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0032549488
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of special interest. A comparison of rAAV production techniques on a large scale. With direct comparisons of particle number and infectious particle number as well as levels of wild type AAV contamination.
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Salvetti A, Oreve S, Chadeuf G, Favre D, Cherel Y, Champion Arnaud P, David-Ameline J, Moullier P. Factors influencing recombinant adeno-associated virus production. of special interest Hum Gene Ther. 9:1998;695-706 A comparison of rAAV production techniques on a large scale. With direct comparisons of particle number and infectious particle number as well as levels of wild type AAV contamination.
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David-Ameline, J.7
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13
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0031926094
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Wang XS, Srivastava A. Rescue and autonomous replication of adeno-associated virus type 2 genomes containing Rep-binding site mutations in the viral p5 promoter. J Virol. 72:1998;4811-4818.
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Wang, X.S.1
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0031019819
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Koeberl DD, Alexander IE, Halbert CL, Russell DW, Miller AD. Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Natl Acad Sci USA. 94:1997;1426-1431.
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15
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0031014018
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The adeno-associated virus (AAV) rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection
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of special interest. This paper begins to describe the complex interaction between the replication proteins of AAV and their promoters during adenovirus infection.
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Pereira DJ, McCarty DM, Muzyczka N. The adeno-associated virus (AAV) rep protein acts as both a repressor and an activator to regulate AAV transcription during a productive infection. of special interest J Virol. 71:1997;1079-1088 This paper begins to describe the complex interaction between the replication proteins of AAV and their promoters during adenovirus infection.
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Pereira, D.J.1
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The cellular transcription factor SP1 and an unknown cellular protein are required to mediate Rep protein activation of the adeno-associated virus p19 promoter
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Pereira DJ, Muzyczka N. The cellular transcription factor SP1 and an unknown cellular protein are required to mediate Rep protein activation of the adeno-associated virus p19 promoter. J Virol. 71:1997;1747-1756.
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Pereira, D.J.1
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17
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18
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of outstanding interest. One of the disadvantages in the production of rAAV is the contaminating adenovirus by-product. This paper brings together new production techniques that result in high titer adenovirus free rAAV
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Xiao X, Li J, Samulski RJ. Production of high-liter recombinant adeno-associated virus vectors in the absence of helper adenovirus. of outstanding interest J Virol. 72:1998;2224-2232 One of the disadvantages in the production of rAAV is the contaminating adenovirus by-product. This paper brings together new production techniques that result in high titer adenovirus free rAAV.
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Xiao, X.1
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of special interest. The authors describe the relationship between helper virus, specific rep proteins, and rep binding element in the terminal repeats on production of capsid proteins.
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Weger S, Wistuba A, Grimm D, Kleinschmidt JA. Control of adeno associated virus type 2 cap gene expression: relative influence of helper virus, terminal repeats, and rep proteins. of special interest J Virol. 71:1997;8437-8447 The authors describe the relationship between helper virus, specific rep proteins, and rep binding element in the terminal repeats on production of capsid proteins.
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Weger, S.1
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21
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Flotte TR, Barraza-Ortiz X, Solow R, Afione SA, Carter BJ, Guggino WB. An improved system for packaging recombinant adeno-associated virus vectors capable of in vivo transduction. Gene Ther. 2:1995;29-37.
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Guggino, W.B.6
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22
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Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products
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of special interest. Rous sarcoma virus long terminal repeat and CMV immediate early promoters were used in place of p5 and p40, respectively. The authors showed that increased synthesis of capsid proteins lead to an increase in rAAV titers, and that increasing rep expression decreased rAAV yeilds and capsid synthesis.
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Vincent KA, Piraino ST, Wadsworth SC. Analysis of recombinant adeno-associated virus packaging and requirements for rep and cap gene products. of special interest J Virol. 71:1997;1897-1905 Rous sarcoma virus long terminal repeat and CMV immediate early promoters were used in place of p5 and p40, respectively. The authors showed that increased synthesis of capsid proteins lead to an increase in rAAV titers, and that increasing rep expression decreased rAAV yeilds and capsid synthesis.
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J Virol
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Vincent, K.A.1
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Wadsworth, S.C.3
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Role for highly regulated rep gene expression in adeno-associated virus vector production
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of special interest. Exogenous promoters were used to increase expression of rep genes in order to determine the effect of rep on rAAV production. The result of increased rep expression was a decrease in both capsid and rAAV production. In an effort to reduce rep expression levels, a mutation in the start codon AUG to ACG was made and this resulted in higher levels of both capsid and rAAV production; further demonstrating the importance of rep regulation for rAAV production.
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Li J, Samulski RJ, Xiao X. Role for highly regulated rep gene expression in adeno-associated virus vector production. of special interest J Virol. 71:1997;5236-5243 Exogenous promoters were used to increase expression of rep genes in order to determine the effect of rep on rAAV production. The result of increased rep expression was a decrease in both capsid and rAAV production. In an effort to reduce rep expression levels, a mutation in the start codon AUG to ACG was made and this resulted in higher levels of both capsid and rAAV production; further demonstrating the importance of rep regulation for rAAV production.
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J Virol
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Li, J.1
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24
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Monahan PE, Samulski RJ, Tazelaar J, Nichols TC, Bellinger DA, Read MS, Walsh CE. Direct intramuscular infection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia. Gene Ther. 5:1998;40-49.
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Tazelaar, J.3
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Read, M.S.6
Walsh, C.E.7
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25
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0030827207
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Conway JE, Zolotukhin S, Muzyczka N, Hayward GS, Byrne BJ. Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex virus type 1 amplicon expression rep and cap. J Virol. 71:1997;8780-8789.
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0031278324
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of special interest. The authors showed for the first time that rAAV can be produced using adenovirus vectors as the source of adenovirus helper function.
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Ferrari FK, Xiao X, McCarty D, Samulski RJ. New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. of special interest Nat Med. 3:1997;1295-1297 The authors showed for the first time that rAAV can be produced using adenovirus vectors as the source of adenovirus helper function.
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Ferrari, F.K.1
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Cell lines inducibly expressing the adeno-associated virus (AAV) rep gene: Requirements for productive replication of rep-negative AAV mutants
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Holscher C, Horer M, Kleinschmidt JA, Zentgraf H, Burkle A, Heilbronn R. Cell lines inducibly expressing the adeno-associated virus (AAV) rep gene: requirements for productive replication of rep-negative AAV mutants. J Virol. 68:1994;7169-7177.
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Burkle, A.5
Heilbronn, R.6
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30
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0029873712
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A new strategy for large-scale preparation of high-titer recombinant adeno-associated virus vectors by using packaging cell lines and sulfonated cellulose column chromotography
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Tamayose K, Hirai Y, Shimada T. A new strategy for large-scale preparation of high-titer recombinant adeno-associated virus vectors by using packaging cell lines and sulfonated cellulose column chromotography. Hum Gene Ther. 7:1996;507-513.
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31
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of special interest. Potentially the safest production approach will be in vitro. The authors demonstrate for the first time the feasability of this approach.
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Ding L, Lu S, Munshi NC. In vitro packaging of an infectious recombinant adeno-associated virus 2. of special interest Gen Ther. 4:1997;1167-1172 Potentially the safest production approach will be in vitro. The authors demonstrate for the first time the feasability of this approach.
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Gen Ther
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Ding, L.1
Lu, S.2
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32
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In vitro packaging of adeno-associated virus DNA
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of special interest. A detailed analysis of the substrates required for small scale in vitro packaging of rAAV, and the different products resulting from in vitro assembly with regards to packaging size and chloroform sensitivity.
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Zhou Z, Muzyczka N. In vitro packaging of adeno-associated virus DNA. of special interest J Virol. 72:1998;3241-3247 A detailed analysis of the substrates required for small scale in vitro packaging of rAAV, and the different products resulting from in vitro assembly with regards to packaging size and chloroform sensitivity.
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0031011987
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of outstanding interest. This paper asks the question of bio-distribution of rAAV2 in mice. After tail-vein injection, PCR amplification was used to detect the presence of rAAV in various tissues at time points ranging from one hour to one week. Although the majority of the virus ends up in the liver, detectable amounts are found in other organs, including-bone marrow, heart, kidney, and lung.
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Ponnazhagan S, Mukherjee P, Yoder MC, Wang XS, Zhou SZ, Kaplan J, Wadsworth S, Srivastava A. Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice. of outstanding interest Gene. 190:1997;203-210 This paper asks the question of bio-distribution of rAAV2 in mice. After tail-vein injection, PCR amplification was used to detect the presence of rAAV in various tissues at time points ranging from one hour to one week. Although the majority of the virus ends up in the liver, detectable amounts are found in other organs, including-bone marrow, heart, kidney, and lung.
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Ponnazhagan, S.1
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Wadsworth, S.7
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Ali RR, Reichel MB, Thrasher AJ, Levinsky RJ, Kinnon C, Kanuga N, Hunt DM, Bhattacharya SS. Gene transfer into the mouse retina mediated by an adeno-associated viral vectors. Hum Mol Gene. 5:1996;591-594.
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Ali RR, Reichel MB, De Alwis M, Kanuga N, Kinnon C, Levinsky RJ, Hunt DM, Bhattacharya SS, Thrasher AJ. Adeno-associated virus gene transfer to mouse retina. Hum Gene Ther. 9:1998;81-86.
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Kanuga, N.4
Kinnon, C.5
Levinsky, R.J.6
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Pfaff, D.W.5
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Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
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Snyder RO, Miao CH, Patijn GA, Spratt SK, Danos O, Nagy D, Gown AM, Winther B, Meuse L, Cohen LK, et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Gene. 16:1997;270-276.
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Rolling F, Nong Z, Pisvin S, Collen D. Adeno-associated virus-mediated gene transfer into rat carotid arteries. Gene Ther. 4:1997;757-761.
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Collen, D.4
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43
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Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
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Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA. 93:1996;14082-14087.
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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
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44
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0031885546
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Long-term in vivo cochlear transgene expression mediated by recombinant adeno-associated virus
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Lalwani A, Walsh B, Reilly P, Carvalho G, Zolotukhin S, Muzyczka N, Mhatre A. Long-term in vivo cochlear transgene expression mediated by recombinant adeno-associated virus. Gene Ther. 5:1998;277-281.
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Lalwani, A.1
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Zolotukhin, S.5
Muzyczka, N.6
Mhatre, A.7
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45
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CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector
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Rubenstein RC, McVeigh U, Flotte TR, Guggino WB, Zeitlin PL. CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector. Gene Ther. 4:1997;384-392.
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Rubenstein, R.C.1
McVeigh, U.2
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Zeitlin, P.L.5
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46
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0030540970
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Long-term gene transfer in porcine myocardium after coronary infusion of an adeno-associated virus vector
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Kaplett MG, Xiao X, Samulski RJ, Li J, Ojamaa K, Klein IL, Makimura H, Kaplitt MJ, Strumpf RK, Diethrich EB. Long-term gene transfer in porcine myocardium after coronary infusion of an adeno-associated virus vector. Ann Thorac Surg. 62:1996;1669-1676.
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Kaplett, M.G.1
Xiao, X.2
Samulski, R.J.3
Li, J.4
Ojamaa, K.5
Klein, I.L.6
Makimura, H.7
Kaplitt, M.J.8
Strumpf, R.K.9
Diethrich, E.B.10
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47
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0031058024
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Recombinant adeno-associated virus for muscle directed gene therapy
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Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K, Pathak R, Raper SE, Wilson JM. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med. 3:1997;306-312.
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Fisher, K.J.1
Jooss, K.2
Alston, J.3
Yang, Y.4
Haecker, S.E.5
High, K.6
Pathak, R.7
Raper, S.E.8
Wilson, J.M.9
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48
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In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector
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During MJ, Samulski RJ, Elsworth JD, Kaplitt MG, Leone P, Xiao X, Li J, Freese A, Taylor JR, Roth RH, et al. In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector. Gene Ther. 5:1998;820-827.
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During, M.J.1
Samulski, R.J.2
Elsworth, J.D.3
Kaplitt, M.G.4
Leone, P.5
Xiao, X.6
Li, J.7
Freese, A.8
Taylor, J.R.9
Roth, R.H.10
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49
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Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates
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of special interest. The authors describe the long-term theraputic expression of monkey erythropoietin from rAAV after single intramuscular injection in baboons. This study demonstrates that rAAV can be used in a large animal model.
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Zhou S, Murphy JE, Escobedo JA, Dwarki VJ. Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates. of special interest Gene Ther. 5:1998;665-670 The authors describe the long-term theraputic expression of monkey erythropoietin from rAAV after single intramuscular injection in baboons. This study demonstrates that rAAV can be used in a large animal model.
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Gene Ther
, vol.5
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Zhou, S.1
Murphy, J.E.2
Escobedo, J.A.3
Dwarki, V.J.4
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50
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Gene transfer by adeno-associated virus vectors into the central nervous system
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Xiao X, Li J, McCown TJ, Samulski RJ. Gene transfer by adeno-associated virus vectors into the central nervous system. Exp Neurol. 144:1997;113-124.
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Xiao, X.1
Li, J.2
McCown, T.J.3
Samulski, R.J.4
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51
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0029883821
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Effects of gamma irradiation on the transduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors
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Alexander IE, Russell DW, Spence AM, Miller AD. Effects of gamma irradiation on the transduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors. Hum Gen Ther. 7:1996;841-850.
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Alexander, I.E.1
Russell, D.W.2
Spence, A.M.3
Miller, A.D.4
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52
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0032550793
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Selective and rapid uptake of adeno-associated virus type 2 in brain
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Bartlett JS, Samulski RJ, McCown TJ. Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum Gene Ther. 9:1998;1181-1186.
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Hum Gene Ther
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Bartlett, J.S.1
Samulski, R.J.2
McCown, T.J.3
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53
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0031014887
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Efficient transfer of green fluorescent proteins in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters
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Peel AL, Zolotukhin S, Schrimsher GW, Muzyczka N, Reir PJ. Efficient transfer of green fluorescent proteins in spinal cord neurons using adeno-associated virus vectors containing cell type-specific promoters. Gene Ther. 4:1997;16-24.
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Peel, A.L.1
Zolotukhin, S.2
Schrimsher, G.W.3
Muzyczka, N.4
Reir, P.J.5
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54
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0030902462
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Adeno-associated virus (AAV) vectors antisense gene transfer in vivo decreases GABA(A) alpha 1 containing receptors and increases inferior collicular seizure sensitivity
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Xiao X, McCown TJ, Li J, Breese GR, Morrow AL, Samulski RJ. Adeno-associated virus (AAV) vectors antisense gene transfer in vivo decreases GABA(A) alpha 1 containing receptors and increases inferior collicular seizure sensitivity. Brain Res. 756:1997;76-83.
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Xiao, X.1
McCown, T.J.2
Li, J.3
Breese, G.R.4
Morrow, A.L.5
Samulski, R.J.6
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55
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0031456051
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Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats
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Mandel RJ, Spratt SK, Snyder RO, Leff SE. Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc Natl Acad Sci USA. 94:1997;14083-14088.
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Mandel, R.J.1
Spratt, S.K.2
Snyder, R.O.3
Leff, S.E.4
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56
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0032101756
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Characterization of intrastriatal recombinant adeno-associated virus mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrogylase I in a rat model of Parkinson's disease
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Mandel RJ, Rendahl K, Spratt S, Snyder R, Cohen L, Leff S. Characterization of intrastriatal recombinant adeno-associated virus mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrogylase I in a rat model of Parkinson's disease. J Neurosci. 18:1998;4271-4284.
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Mandel, R.J.1
Rendahl, K.2
Spratt, S.3
Snyder, R.4
Cohen, L.5
Leff, S.6
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57
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0031969428
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Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers
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of outstanding interest. This is an excellent paper describing the immunologic state in response to rAAVlacZ transduction in muscle cells. It asks the question why rAAV fails to elicit an immune response when other vector systems do. Adoptive transfer experiments clearly indicate the antigen-presenting cells activated by adenovirus lacZ will eliminate lacZ expressing muscle cells previously infected with rAAVlacZ.
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Jooss K, Yang Y, Fisher KJ, Wilson JM. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. of outstanding interest J Virol. 72:1998;4212-4223 This is an excellent paper describing the immunologic state in response to rAAVlacZ transduction in muscle cells. It asks the question why rAAV fails to elicit an immune response when other vector systems do. Adoptive transfer experiments clearly indicate the antigen-presenting cells activated by adenovirus lacZ will eliminate lacZ expressing muscle cells previously infected with rAAVlacZ.
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(1998)
J Virol
, vol.72
, pp. 4212-4223
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Jooss, K.1
Yang, Y.2
Fisher, K.J.3
Wilson, J.M.4
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58
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0030902985
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Stable gene transfer and expression of human coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
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Herzog RW, Hagstrom JN, Kung S-H, Tai SJ, Wilson JM, Fisher KJ, High KA. Stable gene transfer and expression of human coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci USA. 94:1997;5804-5809.
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Proc Natl Acad Sci USA
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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
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59
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0030970938
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Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle
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Clark KR, Sferra TJ, Johnson PR. Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle. Hum Gene Ther. 8:1997;659-669.
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Hum Gene Ther
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Clark, K.R.1
Sferra, T.J.2
Johnson, P.R.3
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60
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0030671462
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Efficient and stable adeno-associated virus-mediated tranduction in the skeletal muscle of adult immunocompetent mice
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Snyder RO, Spratt SK, Lagarde C, Bohl D, Kaspar B, Sloan B, Cohen LK, Danos O. Efficient and stable adeno-associated virus-mediated tranduction in the skeletal muscle of adult immunocompetent mice. Hum Gene Ther. 8:1997;1891-1900.
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Hum Gene Ther
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Snyder, R.O.1
Spratt, S.K.2
Lagarde, C.3
Bohl, D.4
Kaspar, B.5
Sloan, B.6
Cohen, L.K.7
Danos, O.8
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61
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0031058024
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Recombinant adeno-associated virus for muscle directed gene therapy
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Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K, Pathak R, Raper SE, Wilson JM. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med. 3:1997;306-312.
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(1997)
Nat Med
, vol.3
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Fisher, K.J.1
Jooss, K.2
Alston, J.3
Yang, Y.4
Haecker, S.E.5
High, K.6
Pathak, R.7
Raper, S.E.8
Wilson, J.M.9
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62
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0031471130
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Long-term correction of obesity and dabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin
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of outstanding interest. This paper illustrates the potential of rAAV carrying a theraputic for the correction of leptin deficiency in mice. Long-term expression of the leptin gene over a period of 180 days with a range of serum levels between 50-125% of normal. Treated mice showed decreases in food intact and weight, as well as normal glucose levels.
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Murphy JE, Zhou S, Giese K, Williams LT, Escobedo JA, Dwarki VJ. Long-term correction of obesity and dabetes in genetically obese mice by a single intramuscular injection of recombinant adeno-associated virus encoding mouse leptin. of outstanding interest Proc Natl Acad Sci USA. 94:1997;13921-13926 This paper illustrates the potential of rAAV carrying a theraputic for the correction of leptin deficiency in mice. Long-term expression of the leptin gene over a period of 180 days with a range of serum levels between 50-125% of normal. Treated mice showed decreases in food intact and weight, as well as normal glucose levels.
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(1997)
Proc Natl Acad Sci USA
, vol.94
, pp. 13921-13926
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Murphy, J.E.1
Zhou, S.2
Giese, K.3
Williams, L.T.4
Escobedo, J.A.5
Dwarki, V.J.6
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63
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Correction of obesity and diabetes in genetically obese mice by leptin gene therapy
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Muzzin P, Eisensmith RC, Copeland KC, Woo SL. Correction of obesity and diabetes in genetically obese mice by leptin gene therapy. Proc Natl Acad Sci USA. 93:1996;14804-14808.
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Proc Natl Acad Sci USA
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Muzzin, P.1
Eisensmith, R.C.2
Copeland, K.C.3
Woo, S.L.4
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64
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Characterisation of wild-type adeno-associated virus type 2-like particles generated during recombinant viral vector production and strategies for their elimination
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Wang X, Khuntirat B, Qing K, Ponnazhagan S, Kube D, Zhou S, Dwarki Z, Srivastaba A. Characterisation of wild-type adeno-associated virus type 2-like particles generated during recombinant viral vector production and strategies for their elimination. J Virol. 72:1998;5472-5480.
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(1998)
J Virol
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Wang, X.1
Khuntirat, B.2
Qing, K.3
Ponnazhagan, S.4
Kube, D.5
Zhou, S.6
Dwarki, Z.7
Srivastaba, A.8
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