-
1
-
-
84925511283
-
Alipogene tiparvovec: A review of its use in adults with familial lipoprotein lipase deficiency
-
Scott LJ. Alipogene tiparvovec: A review of its use in adults with familial lipoprotein lipase deficiency. Drugs 2015;75:175-182.
-
(2015)
Drugs
, vol.75
, pp. 175-182
-
-
Scott, L.J.1
-
2
-
-
84911383748
-
Longterm safety and efficacy of factor IX gene therapy in hemophilia B
-
Nathwani AC, Reiss UM, Tuddenham EG, et al. Longterm safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014;371:1994-2004.
-
(2014)
N Engl J Med
, vol.371
, pp. 1994-2004
-
-
Nathwani, A.C.1
Reiss, U.M.2
Tuddenham, E.G.3
-
3
-
-
84878581647
-
Three-year follow-up after unilateral subretinal delivery of adeno-Associated virus in patients with Leber congenital amaurosis type 2
-
Testa F, Maguire AM, Rossi S, et al. Three-year follow-up after unilateral subretinal delivery of adeno-Associated virus in patients with Leber congenital amaurosis type 2. Ophthalmology 2013;120:1283-1291.
-
(2013)
Ophthalmology
, vol.120
, pp. 1283-1291
-
-
Testa, F.1
Maguire, A.M.2
Rossi, S.3
-
4
-
-
85007302654
-
AAV2 gene therapy readministration in three adults with congenital blindness
-
Bennett J, Ashtari M, Wellman J, et al. AAV2 gene therapy readministration in three adults with congenital blindness. Sci Transl Med 2012;4:120ra115.
-
(2012)
Sci Transl Med
, vol.4
, pp. 120ra115
-
-
Bennett, J.1
Ashtari, M.2
Wellman, J.3
-
5
-
-
0038150506
-
Helper virusfree, optically controllable, and two-plasmid-based production of adeno-Associated virus vectors of serotypes 1 to 6
-
Grimm D, Kay MA, Kleinschmidt JA. Helper virusfree, optically controllable, and two-plasmid-based production of adeno-Associated virus vectors of serotypes 1 to 6. Mol Ther 2003;7:839-850.
-
(2003)
Mol Ther
, vol.7
, pp. 839-850
-
-
Grimm, D.1
Kay, M.A.2
Kleinschmidt, J.A.3
-
6
-
-
2642642141
-
Production of high-Titer recombinant adeno-Associated virus vectors in the absence of helper adenovirus
-
Xiao X, Li J, Samulski RJ. Production of high-Titer recombinant adeno-Associated virus vectors in the absence of helper adenovirus. J Virol 1998;72:2224-2232.
-
(1998)
J Virol
, vol.72
, pp. 2224-2232
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
7
-
-
0036431701
-
Insect cells as a factory to produce adeno-Associated virus type 2 vectors
-
Urabe M, Ding C, Kotin RM. Insect cells as a factory to produce adeno-Associated virus type 2 vectors. Hum Gene Ther 2002;13:1935-1943.
-
(2002)
Hum Gene Ther
, vol.13
, pp. 1935-1943
-
-
Urabe, M.1
Ding, C.2
Kotin, R.M.3
-
8
-
-
70449103126
-
A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields hightiter rAAV stocks from insect cells
-
Smith RH, Levy JR, Kotin RM. A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields hightiter rAAV stocks from insect cells. Mol Ther 2009;17:1888-1896.
-
(2009)
Mol Ther
, vol.17
, pp. 1888-1896
-
-
Smith, R.H.1
Levy, J.R.2
Kotin, R.M.3
-
9
-
-
65249147237
-
An inducible system for highly efficient production of recombinant adeno-Associated virus (rAAV) vectors in insect Sf9 cells
-
Aslanidi G, Lamb K, Zolotukhin S. An inducible system for highly efficient production of recombinant adeno-Associated virus (rAAV) vectors in insect Sf9 cells. Proc Natl Acad Sci U S A 2009;106:5059-5064.
-
(2009)
Proc Natl Acad Sci U S A
, vol.106
, pp. 5059-5064
-
-
Aslanidi, G.1
Lamb, K.2
Zolotukhin, S.3
-
10
-
-
84896368264
-
OneBac: Platform for scalable and high-Titer production of adeno-Associated virus serotype 1-12 vectors for gene therapy
-
Mietzsch M, Grasse S, Zurawski C, et al. OneBac: platform for scalable and high-Titer production of adeno-Associated virus serotype 1-12 vectors for gene therapy. Hum Gene Ther 2014;25:212-222.
-
(2014)
Hum Gene Ther
, vol.25
, pp. 212-222
-
-
Mietzsch, M.1
Grasse, S.2
Zurawski, C.3
-
11
-
-
84945560129
-
OneBac 2.0: Sf9 cell lines for production of AAV5 vectors with enhanced infectivity and minimal encapsidation of foreign DNA
-
Mietzsch M, Casteleyn V, Weger S, et al. OneBac 2.0: Sf9 cell lines for production of AAV5 vectors with enhanced infectivity and minimal encapsidation of foreign DNA. Hum Gene Ther 2015;26: 688-697.
-
(2015)
Hum Gene Ther
, vol.26
, pp. 688-697
-
-
Mietzsch, M.1
Casteleyn, V.2
Weger, S.3
-
12
-
-
42549137311
-
Intron splicing-mediated expression of AAV Rep and Cap genes and production of AAV vectors in insect cells
-
Chen H. Intron splicing-mediated expression of AAV Rep and Cap genes and production of AAV vectors in insect cells. Mol Ther 2008;16:924-930.
-
(2008)
Mol Ther
, vol.16
, pp. 924-930
-
-
Chen, H.1
-
13
-
-
84896730363
-
Differential adeno-Associated virus serotype-specific interaction patterns with synthetic heparins and other glycans
-
Mietzsch M, Broecker F, Reinhardt A, et al. Differential adeno-Associated virus serotype-specific interaction patterns with synthetic heparins and other glycans. J Virol 2014;88:2991-3003.
-
(2014)
J Virol
, vol.88
, pp. 2991-3003
-
-
Mietzsch, M.1
Broecker, F.2
Reinhardt, A.3
-
14
-
-
77953439661
-
A viral assembly factor promotes AAV2 capsid formation in the nucleolus
-
Sonntag F, Schmidt K, Kleinschmidt JA. A viral assembly factor promotes AAV2 capsid formation in the nucleolus. Proc Natl Acad Sci U S A 2010;107:10220-10225.
-
(2010)
Proc Natl Acad Sci U S A
, vol.107
, pp. 10220-10225
-
-
Sonntag, F.1
Schmidt, K.2
Kleinschmidt, J.A.3
-
15
-
-
0036246727
-
The VP1 capsid protein of adeno-Associated virus type 2 is carrying a phospholipase A2 domain required for virus infectivity
-
Girod A, Wobus CE, Zadori Z, et al. The VP1 capsid protein of adeno-Associated virus type 2 is carrying a phospholipase A2 domain required for virus infectivity. J Gen Virol 2002;83:973-978.
-
(2002)
J Gen Virol
, vol.83
, pp. 973-978
-
-
Girod, A.1
Wobus, C.E.2
Zadori, Z.3
-
16
-
-
84861998784
-
Intracellular transport of recombinant adeno-Associated virus vectors
-
Nonnenmacher M, Weber T. Intracellular transport of recombinant adeno-Associated virus vectors. Gene Ther 2012;19:649-658.
-
(2012)
Gene Ther
, vol.19
, pp. 649-658
-
-
Nonnenmacher, M.1
Weber, T.2
-
17
-
-
0023751447
-
Synthesis of adeno-Associated virus structural proteins requires both alternative mRNA splicing and alternative initiations from a single transcript
-
Becerra SP, Koczot F, Fabisch P, et al. Synthesis of adeno-Associated virus structural proteins requires both alternative mRNA splicing and alternative initiations from a single transcript. J Virol 1988;62:2745-2754.
-
(1988)
J Virol
, vol.62
, pp. 2745-2754
-
-
Becerra, S.P.1
Koczot, F.2
Fabisch, P.3
-
18
-
-
28444473059
-
Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system
-
Kohlbrenner E, Aslanidi G, Nash K, et al. Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system. Mol Ther 2005;12:1217-1225.
-
(2005)
Mol Ther
, vol.12
, pp. 1217-1225
-
-
Kohlbrenner, E.1
Aslanidi, G.2
Nash, K.3
-
19
-
-
58149251820
-
Undetectable transcription of cap in a clinical AAV vector: Implications for preformed capsid in immune responses
-
Hauck B, Murphy SL, Smith PH, et al. Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol Ther 2009;17:144-152.
-
(2009)
Mol Ther
, vol.17
, pp. 144-152
-
-
Hauck, B.1
Murphy, S.L.2
Smith, P.H.3
-
20
-
-
84883020987
-
Generation and characterization of adeno-Associated virus producer cell lines for research and preclinical vector production
-
Martin J, Frederick A, Luo Y, et al. Generation and characterization of adeno-Associated virus producer cell lines for research and preclinical vector production. Hum Gene Ther Methods 2013;24:253-269.
-
(2013)
Hum Gene Ther Methods
, vol.24
, pp. 253-269
-
-
Martin, J.1
Frederick, A.2
Luo, Y.3
-
21
-
-
84959337169
-
Advanced characterization of DNA molecules in rAAV vector preparations by single-stranded virus next-generation sequencing
-
Lecomte E, Tournaire B, Cogne B, et al. Advanced characterization of DNA molecules in rAAV vector preparations by single-stranded virus next-generation sequencing. Mol Ther Nucleic Acids 2015;4:e260.
-
(2015)
Mol Ther Nucleic Acids
, vol.4
, pp. e260
-
-
Lecomte, E.1
Tournaire, B.2
Cogne, B.3
-
22
-
-
0344406980
-
Packaging of human chromosome 19-specific adeno-Associated virus (AAV) integration sites in AAV virions during AAV wild-Type and recombinant AAV vector production
-
Hüser D, Weger S, Heilbronn R. Packaging of human chromosome 19-specific adeno-Associated virus (AAV) integration sites in AAV virions during AAV wild-Type and recombinant AAV vector production. J Virol 2003;77:4881-4887.
-
(2003)
J Virol
, vol.77
, pp. 4881-4887
-
-
Hüser, D.1
Weger, S.2
Heilbronn, R.3
-
23
-
-
84906058160
-
A draft genome assembly of the army worm Spodoptera frugiperda
-
Kakumani PK, Malhotra P, Mukherjee SK, et al. A draft genome assembly of the army worm, Spodoptera frugiperda. Genomics 2014;104:134-143.
-
(2014)
Genomics
, vol.104
, pp. 134-143
-
-
Kakumani, P.K.1
Malhotra, P.2
Mukherjee, S.K.3
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