메뉴 건너뛰기




Volumn 29, Issue 3, 2018, Pages 146-155

In Vivo Potency Assay for Adeno-Associated Virus-Based Gene Therapy Vectors Using AAVrh.10 as an Example

Author keywords

AAV Vectors; Gene therapy; potency; quality control

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; DNA; MESSENGER RNA; RNA; RNA 18S;

EID: 85048932212     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2017.246     Document Type: Article
Times cited : (17)

References (35)
  • 1
    • 85048935321 scopus 로고    scopus 로고
    • Food and Drug Administration. Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products (last accessed May 21
    • Food and Drug Administration. Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products. https://www.fda.gov/downloads/ biologicsbloodvaccines/guidancecomplianceregulatory information/guidances/cellularandgenetherapy/ ucm243392.pdf (last accessed May 21, 2018).
    • (2018)
  • 2
    • 84939246896 scopus 로고    scopus 로고
    • Preclinical potency and biodistribution studies of an AAV 5 vector expressing human interferonbeta (ART-I02) for local treatment of patients with rheumatoid arthritis
    • Aalbers CJ, Bevaart L, Loiler S, et al. Preclinical potency and biodistribution studies of an AAV 5 vector expressing human interferonbeta (ART-I02) for local treatment of patients with rheumatoid arthritis. PLoS One 2015;10: e0130612.
    • (2015) PLoS One , vol.10 , pp. e0130612
    • Aalbers, C.J.1    Bevaart, L.2    Loiler, S.3
  • 3
    • 79955405275 scopus 로고    scopus 로고
    • Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial
    • Allay JA, Sleep S, Long S, et al. Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum Gene Ther 2011;22: 595-604.
    • (2011) Hum Gene Ther , vol.22 , pp. 595-604
    • Allay, J.A.1    Sleep, S.2    Long, S.3
  • 4
    • 79951817601 scopus 로고    scopus 로고
    • Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method
    • Chulay JD, Ye GJ, Thomas DL, et al. Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther 2011;22:155-165.
    • (2011) Hum Gene Ther , vol.22 , pp. 155-165
    • Chulay, J.D.1    Ye, G.J.2    Thomas, D.L.3
  • 5
    • 0038488551 scopus 로고    scopus 로고
    • Phase i trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: A two-part clinical study
    • Flotte TR, Zeitlin PL, Reynolds TC, et al. Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study. Hum Gene Ther 2003;14:1079-1088.
    • (2003) Hum Gene Ther , vol.14 , pp. 1079-1088
    • Flotte, T.R.1    Zeitlin, P.L.2    Reynolds, T.C.3
  • 6
    • 0037143294 scopus 로고    scopus 로고
    • Clinical protocol gene therapy of canavan disease: Aav-2 vector for neurosurgical delivery of aspartoacylase gene (aspa) to the human brain
    • Janson C, McPhee S, Bilaniuk L, et al. Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain. Hum Gene Ther 2002;13:1391-1412.
    • (2002) Hum Gene Ther , vol.13 , pp. 1391-1412
    • Janson, C.1    McPhee, S.2    Bilaniuk, L.3
  • 7
    • 44249085878 scopus 로고    scopus 로고
    • Safety and efficacy of gene transfer for Leber's congenital amaurosis
    • Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008;358:2240-2248.
    • (2008) N Engl J Med , vol.358 , pp. 2240-2248
    • Maguire, A.M.1    Simonelli, F.2    Pierce, E.A.3
  • 8
    • 78349247631 scopus 로고    scopus 로고
    • Gene delivery of AAV2-neurturin for Parkinson's disease: A double-blind, randomised, controlled trial
    • Marks WJ, Jr., Bartus RT, Siffert J, et al. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol 2010;9:1164-1172.
    • (2010) Lancet Neurol , vol.9 , pp. 1164-1172
    • Marks, W.J.1    Bartus, R.T.2    Siffert, J.3
  • 9
    • 33744825168 scopus 로고    scopus 로고
    • Immune responses to AAV in a Phase i study for Canavan disease
    • McPhee SW, Janson CG, Li C, et al. Immune responses to AAV in a Phase I study for Canavan disease. J Gene Med 2006;8:577-588.
    • (2006) J Gene Med , vol.8 , pp. 577-588
    • McPhee, S.W.1    Janson, C.G.2    Li, C.3
  • 10
    • 84855161388 scopus 로고    scopus 로고
    • Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    • Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011;365: 2357-2365.
    • (2011) N Engl J Med , vol.365 , pp. 2357-2365
    • Nathwani, A.C.1    Tuddenham, E.G.2    Rangarajan, S.3
  • 11
    • 84902952909 scopus 로고    scopus 로고
    • Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: Results of a Phase I/II trial
    • Tardieu M, Zerah M, Husson B, et al. Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a Phase I/II trial. Hum Gene Ther 2014;25:506-516.
    • (2014) Hum Gene Ther , vol.25 , pp. 506-516
    • Tardieu, M.1    Zerah, M.2    Husson, B.3
  • 12
    • 44349136847 scopus 로고    scopus 로고
    • Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adenoassociated virus expressing CLN2 cDNA
    • Worgall S, Sondhi D, Hackett NR, et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adenoassociated virus expressing CLN2 cDNA. Hum Gene Ther 2008;19:463-474.
    • (2008) Hum Gene Ther , vol.19 , pp. 463-474
    • Worgall, S.1    Sondhi, D.2    Hackett, N.R.3
  • 13
    • 44049108096 scopus 로고    scopus 로고
    • Manufacturing and characterizing AAV-based vectors for use in clinical studies
    • Wright JF. Manufacturing and characterizing AAV-based vectors for use in clinical studies. Gene Ther 2008;15:840-848.
    • (2008) Gene Ther , vol.15 , pp. 840-848
    • Wright, J.F.1
  • 14
    • 0034572459 scopus 로고    scopus 로고
    • Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors
    • Chao H, Liu Y, Rabinowitz J, et al. Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther 2000;2:619-623.
    • (2000) Mol Ther , vol.2 , pp. 619-623
    • Chao, H.1    Liu, Y.2    Rabinowitz, J.3
  • 15
    • 84874534423 scopus 로고    scopus 로고
    • A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype
    • Ellis BL, Hirsch ML, Barker JC, et al. A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype. Virol J 2013;10:74.
    • (2013) Virol J , vol.10 , pp. 74
    • Ellis, B.L.1    Hirsch, M.L.2    Barker, J.C.3
  • 16
    • 0141456083 scopus 로고    scopus 로고
    • Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector
    • Scallan CD, Lillicrap D, Jiang H, et al. Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector. Blood 2003;102:2031-2037.
    • (2003) Blood , vol.102 , pp. 2031-2037
    • Scallan, C.D.1    Lillicrap, D.2    Jiang, H.3
  • 17
    • 19444365150 scopus 로고    scopus 로고
    • Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models
    • Davidoff AM, Gray JT, Ng CY, et al. Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol Ther 2005;11:875-888.
    • (2005) Mol Ther , vol.11 , pp. 875-888
    • Davidoff, A.M.1    Gray, J.T.2    Ng, C.Y.3
  • 18
    • 33645992763 scopus 로고    scopus 로고
    • Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype
    • Grimm D, Pandey K, Nakai H, et al. Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J Virol 2006;80: 426-439.
    • (2006) J Virol , vol.80 , pp. 426-439
    • Grimm, D.1    Pandey, K.2    Nakai, H.3
  • 19
    • 77956628142 scopus 로고    scopus 로고
    • RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy
    • Hu C, Busuttil RW, Lipshutz GS. RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy. J Gene Med 2010;12:766-778.
    • (2010) J Gene Med , vol.12 , pp. 766-778
    • Hu, C.1    Busuttil, R.W.2    Lipshutz, G.S.3
  • 20
    • 58149459993 scopus 로고    scopus 로고
    • Enhancing transduction of the liver by adenoassociated viral vectors
    • Nathwani AC, Cochrane M, McIntosh J, et al. Enhancing transduction of the liver by adenoassociated viral vectors. Gene Ther 2009;16: 60-69.
    • (2009) Gene Ther , vol.16 , pp. 60-69
    • Nathwani, A.C.1    Cochrane, M.2    McIntosh, J.3
  • 21
    • 83455225637 scopus 로고    scopus 로고
    • AAV-mediated liver-directed gene therapy
    • Sands MS. AAV-mediated liver-directed gene therapy. Methods Mol Biol 2011;807:141-157.
    • (2011) Methods Mol Biol , vol.807 , pp. 141-157
    • Sands, M.S.1
  • 22
    • 85032437232 scopus 로고    scopus 로고
    • Aav biology, infectivity and therapeutic use from bench to clinic
    • In: Hashad D, ed. Rijeka, Croatia: InTech
    • Vance MA, Mitchell A, Samulski RJ. AAV biology, infectivity and therapeutic use from bench to Clinic. In: Hashad D, ed. Gene Therapy-Principles and Challenges. Rijeka, Croatia: InTech, 2015:119-143.
    • (2015) Gene Therapy-Principles and Challenges , pp. 119-143
    • Vance, M.A.1    Mitchell, A.2    Samulski, R.J.3
  • 23
    • 44349170706 scopus 로고    scopus 로고
    • Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
    • Zincarelli C, Soltys S, Rengo G, et al. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008;16:1073-1080.
    • (2008) Mol Ther , vol.16 , pp. 1073-1080
    • Zincarelli, C.1    Soltys, S.2    Rengo, G.3
  • 24
    • 0037818636 scopus 로고    scopus 로고
    • Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway
    • Davidoff AM, Ng CY, Zhou J, et al. Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood 2003;102:480-488.
    • (2003) Blood , vol.102 , pp. 480-488
    • Davidoff, A.M.1    Ng, C.Y.2    Zhou, J.3
  • 25
    • 28844483864 scopus 로고    scopus 로고
    • High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses
    • De BP, Heguy A, Hackett NR, et al. High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses. Mol Ther 2006;13:67-76.
    • (2006) Mol Ther , vol.13 , pp. 67-76
    • De, B.P.1    Heguy, A.2    Hackett, N.R.3
  • 26
    • 33745143956 scopus 로고    scopus 로고
    • Robust systemic transduction with AAV9 vectors in mice: Efficient global cardiac gene transfer superior to that of AAV8
    • Inagaki K, Fuess S, Storm TA, et al. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol Ther 2006;14:45-53.
    • (2006) Mol Ther , vol.14 , pp. 45-53
    • Inagaki, K.1    Fuess, S.2    Storm, T.A.3
  • 27
    • 80455164698 scopus 로고    scopus 로고
    • Systemic administration of AAV8-alpha-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression
    • Nietupski JB, Hurlbut GD, Ziegler RJ, et al. Systemic administration of AAV8-alpha-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression. Mol Ther 2011;19:1999-2011.
    • (2011) Mol Ther , vol.19 , pp. 1999-2011
    • Nietupski, J.B.1    Hurlbut, G.D.2    Ziegler, R.J.3
  • 28
    • 84921528269 scopus 로고    scopus 로고
    • Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease
    • Wang G, Young SP, Bali D, et al. Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease. Mol Ther Methods Clin Dev 2014;1:14018.
    • (2014) Mol Ther Methods Clin Dev , vol.1 , pp. 14018
    • Wang, G.1    Young, S.P.2    Bali, D.3
  • 29
    • 38649139896 scopus 로고    scopus 로고
    • Optimization of selfcomplementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose
    • Wu Z, Sun J, Zhang T, et al. Optimization of selfcomplementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose. Mol Ther 2008; 16:280-289.
    • (2008) Mol Ther , vol.16 , pp. 280-289
    • Wu, Z.1    Sun, J.2    Zhang, T.3
  • 30
    • 84936115156 scopus 로고    scopus 로고
    • Genomic copy number variation in Mus musculus
    • Locke ME, Milojevic M, Eitutis ST, et al. Genomic copy number variation in Mus musculus. BMC Genomics 2015;16:497.
    • (2015) BMC Genomics , vol.16 , pp. 497
    • Locke, M.E.1    Milojevic, M.2    Eitutis, S.T.3
  • 31
    • 79959446383 scopus 로고    scopus 로고
    • TFRC and ACTB as the best reference genes to quantify Urokinase Plasminogen Activator in breast cancer
    • Majidzadeh AK, Esmaeili R, Abdoli N. TFRC and ACTB as the best reference genes to quantify Urokinase Plasminogen Activator in breast cancer. BMC Res Notes 2011;4:215.
    • (2011) BMC Res Notes , vol.4 , pp. 215
    • Majidzadeh, A.K.1    Esmaeili, R.2    Abdoli, N.3
  • 32
    • 36849064740 scopus 로고    scopus 로고
    • Evaluation and validation of housekeeping genes in response to ionizing radiation and chemical exposure for normalizing RNA expression in real-time PCR
    • Banda M, Bommineni A, Thomas RA, et al. Evaluation and validation of housekeeping genes in response to ionizing radiation and chemical exposure for normalizing RNA expression in real-time PCR. Mutat Res 2008;649: 126-134.
    • (2008) Mutat Res , vol.649 , pp. 126-134
    • Banda, M.1    Bommineni, A.2    Thomas, R.A.3
  • 33
    • 0041441131 scopus 로고    scopus 로고
    • Development and assessment of a quantitative reverse transcription-PCR assay for simultaneous measurement of four amplicons
    • Grace MB, McLeland CB, Gagliardi SJ, et al. Development and assessment of a quantitative reverse transcription-PCR assay for simultaneous measurement of four amplicons. Clin Chem 2003; 49:1467-1475.
    • (2003) Clin Chem , vol.49 , pp. 1467-1475
    • Grace, M.B.1    McLeland, C.B.2    Gagliardi, S.J.3
  • 34
    • 84867037745 scopus 로고    scopus 로고
    • 18S rRNA is a reliable normalisation gene for real time PCR based on influenza virus infected cells
    • Kuchipudi SV, Tellabati M, Nelli RK, et al. 18S rRNA is a reliable normalisation gene for real time PCR based on influenza virus infected cells. Virol J 2012;9:230.
    • (2012) Virol J , vol.9 , pp. 230
    • Kuchipudi, S.V.1    Tellabati, M.2    Nelli, R.K.3
  • 35
    • 27944437854 scopus 로고    scopus 로고
    • AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL
    • Sondhi D, Peterson DA, Giannaris EL, et al. AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL. Gene Ther 2005;12:1618-1632.
    • (2005) Gene Ther , vol.12 , pp. 1618-1632
    • Sondhi, D.1    Peterson, D.A.2    Giannaris, E.L.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.