-
1
-
-
84859440283
-
The AAV vector toolkit: Poised at the clinical crossroads
-
Asokan A, Schaffer DV, Samulski RJ. 2012. The AAV vector toolkit: Poised at the clinical crossroads. Mol Ther 20:699-708.
-
(2012)
Mol Ther
, vol.20
, pp. 699-708
-
-
Asokan, A.1
Schaffer, D.V.2
Samulski, R.J.3
-
2
-
-
84862599159
-
Enhancing the clinical potential of AAV vectors by capsid engineering to evade preexisting immunity
-
Bartel M, Schaffer D, Buning H. 2011. Enhancing the clinical potential of AAV vectors by capsid engineering to evade preexisting immunity. Front Microbiol 2:204.
-
(2011)
Front Microbiol
, vol.2
, pp. 204
-
-
Bartel, M.1
Schaffer, D.2
Buning, H.3
-
3
-
-
77954976233
-
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: Implications for gene therapy using AAV vectors
-
Boutin S, Monteilhet V, Veron P, Leborgne C, Benveniste O, Montus MF, Masurier C. 2010. Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: Implications for gene therapy using AAV vectors. Hum Gene Ther 21: 704-712.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 704-712
-
-
Boutin, S.1
Monteilhet, V.2
Veron, P.3
Leborgne, C.4
Benveniste, O.5
Montus, M.F.6
Masurier, C.7
-
4
-
-
58849086758
-
Worldwide epidemiology of neutralizing antibodies to adenoassociated viruses
-
Calcedo R, Vandenberghe LH, Gao G, Lin J, Wilson JM. 2009. Worldwide epidemiology of neutralizing antibodies to adenoassociated viruses. J Infect Dis 199:381-390.
-
(2009)
J Infect Dis
, vol.199
, pp. 381-390
-
-
Calcedo, R.1
Vandenberghe, L.H.2
Gao, G.3
Lin, J.4
Wilson, J.M.5
-
5
-
-
84864471392
-
Changing pattern of hepatitis a virus epidemiology in an area of high endemicity
-
Campagna M, Siddu A, Meloni A, Basciu C, Ferrai L, Pettinau A, Cardia C, Masia G, Coppola RC. 2012. Changing pattern of hepatitis a virus epidemiology in an area of high endemicity. Hepat Mon 12:382-385.
-
(2012)
Hepat Mon
, vol.12
, pp. 382-385
-
-
Campagna, M.1
Siddu, A.2
Meloni, A.3
Basciu, C.4
Ferrai, L.5
Pettinau, A.6
Cardia, C.7
Masia, G.8
Coppola, R.C.9
-
6
-
-
79955596992
-
A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates
-
Chandler RJ, Venditti CP. 2011. A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates. Mol Ther 19:826-827.
-
(2011)
Mol Ther
, vol.19
, pp. 826-827
-
-
Chandler, R.J.1
Venditti, C.P.2
-
8
-
-
0345411588
-
Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV)
-
Erles K, Sebokova P, Schlehofer JR. 1999. Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV). J Med Virol 59:406-411.
-
(1999)
J Med Virol
, vol.59
, pp. 406-411
-
-
Erles, K.1
Sebokova, P.2
Schlehofer, J.R.3
-
9
-
-
84864343619
-
Genetic therapeutic approaches for Duchenne muscular dystrophy
-
Foster H, Popplewell L, Dickson G. 2012. Genetic therapeutic approaches for Duchenne muscular dystrophy. Hum Gene Ther 23:676-687.
-
(2012)
Hum Gene Ther
, vol.23
, pp. 676-687
-
-
Foster, H.1
Popplewell, L.2
Dickson, G.3
-
10
-
-
2642512201
-
Clades of adeno-associated viruses are widely disseminated in human tissues
-
Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, Wilson JM. 2004. Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol 78:6381-6388.
-
(2004)
J Virol
, vol.78
, pp. 6381-6388
-
-
Gao, G.1
Vandenberghe, L.H.2
Alvira, M.R.3
Lu, Y.4
Calcedo, R.5
Zhou, X.6
Wilson, J.M.7
-
11
-
-
70349110562
-
Adenoassociated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses
-
Gao G, Wang Q, Calcedo R, Mays L, Bell P, Wang L, Vandenberghe LH, Grant R, Sanmiguel J, Furth EE, Wilson JM. 2009. Adenoassociated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther 20:930-942.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 930-942
-
-
Gao, G.1
Wang, Q.2
Calcedo, R.3
Mays, L.4
Bell, P.5
Wang, L.6
Vandenberghe, L.H.7
Grant, R.8
Sanmiguel, J.9
Furth, E.E.10
Wilson, J.M.11
-
12
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. 2002. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99:11854-11859.
-
(2002)
Proc Natl Acad Sci USA
, vol.99
, pp. 11854-11859
-
-
Gao, G.P.1
Alvira, M.R.2
Wang, L.3
Calcedo, R.4
Johnston, J.5
Wilson, J.M.6
-
13
-
-
84857602982
-
Adeno-associated virus vectorology, manufacturing, and clinical applications
-
Grieger JC, Samulski RJ. 2012. Adeno-associated virus vectorology, manufacturing, and clinical applications. Methods Enzymol 507:229-254.
-
(2012)
Methods Enzymol
, vol.507
, pp. 229-254
-
-
Grieger, J.C.1
Samulski, R.J.2
-
14
-
-
44049098137
-
Update on progress and hurdles in novel genetic therapies for hemophilia
-
High, K. 2007. Update on progress and hurdles in novel genetic therapies for hemophilia. Hematology Am Soc Hematol Educ Program 2007:466-472.
-
(2007)
Hematology Am Soc Hematol Educ Program
, vol.2007
, pp. 466-472
-
-
High, K.1
-
16
-
-
33751181882
-
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
-
Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Mingozzi F, High KA, Pierce GF. 2006. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 108:3321-3328.
-
(2006)
Blood
, vol.108
, pp. 3321-3328
-
-
Jiang, H.1
Couto, L.B.2
Patarroyo-White, S.3
Liu, T.4
Nagy, D.5
Vargas, J.A.6
Zhou, S.7
Scallan, C.D.8
Sommer, J.9
Vijay, S.10
Mingozzi, F.11
High, K.A.12
Pierce, G.F.13
-
17
-
-
0034050904
-
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
-
Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA. 2000. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24:257-261.
-
(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
-
18
-
-
67449093069
-
Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo
-
Li C, Hirsch M, DiPrimio N, Asokan A, Goudy K, Tisch R, Samulski RJ. 2009. Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo. J Virol 83:6817-6824.
-
(2009)
J Virol
, vol.83
, pp. 6817-6824
-
-
Li, C.1
Hirsch, M.2
DiPrimio, N.3
Asokan, A.4
Goudy, K.5
Tisch, R.6
Samulski, R.J.7
-
19
-
-
84858005636
-
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
-
Li C, Narkbunnam N, Samulski RJ, Asokan A, Hu G, Jacobson LJ, Manco-Johnson MJ, Monahan PE. 2012. Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. Gene Ther 19:288-294.
-
(2012)
Gene Ther
, vol.19
, pp. 288-294
-
-
Li, C.1
Narkbunnam, N.2
Samulski, R.J.3
Asokan, A.4
Hu, G.5
Jacobson, L.J.6
Manco-Johnson, M.J.7
Monahan, P.E.8
-
20
-
-
0013442744
-
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
-
Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B. 2003. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101:2963-2972.
-
(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
Couto, 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..
-
21
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
-
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. 2006. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12:342-347.
-
(2006)
Nat Med
, vol.12
, pp. 342-347
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
Glader, B.4
Ragni, M.5
Rasko, J.J.6
Ozelo, M.C.7
Hoots, K.8
Blatt, P.9
Konkle, B.10
Dake, M.11
Kaye, R.12
Razavi, M.13
Zajko, A.14
Zehnder, J.15
Rustagi, P.K.16
Nakai, H.17
Chew, A.18
Leonard, D.19
Wright, J.F.20
Lessard, R.R.21
Sommer, J.M.22
Tigges, M.23
Sabatino, D.24
Luk, A.25
Jiang, H.26
Mingozzi, F.27
Couto, L.28
Ertl, H.C.29
High, K.A.30
Kay, M.A.31
more..
-
22
-
-
84873337445
-
Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors
-
Mimuro J, Mizukami H, Hishikawa S, Ikemoto T, Ishiwata A, Sakata A, Ohmori T, Madoiwa S, Ono F, Ozawa K, Sakata Y. 2013. Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors. Mol Ther 21:318-323.
-
(2013)
Mol Ther
, vol.21
, pp. 318-323
-
-
Mimuro, J.1
Mizukami, H.2
Hishikawa, S.3
Ikemoto, T.4
Ishiwata, A.5
Sakata, A.6
Ohmori, T.7
Madoiwa, S.8
Ono, F.9
Ozawa, K.10
Sakata, Y.11
-
23
-
-
79959932534
-
Immune responses to AAV in clinical trials
-
Mingozzi F, High KA. 2011. Immune responses to AAV in clinical trials. Curr Gene Ther 11:321-330.
-
(2011)
Curr Gene Ther
, vol.11
, pp. 321-330
-
-
Mingozzi, F.1
High, K.A.2
-
24
-
-
34147098413
-
CD8(+) T-cell responses to adeno-associated virus capsid in humans
-
Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertl HC, High KA. 2007. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 13:419-422.
-
(2007)
Nat Med
, vol.13
, pp. 419-422
-
-
Mingozzi, F.1
Maus, M.V.2
Hui, D.J.3
Sabatino, D.E.4
Murphy, S.L.5
Rasko, J.E.6
Ragni, M.V.7
Manno, C.S.8
Sommer, J.9
Jiang, H.10
Pierce, G.F.11
Ertl, H.C.12
High, K.A.13
-
25
-
-
69949160511
-
AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
-
Mingozzi F, Meulenberg JJ, Hui DJ, Basner-Tschakarjan E, Hasbrouck NC, Edmonson SA, Hutnick NA, Betts MR, Kastelein JJ, Stroes ES, High KA. 2009. AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood 114:2077-2086.
-
(2009)
Blood
, vol.114
, pp. 2077-2086
-
-
Mingozzi, F.1
Meulenberg, J.J.2
Hui, D.J.3
Basner-Tschakarjan, E.4
Hasbrouck, N.C.5
Edmonson, S.A.6
Hutnick, N.A.7
Betts, M.R.8
Kastelein, J.J.9
Stroes, E.S.10
High, K.A.11
-
26
-
-
33750611942
-
Adipose tissue as a novel target for in vivo gene transfer by adeno-associated viral vectors
-
Mizukami H, Mimuro J, Ogura T, Okada T, Urabe M, Kume A, Sakata Y, Ozawa K. 2006. Adipose tissue as a novel target for in vivo gene transfer by adeno-associated viral vectors. Hum Gene Ther 17:921-928.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 921-928
-
-
Mizukami, H.1
Mimuro, J.2
Ogura, T.3
Okada, T.4
Urabe, M.5
Kume, A.6
Sakata, Y.7
Ozawa, K.8
-
27
-
-
3042527542
-
Expression of the adenovirus E4 34k oncoprotein inhibits repair of double strand breaks in the cellular genome of a 293-based inducible cell line
-
Mohammadi ES, Ketner EA, Johns DC, Ketner G. 2004. Expression of the adenovirus E4 34k oncoprotein inhibits repair of double strand breaks in the cellular genome of a 293-based inducible cell line. Nucleic Acids Res 32:2652-2659.
-
(2004)
Nucleic Acids Res
, vol.32
, pp. 2652-2659
-
-
Mohammadi, E.S.1
Ketner, E.A.2
Johns, D.C.3
Ketner, G.4
-
28
-
-
77956262279
-
A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease
-
Muramatsu S, Fujimoto K, Kato S, Mizukami H, Asari S, Ikeguchi K, Kawakami T, Urabe M, Kume A, Sato T, Watanabe E, Ozawa K, Nakano I. 2010. A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol Ther 18:1731-1735.
-
(2010)
Mol Ther
, vol.18
, pp. 1731-1735
-
-
Muramatsu, S.1
Fujimoto, K.2
Kato, S.3
Mizukami, H.4
Asari, S.5
Ikeguchi, K.6
Kawakami, T.7
Urabe, M.8
Kume, A.9
Sato, T.10
Watanabe, E.11
Ozawa, K.12
Nakano, I.13
-
29
-
-
33645528206
-
Self-complementary adenoassociated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
-
Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, DavidoffAM. 2006. Self-complementary adenoassociated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 107:2653-2661.
-
(2006)
Blood
, vol.107
, pp. 2653-2661
-
-
Nathwani, A.C.1
Gray, J.T.2
Ng, C.Y.3
Zhou, J.4
Spence, Y.5
Waddington, S.N.6
Tuddenham, E.G.7
Kemball-Cook, G.8
McIntosh, J.9
Boon-Spijker, M.10
Mertens, K.11
Davidoff, A.M.12
-
30
-
-
79955597265
-
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins
-
Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, DavidoffAM. 2011a. Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. Mol Ther 19:876-885.
-
(2011)
Mol Ther
, vol.19
, pp. 876-885
-
-
Nathwani, A.C.1
Rosales, C.2
McIntosh, J.3
Rastegarlari, G.4
Nathwani, D.5
Raj, D.6
Nawathe, S.7
Waddington, S.N.8
Bronson, R.9
Jackson, S.10
Donahue, R.E.11
High, K.A.12
Mingozzi, F.13
Ng, C.Y.14
Zhou, J.15
Spence, Y.16
McCarville, M.B.17
Valentine, M.18
Allay, J.19
Coleman, J.20
Sleep, S.21
Gray, J.T.22
Nienhuis, A.W.23
Davidoff, A.M.24
more..
-
31
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, DavidoffAM. 2011b. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 365:2357-2365.
-
(2011)
N Engl J Med
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
Rosales, C.4
McIntosh, J.5
Linch, D.C.6
Chowdary, P.7
Riddell, A.8
Pie, A.J.9
Harrington, C.10
O'Beirne, J.11
Smith, K.12
Pasi, J.13
Glader, B.14
Rustagi, P.15
Ng, C.Y.16
Kay, M.A.17
Zhou, J.18
Spence, Y.19
Morton, C.L.20
Allay, J.21
Coleman, J.22
Sleep, S.23
Cunningham, J.M.24
Srivastava, D.25
Basner-Tschakarjan, E.26
Mingozzi, F.27
High, K.A.28
Gray, J.T.29
Reiss, U.M.30
Nienhuis, A.W.31
Davidoff, A.M.32
more..
-
32
-
-
84856958590
-
Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models
-
Rapti K, Louis-Jeune V, Kohlbrenner E, Ishikawa K, Ladage D, Zolotukhin S, Hajjar RJ, Weber T. 2012. Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models. Mol Ther 20:73-83.
-
(2012)
Mol Ther
, vol.20
, pp. 73-83
-
-
Rapti, K.1
Louis-Jeune, V.2
Kohlbrenner, E.3
Ishikawa, K.4
Ladage, D.5
Zolotukhin, S.6
Hajjar, R.J.7
Weber, T.8
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