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Volumn 507, Issue , 2012, Pages 297-311

Recombinant adeno-associated viral vector reference standards

Author keywords

Adeno associated virus; Characterization; Gene transfer; Quality control; Reference standard; Safety

Indexed keywords

ENDOTOXIN; PARVOVIRUS VECTOR;

EID: 84857582187     PISSN: 00766879     EISSN: 15577988     Source Type: Book Series    
DOI: 10.1016/B978-0-12-386509-0.00015-6     Document Type: Chapter
Times cited : (4)

References (48)
  • 1
    • 79955405275 scopus 로고    scopus 로고
    • Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial
    • J.A. Allay Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial Hum. Gene Ther. 22 2011 595 604
    • (2011) Hum. Gene Ther. , vol.22 , pp. 595-604
    • Allay, J.A.1
  • 3
    • 70349481529 scopus 로고    scopus 로고
    • Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
    • M.L. Brantly Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy Proc. Natl. Acad. Sci. USA 106 2009 16363 16368
    • (2009) Proc. Natl. Acad. Sci. USA , vol.106 , pp. 16363-16368
    • Brantly, M.L.1
  • 4
    • 4344566457 scopus 로고    scopus 로고
    • Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
    • DOI 10.1016/j.ymthe.2004.05.024, PII S1525001604001911
    • C. Burger Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system Mol. Ther. 10 2004 302 317 (Pubitemid 39117875)
    • (2004) Molecular Therapy , vol.10 , Issue.2 , 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
    • 0034989793 scopus 로고    scopus 로고
    • A death in the laboratory: The politics of the Gelsinger aftermath
    • DOI 10.1006/mthe.2001.0305
    • I.H. Carmen A death in the laboratory: The politics of the Gelsinger aftermath Mol. Ther. 3 2001 425 428 (Pubitemid 32509834)
    • (2001) Molecular Therapy , vol.3 , Issue.4 , pp. 425-428
    • Carmen, I.H.1
  • 6
    • 0034218551 scopus 로고    scopus 로고
    • Efficient recombinant adeno-associated virus production by a stable rep-cap HeLa cell line correlates with adenovirus-induced amplification of the integrated rep-cap genome
    • (In process citation)
    • G. Chadeuf Efficient recombinant adeno-associated virus production by a stable rep-cap HeLa cell line correlates with adenovirus-induced amplification of the integrated rep-cap genome J. Gene Med. 2 2000 260 268 (In process citation)
    • (2000) J. Gene Med. , vol.2 , pp. 260-268
    • Chadeuf, G.1
  • 7
    • 70349105559 scopus 로고    scopus 로고
    • Human RPE65 gene therapy for Leber congenital amaurosis: Persistence of early visual improvements and safety at 1 year
    • A.V. Cideciyan Human RPE65 gene therapy for Leber congenital amaurosis: Persistence of early visual improvements and safety at 1 year Hum. Gene Ther. 20 2009 999 1004
    • (2009) Hum. Gene Ther. , vol.20 , pp. 999-1004
    • Cideciyan, A.V.1
  • 8
    • 0030458783 scopus 로고    scopus 로고
    • A stable cell line carrying adenovirus-inducible rep and cap genes allows for infectivity titration of adeno-associated virus vectors
    • K.R. Clark, F. Voulgaropoulou, and P.R. Johnson A stable cell line carrying adenovirus-inducible rep and cap genes allows for infectivity titration of adeno-associated virus vectors Gene Ther. 3 1996 1124 1132
    • (1996) Gene Ther. , vol.3 , pp. 1124-1132
    • Clark, K.R.1    Voulgaropoulou, F.2    Johnson, P.R.3
  • 9
    • 0033541640 scopus 로고    scopus 로고
    • Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses
    • DOI 10.1089/10430349950018427
    • K.R. Clark, X. Liu, J.P. McGrath, and P.R. Johnson 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 (Pubitemid 29195432)
    • (1999) Human Gene Therapy , vol.10 , Issue.6 , pp. 1031-1039
    • Clark, K.R.1    Liu, X.2    Mcgrath, J.P.3    Johnson, P.R.4
  • 12
    • 0034130380 scopus 로고    scopus 로고
    • High throughput production, screening and analysis of adeno-associated viral vectors
    • L. Drittanti, C. Rivet, P. Manceau, O. Danos, and M. Vega High throughput production, screening and analysis of adeno-associated viral vectors Gene Ther. 7 2000 924 929 (Pubitemid 30386656)
    • (2000) Gene Therapy , vol.7 , Issue.11 , pp. 924-929
    • Drittanti, L.1    Rivet, C.2    Manceau, P.3    Danos, O.4    Vega, M.5
  • 13
    • 0035839562 scopus 로고    scopus 로고
    • Subthalamic GAD gene transfer in Parkinson disease patients who are candidates for deep brain stimulation
    • M.J. During, M.G. Kaplitt, M.B. Stern, and D. Eidelberg 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
  • 14
    • 33645874186 scopus 로고    scopus 로고
    • Utility of a recombinant adeno-associated viral vector reference standard
    • T.R. Flotte, P. Burd, and R.O. Snyder Utility of a recombinant adeno-associated viral vector reference standard Bioprocessing 1 2002 75 77
    • (2002) Bioprocessing , vol.1 , pp. 75-77
    • Flotte, T.R.1    Burd, P.2    Snyder, R.O.3
  • 16
    • 0742307402 scopus 로고    scopus 로고
    • Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Alpha 1-Antitrypsin (rAAV2-CB-hAAT) Gene Vector to AAT-Deficient Adults
    • DOI 10.1089/10430340460732490
    • T.R. Flotte 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 (Pubitemid 38147192)
    • (2004) Human Gene Therapy , vol.15 , Issue.1 , pp. 93-128
    • Flotte, T.R.1    Brantly, M.L.2    Spencer, L.T.3    Byrne, B.J.4    Spencer, C.T.5    Baker, D.J.6    Humphries, M.7
  • 18
    • 85018823083 scopus 로고    scopus 로고
    • Gene Therapy Clinical Trials Worldwide
    • June 2011 Update
    • Gene Therapy Clinical Trials Worldwide. June 2011 Update. J. Gene Med. http://www.wiley.co.uk/genmed/clinical/
    • J. Gene Med.
  • 19
    • 0032773889 scopus 로고    scopus 로고
    • Titration of AAV-2 particles via a novel capsid ELISA: Packaging of genomes can limit production of recombinant AAV-2
    • DOI 10.1038/sj.gt.3300946
    • D. Grimm, A. Kern, M. Pawlita, F. Ferrari, R. Samulski, and J. Kleinschmidt 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 (Pubitemid 29359028)
    • (1999) Gene Therapy , vol.6 , Issue.7 , pp. 1322-1330
    • Grimm, D.1    Kern, A.2    Pawlita, M.3    Ferrari, F.K.4    Samulski, R.J.5    Kleinschmidt, J.A.6
  • 20
    • 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
    • DOI 10.1016/S1525-0016(03)00095-9
    • D. Grimm, M.A. Kay, and J.A. Kleinschmidt Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6 Mol. Ther. 7 2003 839 850 (Pubitemid 36790261)
    • (2003) Molecular Therapy , vol.7 , Issue.6 , pp. 839-850
    • Grimm, D.1    Kay, M.A.2    Kleinschmidt, J.A.3
  • 21
    • 44049098137 scopus 로고    scopus 로고
    • Update on progress and hurdles in novel genetic therapies for hemophilia
    • K.A. High Update on progress and hurdles in novel genetic therapies for hemophilia Hematol. Am. Soc. Hematol. Educ. Program 2007 2007 466 472
    • (2007) Hematol. Am. Soc. Hematol. Educ. Program , vol.2007 , pp. 466-472
    • High, K.A.1
  • 22
    • 0346106067 scopus 로고    scopus 로고
    • Development of a reference material for characterizing adenovirus vectors
    • B. Hutchins Development of a reference material for characterizing adenovirus vectors Bioprocessing 1 2002 25 28
    • (2002) Bioprocessing , vol.1 , pp. 25-28
    • Hutchins, B.1
  • 23
    • 0034567122 scopus 로고    scopus 로고
    • Working toward an adenoviral vector testing standard
    • B. Hutchins Working toward an adenoviral vector testing standard Mol. Ther. 2 2000 532 534
    • (2000) Mol. Ther. , vol.2 , pp. 532-534
    • Hutchins, B.1
  • 25
    • 79958138516 scopus 로고    scopus 로고
    • Adeno-associated viral vector-mediated transgene expression is independent of DNA methylation in primate liver and skeletal muscle
    • A. Leger Adeno-associated viral vector-mediated transgene expression is independent of DNA methylation in primate liver and skeletal muscle PLoS One 6 2011 e20881
    • (2011) PLoS One , vol.6 , pp. 20881
    • Leger, A.1
  • 26
    • 77958099436 scopus 로고    scopus 로고
    • Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material
    • M. Lock Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material Hum. Gene Ther. 21 2010 1273 1285
    • (2010) Hum. Gene Ther. , vol.21 , pp. 1273-1285
    • Lock, M.1
  • 30
    • 0346777307 scopus 로고    scopus 로고
    • Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
    • DOI 10.1038/sj.gt.3302134
    • D.M. McCarty, H. Fu, P.E. Monahan, C.E. Toulson, P. Naik, and R.J. Samulski Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo Gene Ther. 10 2003 2112 2118 (Pubitemid 37536760)
    • (2003) Gene Therapy , vol.10 , Issue.26 , pp. 2112-2118
    • McCarty, D.M.1    Fu, H.2    Monahan, P.E.3    Toulson, C.E.4    Naik, P.5    Samulski, R.J.6
  • 31
    • 77957725001 scopus 로고    scopus 로고
    • Dystrophin immunity in Duchenne's muscular dystrophy
    • J.R. Mendell Dystrophin immunity in Duchenne's muscular dystrophy N. Engl. J. Med. 363 2010 1429 1437
    • (2010) N. Engl. J. Med. , vol.363 , pp. 1429-1437
    • Mendell, J.R.1
  • 32
    • 69949160511 scopus 로고    scopus 로고
    • AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
    • F. Mingozzi AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells Blood 114 2009 2077 2086
    • (2009) Blood , vol.114 , pp. 2077-2086
    • Mingozzi, F.1
  • 34
    • 45749101234 scopus 로고    scopus 로고
    • International efforts for recombinant adeno-associated viral vector reference standards
    • DOI 10.1038/mt.2008.125, PII MT2008125
    • P. Moullier, and R.O. Snyder International efforts for recombinant adeno-associated viral vector reference standards Mol. Ther. 16 2008 1185 1188 (Pubitemid 351866048)
    • (2008) Molecular Therapy , vol.16 , Issue.7 , pp. 1185-1188
    • Moullier, P.1    Snyder, R.O.2
  • 35
    • 0038054338 scopus 로고    scopus 로고
    • AAV serotype 2 vectors preferentially integrate into active genes in mice
    • DOI 10.1038/ng1179
    • H. Nakai, E. Montini, S. Fuess, T.A. Storm, M. Grompe, and M.A. Kay AAV serotype 2 vectors preferentially integrate into active genes in mice Nat. Genet. 34 2003 297 302 (Pubitemid 36792862)
    • (2003) Nature Genetics , vol.34 , Issue.3 , pp. 297-302
    • Nakai, H.1    Montini, E.2    Fuess, S.3    Storm, T.A.4    Grompe, M.5    Kay, M.A.6
  • 36
    • 49149113894 scopus 로고    scopus 로고
    • Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle
    • M. Penaud-Budloo Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle J. Virol. 82 2008 7875 7885
    • (2008) J. Virol. , vol.82 , pp. 7875-7885
    • Penaud-Budloo, M.1
  • 37
    • 79952149008 scopus 로고    scopus 로고
    • Hemophilia gene therapy: A holy grail found
    • K.P. Ponder Hemophilia gene therapy: A holy grail found Mol. Ther. 19 2011 427 428
    • (2011) Mol. Ther. , vol.19 , pp. 427-428
    • Ponder, K.P.1
  • 38
    • 77958087392 scopus 로고    scopus 로고
    • Manufacture and stability study of the recombinant adeno-associated virus serotype 2 vector reference standard
    • M. Potter Manufacture and stability study of the recombinant adeno-associated virus serotype 2 vector reference standard Bioprocessing 7 2008 8 14
    • (2008) Bioprocessing , vol.7 , pp. 8-14
    • Potter, M.1
  • 39
    • 0036811528 scopus 로고    scopus 로고
    • Production of clinical-grade recombinant adeno-associated virus vectors
    • DOI 10.1016/S0958-1669(02)00369-5
    • R.O. Snyder, and T.R. Flotte Production of clinical-grade recombinant adeno-associated virus vectors Curr. Opin. Biotechnol. 13 2002 418 423 (Pubitemid 35379925)
    • (2002) Current Opinion in Biotechnology , vol.13 , Issue.5 , pp. 418-423
    • Snyder, R.O.1    Flotte, T.R.2
  • 40
    • 0002988871 scopus 로고    scopus 로고
    • Production of recombinant adeno-associated viral vectors
    • N. Dracopoli, John Wiley and Sons New York
    • R.O. Snyder, X. Xiao, and R.J. Samulski Production of recombinant adeno-associated viral vectors N. Dracopoli, Current Protocols in Human Genetics 1996 John Wiley and Sons New York 12.11.11 12.11.24
    • (1996) Current Protocols in Human Genetics , pp. 121111-121124
    • Snyder, R.O.1    Xiao, X.2    Samulski, R.J.3
  • 41
    • 0034689207 scopus 로고    scopus 로고
    • Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: α-, β-, γ-, or Δ-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors
    • DOI 10.1089/10430340050015671
    • H. Stedman, J.M. Wilson, R. Finke, A.L. Kleckner, and J. Mendell Phase I clinical trial utilizing gene therapy for limb girdle muscular dystrophy: Alpha-, beta-, gamma-, or delta-sarcoglycan gene delivered with intramuscular instillations of adeno-associated vectors Hum. Gene Ther. 11 2000 777 790 (Pubitemid 30180282)
    • (2000) Human Gene Therapy , vol.11 , Issue.5 , pp. 777-790
    • Stedman, H.1    Wilson, J.M.2    Finke, R.3    Kleckner, A.-L.4    Mendell, J.5
  • 42
    • 77955008397 scopus 로고    scopus 로고
    • Molecular analysis of vector genome structures after liver transduction by conventional and self-complementary adeno-associated viral serotype vectors in murine and nonhuman primate models
    • X. Sun, Y. Lu, L.T. Bish, R. Calcedo, J.M. Wilson, and G. Gao Molecular analysis of vector genome structures after liver transduction by conventional and self-complementary adeno-associated viral serotype vectors in murine and nonhuman primate models Hum. Gene Ther. 21 2010 750 761
    • (2010) Hum. Gene Ther. , vol.21 , pp. 750-761
    • Sun, X.1    Lu, Y.2    Bish, L.T.3    Calcedo, R.4    Wilson, J.M.5    Gao, G.6
  • 44
  • 47
    • 0031015285 scopus 로고    scopus 로고
    • Subcellular compartmentalization of adeno-associated virus type 2 assembly
    • A. Wistuba, A. Kern, S. Weger, D. Grimm, and J.A. Kleinschmidt Subcellular compartmentalization of adeno-associated virus type 2 assembly J. Virol. 71 1997 1341 1352 (Pubitemid 27030308)
    • (1997) Journal of Virology , vol.71 , Issue.2 , pp. 1341-1352
    • Wistuba, A.1    Kern, A.2    Weger, S.3    Grimm, D.4    Kleinschmidt, J.A.5
  • 48
    • 3142655660 scopus 로고    scopus 로고
    • Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events
    • DOI 10.1089/1043034041361262
    • Z. Zhen, Y. Espinoza, T. Bleu, J.M. Sommer, and J.F. Wright Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events Hum. Gene Ther. 15 2004 709 715 (Pubitemid 38925223)
    • (2004) Human Gene Therapy , vol.15 , Issue.7 , pp. 709-715
    • Zhen, Z.1    Espinoza, Y.2    Bleu, T.3    Sommer, J.M.4    Wright, J.F.5


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