicao C.M., and Wilson J.M. Purification of recombinant adeno-associated virus vectors by column chromatography and its performance in vivo. Hum. Gene Ther. 11 (2000) 2079-2091
-
(2000)
Hum. Gene Ther.
, vol.11
, pp. 2079-2091
-
-
Gao, G.1
Qu, G.2
Burnham, M.S.3
Huang, J.4
Chirmule, N.5
Joshi, B.6
Yu, Q.C.7
Marsh, J.A.8
Concicao, C.M.9
Wilson, J.M.10
-
10
-
-
0015847039
-
A new technique for assay of infectivity of human adenovirus 5 DNA
-
Graham F.L., and van der Eb A.J. A new technique for assay of infectivity of human adenovirus 5 DNA. Virology 52 (1973) 456-467
-
(1973)
Virology
, vol.52
, pp. 456-467
-
-
Graham, F.L.1
van der Eb, A.J.2
-
11
-
-
0032773889
-
Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2
-
Grimm D., Kern A., Pawlita M., Ferari F., Samulski R., and Klenschmidt J. Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2. Gene Ther. 6 (1999) 1322-1330
-
(1999)
Gene Ther.
, vol.6
, pp. 1322-1330
-
-
Grimm, D.1
Kern, A.2
Pawlita, M.3
Ferari, F.4
Samulski, R.5
Klenschmidt, J.6
-
12
-
-
0141482003
-
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy
-
Grimm D., Zhou S., Nakai H., Thomas C.E., Storm T.A., Fuess S., Matsushita T., Allen J., Surosky R., Lochrie M., Meuse L., McClelland A., Colosi P., and Kay M.A. Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 102 (2003) 2412-2419
-
(2003)
Blood
, vol.102
, pp. 2412-2419
-
-
Grimm, D.1
Zhou, S.2
Nakai, H.3
Thomas, C.E.4
Storm, T.A.5
Fuess, S.6
Matsushita, T.7
Allen, J.8
Surosky, R.9
Lochrie, M.10
Meuse, L.11
McClelland, A.12
Colosi, P.13
Kay, M.A.14
-
13
-
-
0030902985
-
Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
-
Herzog R.W., Hagstrom J.N., Kung S.H., Tai S.J., Wilson J.M., Fisher K.J., and High K.A. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. U.S.A. 94 (1997) 3809-5804
-
(1997)
Proc. Natl. Acad. Sci. U.S.A.
, vol.94
, pp. 3809-5804
-
-
Herzog, R.W.1
Hagstrom, J.N.2
Kung, S.H.3
Tai, S.J.4
Wilson, J.M.5
Fisher, K.J.6
High, K.A.7
-
14
-
-
0036019204
-
Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography
-
Kaludov N., Handelman B., and Chirini J.A. Scalable purification of adeno-associated virus type 2, 4, or 5 using ion-exchange chromatography. Hum. Gene Ther. 13 (2002) 1235-1243
-
(2002)
Hum. Gene Ther.
, vol.13
, pp. 1235-1243
-
-
Kaludov, N.1
Handelman, B.2
Chirini, J.A.3
-
15
-
-
0034050904
-
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
-
Kay M.A., Manno C.S., Ragni M.V., Larson P.J., Couto L.B., McClelland A., Glader B., Chew A.J., Tai S.J., Herzog R.W., Arruda V., Johnson F., Scallan C., Skarsgard E., Flake A.W., and High K.A. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat. Genet. 24 (2000) 257-261
-
(2000)
Nat. Genet.
, vol.24
, pp. 257-261
-
-
Kay, M.A.1
Manno, C.S.2
Ragni, M.V.3
Larson, P.J.4
Couto, L.B.5
McClelland, A.6
Glader, B.7
Chew, A.J.8
Tai, S.J.9
Herzog, R.W.10
Arruda, V.11
Johnson, F.12
Scallan, C.13
Skarsgard, E.14
Flake, A.W.15
High, K.A.16
-
16
-
-
0030451214
-
Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
-
Kessler P.D., Podsakoff G.M., Chen X., Mcquiston S.A., Colosi P.C., Matelis L.A., Kurtzman G.J., and Byrne B.J. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. U.S.A. 93 (1996) 14082-14087
-
(1996)
Proc. Natl. Acad. Sci. U.S.A.
, vol.93
, pp. 14082-14087
-
-
Kessler, P.D.1
Podsakoff, G.M.2
Chen, X.3
Mcquiston, S.A.4
Colosi, P.C.5
Matelis, L.A.6
Kurtzman, G.J.7
Byrne, B.J.8
-
17
-
-
0014967250
-
Kinetic study of the deamidation of growth hormone and prolactin
-
Lewis U.J., Cheever E.V., and Hopkins W.C. Kinetic study of the deamidation of growth hormone and prolactin. Biochim. Biophys. Acta 214 (1970) 498-508
-
(1970)
Biochim. Biophys. Acta
, vol.214
, pp. 498-508
-
-
Lewis, U.J.1
Cheever, E.V.2
Hopkins, W.C.3
-
18
-
-
0013442744
-
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
-
Manno C.S., Chew A.J., Hutchison S., Larson P.J., Herzog R.W., Arruda V.R., Tai S.J., Ragni M.V., Thompson A., Ozelo M., Cotto L.B., Leonard D.G., Johnson F.A., McClelland A., Scallan C., Skarsgard E., Flake A.W., Kay M.A., High K.A., and Glader B. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101 (2003) 2963-2972
-
(2003)
Blood
, vol.101
, pp. 2963-2972
-
-
Manno, C.S.1
Chew, A.J.2
Hutchison, S.3
Larson, P.J.4
Herzog, R.W.5
Arruda, V.R.6
Tai, S.J.7
Ragni, M.V.8
Thompson, A.9
Ozelo, M.10
Cotto, L.B.11
Leonard, D.G.12
Johnson, F.A.13
McClelland, A.14
Scallan, C.15
Skarsgard, E.16
Flake, A.W.17
Kay, M.A.18
High, K.A.19
Glader, B.20
more..
-
19
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations of imposed by the host immune response
-
Manno C.S., Arruda V.R., Pierce G.F., Ragni M., Rasko J., Ozelo M.C., Hoots K., Blatt P., Konkle B., Dake M., Kaye R., Razavi M., Zakjo A., Zehnder J., Nakai H., Chew A., Leonard D., Wright J.F., Lessard R.R., Sommer J.M., Tiggees M., Sabatono D., Luke A., Jiang H., Mingozzi F., Couto L., Ertl H.C., High K.A., and Kay M.A. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations of imposed by the host immune response. Nat. Med. 12 (2006) 342-347
-
(2006)
Nat. Med.
, vol.12
, pp. 342-347
-
-
Manno, C.S.1
Arruda, V.R.2
Pierce, G.F.3
Ragni, M.4
Rasko, J.5
Ozelo, M.C.6
Hoots, K.7
Blatt, P.8
Konkle, B.9
Dake, M.10
Kaye, R.11
Razavi, M.12
Zakjo, A.13
Zehnder, J.14
Nakai, H.15
Chew, A.16
Leonard, D.17
Wright, J.F.18
Lessard, R.R.19
Sommer, J.M.20
Tiggees, M.21
Sabatono, D.22
Luke, A.23
Jiang, H.24
Mingozzi, F.25
Couto, L.26
Ertl, H.C.27
High, K.A.28
Kay, M.A.29
more..
-
20
-
-
0031875874
-
Adeno-associated virus vectors can be efficiently produced without helper virus
-
Matsushita T., Elliger S., Elliger C., Podsakoff G., Villarreal L., Kurtzman G.J., Iwaki Y., and Colosi P. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5 (1998) 938-945
-
(1998)
Gene Ther.
, vol.5
, pp. 938-945
-
-
Matsushita, T.1
Elliger, S.2
Elliger, C.3
Podsakoff, G.4
Villarreal, L.5
Kurtzman, G.J.6
Iwaki, Y.7
Colosi, P.8
-
21
-
-
0031838721
-
Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver
-
Nakai H., Herzog R.W., Hagstrom J.N., Walter J., Kung S.H., Yang R.Y., Tai S.J., Iwaki Y., Kurtzman G.J., Fisher K.J., Colosi P., Couto L.B., and High K.A. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood 91 (1998) 4600-4607
-
(1998)
Blood
, vol.91
, pp. 4600-4607
-
-
Nakai, H.1
Herzog, R.W.2
Hagstrom, J.N.3
Walter, J.4
Kung, S.H.5
Yang, R.Y.6
Tai, S.J.7
Iwaki, Y.8
Kurtzman, G.J.9
Fisher, K.J.10
Colosi, P.11
Couto, L.B.12
High, K.A.13
-
22
-
-
0038618702
-
Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice
-
Passini M.A., Watson D.J., Vite C.H., Landsburg D.J., Feigenbaum A.L., and Wolfe J.H. Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice. J. Virol. 77 (2003) 7034-7040
-
(2003)
J. Virol.
, vol.77
, pp. 7034-7040
-
-
Passini, M.A.1
Watson, D.J.2
Vite, C.H.3
Landsburg, D.J.4
Feigenbaum, A.L.5
Wolfe, J.H.6
-
23
-
-
0036366386
-
Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors
-
Potter M., Chestnut K., Muzyczka N., Flotte T., and Zolotukhin S. Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors. Methods Enzymol. 346 (2002) 413-430
-
(2002)
Methods Enzymol.
, vol.346
, pp. 413-430
-
-
Potter, M.1
Chestnut, K.2
Muzyczka, N.3
Flotte, T.4
Zolotukhin, S.5
-
24
-
-
28444491760
-
Identification of mouse AAV capsid-specific CD8 + T cell epitopes
-
Sabatino D.E., Mingozzi F., Hui K.J., Chen H., Colosi P., Ertl H.C., and High K.A. Identification of mouse AAV capsid-specific CD8 + T cell epitopes. Mol. Ther. 12 (2005) 1023-1033
-
(2005)
Mol. Ther.
, vol.12
, pp. 1023-1033
-
-
Sabatino, D.E.1
Mingozzi, F.2
Hui, K.J.3
Chen, H.4
Colosi, P.5
Ertl, H.C.6
High, K.A.7
-
25
-
-
0242643501
-
Serum-free production and column purification of adeno-associated virus type 5
-
Smith R.H., Ding C., and Kotin R.M. Serum-free production and column purification of adeno-associated virus type 5. J. Virol. Methods 114 (2003) 115-124
-
(2003)
J. Virol. Methods
, vol.114
, pp. 115-124
-
-
Smith, R.H.1
Ding, C.2
Kotin, R.M.3
-
26
-
-
0036811528
-
Production of clinical-grade recombinant adeno-associated virus vectors
-
Snyder R.O., and Flotte T.R. Production of clinical-grade recombinant adeno-associated virus vectors. Curr. Opin. Biotechnol. 13 (2002) 418-423
-
(2002)
Curr. Opin. Biotechnol.
, vol.13
, pp. 418-423
-
-
Snyder, R.O.1
Flotte, T.R.2
-
27
-
-
0037223085
-
Quantification of adeno-associated virus particles and empty capsids by optical density measurement
-
Sommer J.M., Smith P.H., Parthasarathy S., Isaacs J., Vijay S., Kieran J., Powell S.K., McClelland A., and Wright J.F. Quantification of adeno-associated virus particles and empty capsids by optical density measurement. Mol. Ther. 7 (2003) 122-128
-
(2003)
Mol. Ther.
, vol.7
, pp. 122-128
-
-
Sommer, J.M.1
Smith, P.H.2
Parthasarathy, S.3
Isaacs, J.4
Vijay, S.5
Kieran, J.6
Powell, S.K.7
McClelland, A.8
Wright, J.F.9
-
28
-
-
0031906147
-
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
-
Summerford C., and Samulski R.J. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72 (1998) 1438-1445
-
(1998)
J. Virol.
, vol.72
, pp. 1438-1445
-
-
Summerford, C.1
Samulski, R.J.2
-
29
-
-
33645516194
-
Removal of empty particles from type 1 adeno-associated virus vector stocks by ion exchange chromatography potentiates transgene expression
-
Urabe M., Xin K.Q., Obara Y., Nakkura T., Mizukami H., Kume A., Okuda K., and Ozawa K. Removal of empty particles from type 1 adeno-associated virus vector stocks by ion exchange chromatography potentiates transgene expression. Mol. Ther. 13 (2006) S427
-
(2006)
Mol. Ther.
, vol.13
-
-
Urabe, M.1
Xin, K.Q.2
Obara, Y.3
Nakkura, T.4
Mizukami, H.5
Kume, A.6
Okuda, K.7
Ozawa, K.8
-
30
-
-
0036377851
-
A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies
-
Wagner J.A., Nepomuceno I.B., Messner A.H., Moran M.L., Batson E.P., Dimicell S., Brown B.W., Desch J.K., Norbash A.M., Conard C.K., Guggino W.B., Flotter T.R., Wine J.J., Carter B.J., Reynolds T.C., Moss R.B., and Gardner P. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. Hum. Gene Ther. 13 (2002) 1349-1359
-
(2002)
Hum. Gene Ther.
, vol.13
, pp. 1349-1359
-
-
Wagner, J.A.1
Nepomuceno, I.B.2
Messner, A.H.3
Moran, M.L.4
Batson, E.P.5
Dimicell, S.6
Brown, B.W.7
Desch, J.K.8
Norbash, A.M.9
Conard, C.K.10
Guggino, W.B.11
Flotter, T.R.12
Wine, J.J.13
Carter, B.J.14
Reynolds, T.C.15
Moss, R.B.16
Gardner, P.17
-
31
-
-
20844434555
-
Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation
-
Wright J.F., Le T., Prado J., Bahr-Davidson J., Smith P.H., Zhen Z., Sommer J.M., Pierce G.F., and Qu G. Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation. Mol. Ther. 12 (2005) 171-178
-
(2005)
Mol. Ther.
, vol.12
, pp. 171-178
-
-
Wright, J.F.1
Le, T.2
Prado, J.3
Bahr-Davidson, J.4
Smith, P.H.5
Zhen, Z.6
Sommer, J.M.7
Pierce, G.F.8
Qu, G.9
-
32
-
-
0035003230
-
A novel method for purification of recombinant adeno-associated virus vectors on a large scale
-
Wu X. A novel method for purification of recombinant adeno-associated virus vectors on a large scale. Chin. Sci. Bull. 46 (2001) 485-489
-
(2001)
Chin. Sci. Bull.
, vol.46
, pp. 485-489
-
-
Wu, X.1
-
33
-
-
0031834034
-
Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates
-
Zhou S., Murphy J.E., Escobedo J.A., and Dwarki V.J. Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates. Gene Ther. 5 (1998) 665-670
-
(1998)
Gene Ther.
, vol.5
, pp. 665-670
-
-
Zhou, S.1
Murphy, J.E.2
Escobedo, J.A.3
Dwarki, V.J.4
-
34
-
-
0036428801
-
Production and purification of serotypes 1, 2, and 5 recombinant adeno-associated viral vectors
-
Zolotukhin S., Potter M., Zolotukhin I., Sakai Y., Loiler S., Fraites T.J.J.R., Chiodo V.A., Philpsberg T., Muzyczka N., Hausirth W.W., Flotter T.R., Byrne B.J., and Snyder R.O. Production and purification of serotypes 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 28 (2002) 58-67
-
(2002)
Methods
, vol.28
, pp. 58-67
-
-
Zolotukhin, S.1
Potter, M.2
Zolotukhin, I.3
Sakai, Y.4
Loiler, S.5
Fraites, T.J.J.R.6
Chiodo, V.A.7
Philpsberg, T.8
Muzyczka, N.9
Hausirth, W.W.10
Flotter, T.R.11
Byrne, B.J.12
Snyder, R.O.13
|