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Volumn 4, Issue , 2017, Pages 511-534

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race between Clearance, Tolerance, Neutralization, and Escape

Author keywords

AAV; Gene therapy; Immunomonitoring; Immunotoxicity; Neutralizing antibodies; Tolerance

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; NEUTRALIZING ANTIBODY;

EID: 85030325583     PISSN: 2327056X     EISSN: 23270578     Source Type: Journal    
DOI: 10.1146/annurev-virology-101416-041936     Document Type: Article
Times cited : (149)

References (142)
  • 1
    • 0014143050 scopus 로고
    • Isolation of adenovirus-associated viruses from man
    • Blacklow NR, Hoggan MD, Rowe WP. 1967. Isolation of adenovirus-associated viruses from man. PNAS 58:1410-15
    • (1967) PNAS , vol.58 , pp. 1410-1415
    • Blacklow, N.R.1    Hoggan, M.D.2    Rowe, W.P.3
  • 2
    • 77953439661 scopus 로고    scopus 로고
    • A viral assembly factor promotes AAV2 capsid formation in the nucleolus
    • Sonntag F, Schmidt K, Kleinschmidt JA. 2010. A viral assembly factor promotes AAV2 capsid formation in the nucleolus. PNAS 107:10220-25
    • (2010) PNAS , vol.107 , pp. 10220-10225
    • Sonntag, F.1    Schmidt, K.2    Kleinschmidt, J.A.3
  • 3
    • 10644271519 scopus 로고    scopus 로고
    • Inverted terminal repeat sequences are important for intermolecular recombination and circularization of adeno-associated virus genomes
    • Yan Z, Zak R, Zhang Y, Engelhardt JF. 2004. Inverted terminal repeat sequences are important for intermolecular recombination and circularization of adeno-associated virus genomes. J. Virol. 79:364-79
    • (2004) J. Virol , vol.79 , pp. 364-379
    • Yan, Z.1    Zak, R.2    Zhang, Y.3    Engelhardt, J.F.4
  • 4
    • 2642642141 scopus 로고    scopus 로고
    • Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
    • Xiao X, Li J, Samulski RJ. 1998. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72:2224-32
    • (1998) J. Virol , vol.72 , pp. 2224-2232
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 5
    • 0036431701 scopus 로고    scopus 로고
    • Insect cells as a factory to produce adeno-associated virus type 2 vectors
    • Urabe M, Ding C, Kotin RM. 2002. Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum. Gene Ther. 13:1935-43
    • (2002) Hum. Gene Ther , vol.13 , pp. 1935-1943
    • Urabe, M.1    Ding, C.2    Kotin, R.M.3
  • 6
    • 84976453525 scopus 로고    scopus 로고
    • Progress and challenges in viral vector manufacturing
    • van der Loo JCM, Wright JF. 2016. Progress and challenges in viral vector manufacturing. Hum. Mol. Genet. 25:R42-52
    • (2016) Hum. Mol. Genet , vol.25 , pp. R42-52
    • Van Der Loo, J.C.M.1    Wright, J.F.2
  • 7
    • 84959530609 scopus 로고    scopus 로고
    • Production of recombinant adeno-associated virus vectors using suspension HEK293 cells and continuous harvest of vector from the culture media for GMP FIX and FLT1 clinical vector
    • Grieger JC, Soltys SM, Samulski RJ. 2016. Production of recombinant adeno-associated virus vectors using suspension HEK293 cells and continuous harvest of vector from the culture media for GMP FIX and FLT1 clinical vector. Mol. Ther. 24:287-97
    • (2016) Mol. Ther , vol.24 , pp. 287-297
    • Grieger, J.C.1    Soltys, S.M.2    Samulski, R.J.3
  • 8
    • 0031906147 scopus 로고    scopus 로고
    • Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
    • Summerford C, Samulski RJ. 1998. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72:1438-45
    • (1998) J. Virol , vol.72 , pp. 1438-1445
    • Summerford, C.1    Samulski, R.J.2
  • 9
    • 84957599961 scopus 로고    scopus 로고
    • An essential receptor for adenoassociated virus infection
    • Pillay S, Meyer NL, Puschnik AS, Davulcu O, Diep J, et al. 2016. An essential receptor for adenoassociated virus infection. Nature 530:108-12
    • (2016) Nature , vol.530 , pp. 108-112
    • Pillay, S.1    Meyer, N.L.2    Puschnik, A.S.3    Davulcu, O.4    Diep, J.5
  • 10
    • 20744433079 scopus 로고    scopus 로고
    • Intracellular trafficking of adeno-associated viral vectors
    • Ding W, Zhang L, Yan Z, Engelhardt JF. 2005. Intracellular trafficking of adeno-associated viral vectors. Gene Ther. 12:873-80
    • (2005) Gene Ther , vol.12 , pp. 873-880
    • Ding, W.1    Zhang, L.2    Yan, Z.3    Engelhardt, J.F.4
  • 11
    • 34147098413 scopus 로고    scopus 로고
    • CD8+ T-cell responses to adeno-associated virus capsid in humans
    • Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, et al. 2007. CD8+ T-cell responses to adeno-associated virus capsid in humans. Nat. Med. 13:419-22
    • (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
  • 12
    • 84862599629 scopus 로고    scopus 로고
    • Humoral and cellular capsidspecific immune responses to adeno-associated virus type 1 in randomized healthy donors
    • Veron P, Leborgne C, Monteilhet V, Boutin S, Martin S, et al. 2012. Humoral and cellular capsidspecific immune responses to adeno-associated virus type 1 in randomized healthy donors. J. Immunol. 188:6418-24
    • (2012) J. Immunol , vol.188 , pp. 6418-6424
    • Veron, P.1    Leborgne, C.2    Monteilhet, V.3    Boutin, S.4    Martin, S.5
  • 14
    • 84883713705 scopus 로고    scopus 로고
    • Anti-dystrophinTcell responses in Duchenne muscular dystrophy: Prevalence and a glucocorticoid treatment effect
    • FlaniganKM,Campbell K, ViolletL,WangW,Gomez AM, et al. 2013. Anti-dystrophinTcell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect. Hum. Gene Ther. 24:797-806
    • (2013) Hum. Gene Ther , vol.24 , pp. 797-806
    • Flanigan, K.M.1    Campbell, K.2    Viollet, L.3    Wang, W.4    Gomez, A.M.5
  • 15
    • 78651010946 scopus 로고
    • Immunity to homologous grafted skin; The fate of skin homografts transplanted to the brain, to subcutaneous tissue, and to the anterior chamber of the eye
    • Medawar PB. 1948. Immunity to homologous grafted skin; the fate of skin homografts transplanted to the brain, to subcutaneous tissue, and to the anterior chamber of the eye. Br. J. Exp. Pathol. 29:58-69
    • (1948) Br. J. Exp. Pathol , vol.29 , pp. 58-69
    • Medawar, P.B.1
  • 16
    • 84883719513 scopus 로고    scopus 로고
    • Immunology of AAV-mediated gene transfer in the eye
    • Willett K, Bennett J. 2013. Immunology of AAV-mediated gene transfer in the eye. Front. Immunol. 4:261
    • (2013) Front. Immunol , vol.4 , pp. 261
    • Willett, K.1    Bennett, J.2
  • 19
    • 54949104686 scopus 로고    scopus 로고
    • Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: Short-term results of a phase i trial
    • Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, et al. 2008. Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum. Gene Ther. 19:979-90
    • (2008) Hum. Gene Ther , vol.19 , pp. 979-990
    • Hauswirth, W.W.1    Aleman, T.S.2    Kaushal, S.3    Cideciyan, A.V.4    Schwartz, S.B.5
  • 21
    • 85023744466 scopus 로고    scopus 로고
    • Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in subjects with RPE65-mediated inherited retinal dystrophy: A randomised, controlled, open-label, phase 3 trial
    • Russell S, Bennett J, Wellman JA, ChungDC, Yu ZF, et al. 2017. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in subjects with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. https://doi.org/10.1016/S0140-6736(17)31868-8
    • (2017) Lancet
    • Russell, S.1    Bennett, J.2    Wellman, J.A.3    Chung, D.C.4    Yu, Z.F.5
  • 22
    • 84991408036 scopus 로고    scopus 로고
    • Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: A follow-on phase 1 trial
    • Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, et al. 2016. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Lancet 388:661-72
    • (2016) Lancet , vol.388 , pp. 661-672
    • Bennett, J.1    Wellman, J.2    Marshall, K.A.3    McCague, S.4    Ashtari, M.5
  • 23
    • 77952983302 scopus 로고    scopus 로고
    • Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness
    • Amado D, Mingozzi F, Hui D, Bennicelli JL, Wei Z, et al. 2010. Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness. Sci. Translational Med. 2:21ra16
    • (2010) Sci. Translational Med , vol.2 , pp. 21ra16
    • Amado, D.1    Mingozzi, F.2    Hui, D.3    Bennicelli, J.L.4    Wei, Z.5
  • 24
    • 79551622680 scopus 로고    scopus 로고
    • Preclinical safety evaluation of AAV2-sFLT01-A gene therapy for age-related macular degeneration
    • MacLachlan TK, Lukason M, Collins M, Munger R, Isenberger E, et al. 2011. Preclinical safety evaluation of AAV2-sFLT01-a gene therapy for age-related macular degeneration. Mol. Ther. 19:326-34
    • (2011) Mol. Ther , vol.19 , pp. 326-334
    • MacLachlan, T.K.1    Lukason, M.2    Collins, M.3    Munger, R.4    Isenberger, E.5
  • 25
    • 85030310446 scopus 로고    scopus 로고
    • A recombinant AAV2/2 carrying the wild-type ND4 gene for the treatment of LHON: Preliminary results of a first-in-man study and upcoming pivotal efficacy trials
    • (ISOPT Clin.), July 9-12, Berlin
    • Uretsky S, Sahel JA, Galy A, Thomasson N, Honnet G, et al. 2015. A recombinant AAV2/2 carrying the wild-type ND4 gene for the treatment of LHON: preliminary results of a first-in-man study and upcoming pivotal efficacy trials. Presented at 12th Int. Symp. Ocul. Pharmacol. Ther. (ISOPT Clin.), July 9-12, Berlin
    • (2015) Present at 12th Int. Symp. Ocul. Pharmacol. Ther
    • Uretsky, S.1    Sahel, J.A.2    Galy, A.3    Thomasson, N.4    Honnet, G.5
  • 26
    • 79954622209 scopus 로고    scopus 로고
    • Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
    • Mingozzi F, High KA. 2011. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat. Rev. Genet. 12:341-55
    • (2011) Nat. Rev. Genet , vol.12 , pp. 341-355
    • Mingozzi, F.1    High, K.A.2
  • 27
    • 77958557979 scopus 로고    scopus 로고
    • Antigen-presenting cell function in the tolerogenic liver environment
    • Thomson AW, Knolle PA. 2010. Antigen-presenting cell function in the tolerogenic liver environment. Nat. Rev. Immunol. 10:753-66
    • (2010) Nat. Rev. Immunol , vol.10 , pp. 753-766
    • Thomson, A.W.1    Knolle, P.A.2
  • 28
    • 33745804056 scopus 로고    scopus 로고
    • An appraisal of tolerance in liver transplantation
    • Lerut J, Sanchez-Fueyo A. 2006. An appraisal of tolerance in liver transplantation. Am. J. Transplant. 6:1774-80
    • (2006) Am. J. Transplant , vol.6 , pp. 1774-1780
    • Lerut, J.1    Sanchez-Fueyo, A.2
  • 29
    • 84905382365 scopus 로고    scopus 로고
    • Acquired atopic disease after liver transplantation in children; Similarities to and differences from adults
    • Topal E, Ç atal F, Selimoglu MA, Karabiber H, Klc T, et al. 2014. Acquired atopic disease after liver transplantation in children; similarities to and differences from adults. Eur. J. Gastroenterol. Hepatol. 26:1055-59
    • (2014) Eur. J. Gastroenterol. Hepatol , vol.26 , pp. 1055-1059
    • Topal, E.1    Atal, F.C.2    Selimoglu, M.A.3    Karabiber, H.4    Klc, T.5
  • 30
    • 34948855431 scopus 로고    scopus 로고
    • Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
    • Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E, Edmonson SA, Hui DJ, et al. 2007. Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood 110:2334-41
    • (2007) Blood , vol.110 , pp. 2334-2341
    • Mingozzi, F.1    Hasbrouck, N.C.2    Basner-Tschakarjan, E.3    Edmonson, S.A.4    Hui, D.J.5
  • 31
    • 0037988905 scopus 로고    scopus 로고
    • Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
    • Mingozzi F, Liu YL, Dobrzynski E, Kaufhold A, Liu JH, et al. 2003. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J. Clin. Investig. 111:1347-56
    • (2003) J. Clin. Investig , vol.111 , pp. 1347-1356
    • Mingozzi, F.1    Liu, Y.L.2    Dobrzynski, E.3    Kaufhold, A.4    Liu, J.H.5
  • 32
    • 34547498762 scopus 로고    scopus 로고
    • Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer
    • Cao O, Dobrzynski E, Wang L, Nayak S, Mingle B, et al. 2007. Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. Blood 110:1132-40
    • (2007) Blood , vol.110 , pp. 1132-1140
    • Cao, O.1    Dobrzynski, E.2    Wang, L.3    Nayak, S.4    Mingle, B.5
  • 33
    • 55849093638 scopus 로고    scopus 로고
    • Ectopic expression of neural autoantigen in mouse liver suppresses experimental autoimmune neuroinflammation by inducing antigen-specific Tregs
    • Lüth S, Huber S, Schramm C, Buch T, Zander S, et al. 2008. Ectopic expression of neural autoantigen in mouse liver suppresses experimental autoimmune neuroinflammation by inducing antigen-specific Tregs. J. Clin. Investig. 118:3403-10
    • (2008) J. Clin. Investig , vol.118 , pp. 3403-3410
    • Lüth, S.1    Huber, S.2    Schramm, C.3    Buch, T.4    Zander, S.5
  • 34
    • 2342561729 scopus 로고    scopus 로고
    • Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice
    • Follenzi A, Battaglia M, Lombardo A, Annoni A, Roncarolo MG, Naldini L. 2004. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood 103:3700-9
    • (2004) Blood , vol.103 , pp. 3700-3709
    • Follenzi, A.1    Battaglia, M.2    Lombardo, A.3    Annoni, A.4    Roncarolo, M.G.5    Naldini, L.6
  • 35
    • 84887110410 scopus 로고    scopus 로고
    • Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies
    • Markusic DM, Hoffman BE, Perrin GQ, Nayak S, Wang X, et al. 2013. Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies. EMBO Mol. Med. 5:1698-709
    • (2013) EMBO Mol. Med , vol.5 , pp. 1698-1709
    • Markusic, D.M.1    Hoffman, B.E.2    Perrin, G.Q.3    Nayak, S.4    Wang, X.5
  • 36
    • 85009728755 scopus 로고    scopus 로고
    • Alloantigen gene transfer to hepatocytes promotes tolerance to pancreatic islet graft by inducing CD8+ regulatory T cells
    • Le Guen V, Judor JP, Boeffard F, Gauttier V, Ferry N, et al. 2017. Alloantigen gene transfer to hepatocytes promotes tolerance to pancreatic islet graft by inducing CD8+ regulatory T cells. J. Hepatol. 66:765-77
    • (2017) J. Hepatol , vol.66 , pp. 765-777
    • Le Guen, V.1    Judor, J.P.2    Boeffard, F.3    Gauttier, V.4    Ferry, N.5
  • 37
    • 77950520150 scopus 로고    scopus 로고
    • BALB/c mice show impaired hepatic tolerogenic response following AAV gene transfer to the liver
    • Breous E, Somanathan S, Wilson JM. 2010. BALB/c mice show impaired hepatic tolerogenic response following AAV gene transfer to the liver. Mol. Ther. 18:766-74
    • (2010) Mol. Ther , vol.18 , pp. 766-774
    • Breous, E.1    Somanathan, S.2    Wilson, J.M.3
  • 38
    • 84979295330 scopus 로고    scopus 로고
    • Phase i open label liverdirected gene therapy clinical trial for acute intermittent porphyria
    • D'Avola D, Lopez-Franco E, Sangro B, Paneda A, Grossios N, et al. 2016. Phase I open label liverdirected gene therapy clinical trial for acute intermittent porphyria. J. Hepatol. 65:776-83
    • (2016) J. Hepatol , vol.65 , pp. 776-783
    • D'Avola, D.1    Lopez-Franco, E.2    Sangro, B.3    Paneda, A.4    Grossios, N.5
  • 39
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response
    • Manno CS, Arruda VR, Pierce GF, Glader B, Ragni M, et al. 2006. Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat. Med. 12:342-47
    • (2006) Nat. Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Arruda, V.R.2    Pierce, G.F.3    Glader, B.4    Ragni, M.5
  • 42
    • 68849103488 scopus 로고    scopus 로고
    • The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
    • Zhu J, Huang X, Yang Y. 2009. The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice. J. Clin. Investig. 119:2388-98
    • (2009) J. Clin. Investig , vol.119 , pp. 2388-2398
    • Zhu, J.1    Huang, X.2    Yang, Y.3
  • 43
    • 84864552366 scopus 로고    scopus 로고
    • MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV
    • Sudres M, Ciré S, Vasseur V, Brault L, Da Rocha S, et al. 2012. MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV. Mol. Ther. 20:1571-81
    • (2012) Mol. Ther , vol.20 , pp. 1571-1581
    • Sudres, M.1    Ciré, S.2    Vasseur, V.3    Brault, L.4    Da Rocha, S.5
  • 45
    • 79959201563 scopus 로고    scopus 로고
    • The genome of selfcomplementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
    • Martino AT, Suzuki M, Markusic DM, Zolotukhin I, Ryals RC, et al. 2011. The genome of selfcomplementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver. Blood 117:6459-68
    • (2011) Blood , vol.117 , pp. 6459-6468
    • Martino, A.T.1    Suzuki, M.2    Markusic, D.M.3    Zolotukhin, I.4    Ryals, R.C.5
  • 46
    • 84155178352 scopus 로고    scopus 로고
    • Toll-like receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors
    • Hösel M, Broxtermann M, Janicki H, Esser K, Arzberger S, et al. 2011. Toll-like receptor 2-mediated innate immune response in human nonparenchymal liver cells toward adeno-associated viral vectors. Hepatology 55:287-97
    • (2011) Hepatology , vol.55 , pp. 287-297
    • Hösel, M.1    Broxtermann, M.2    Janicki, H.3    Esser, K.4    Arzberger, S.5
  • 47
    • 0035138205 scopus 로고    scopus 로고
    • Intracellular trafficking of adeno-associated virus vectors: Routing to the late endosomal compartment and proteasome degradation
    • Douar AM, Poulard K, Stockholm D, Danos O. 2001. Intracellular trafficking of adeno-associated virus vectors: routing to the late endosomal compartment and proteasome degradation. J. Virol. 75:1824-33
    • (2001) J. Virol , vol.75 , pp. 1824-1833
    • Douar, A.M.1    Poulard, K.2    Stockholm, D.3    Danos, O.4
  • 48
    • 0036171821 scopus 로고    scopus 로고
    • Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors
    • Yan Z, Zak R, Luxton GWG, RitchieTC, Bantel-Schaal U, Engelhardt JF. 2002. Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors. J. Virol. 76:2043-53
    • (2002) J. Virol , vol.76 , pp. 2043-2053
    • Yan, Z.1    Zak, R.2    Luxton, G.W.G.3    Ritchie, T.C.4    Bantel-Schaal, U.5    Engelhardt, J.F.6
  • 49
    • 74149089236 scopus 로고    scopus 로고
    • Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation onMHCclass i following transduction
    • Finn JD, Hui D, Downey HD, Dunn D, Pien GC, et al. 2010. Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation onMHCclass I following transduction. Mol. Ther. 18:135-42
    • (2010) Mol. Ther , vol.18 , pp. 135-142
    • Finn, J.D.1    Hui, D.2    Downey, H.D.3    Dunn, D.4    Pien, G.C.5
  • 50
    • 33746801474 scopus 로고    scopus 로고
    • Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
    • Vandenberghe LH, Wang L, Somanathan S, Zhi Y, Figueredo J, et al. 2006. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat. Med. 12:967-71
    • (2006) Nat. Med , vol.12 , pp. 967-971
    • Vandenberghe, L.H.1    Wang, L.2    Somanathan, S.3    Zhi, Y.4    Figueredo, J.5
  • 51
    • 25144482280 scopus 로고    scopus 로고
    • Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery
    • Chadeuf G, Ciron C, Moullier P, Salvetti A. 2005. Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery. Mol. Ther. 12:744-53
    • (2005) Mol. Ther , vol.12 , pp. 744-753
    • Chadeuf, G.1    Ciron, C.2    Moullier, P.3    Salvetti, A.4
  • 52
    • 58149251820 scopus 로고    scopus 로고
    • Undetectable transcription of cap in a clinical AAV vector: Implications for preformed capsid in immune responses
    • Hauck B,Murphy SL, Smith PH, Qu G, Liu X, et al. 2009. Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses. Mol. Ther. 17:144-52
    • (2009) Mol. Ther , vol.17 , pp. 144-152
    • Hauck, B.1    Murphy, S.L.2    Smith, P.H.3    Qu, G.4    Liu, X.5
  • 53
    • 85022134428 scopus 로고    scopus 로고
    • SPK-9001: Adeno-associated virus mediated gene transfer for hemophilia B achieves sustained mean factor IX activity levels of >30% without immunosuppression
    • George LA, Sullivan SK, Giermasz A, Ducore JM, Teitel JM, et al. 2016. SPK-9001: adeno-associated virus mediated gene transfer for hemophilia B achieves sustained mean factor IX activity levels of >30% without immunosuppression. Blood 128:3
    • (2016) Blood , vol.128 , pp. 3
    • George, L.A.1    Sullivan, S.K.2    Giermasz, A.3    Ducore, J.M.4    Teitel, J.M.5
  • 54
    • 67649817113 scopus 로고    scopus 로고
    • Cellular immune response to cryptic epitopes during therapeutic gene transfer
    • Li C, Goudy K, Hirsch M, Asokan A, Fan Y, et al. 2009. Cellular immune response to cryptic epitopes during therapeutic gene transfer. PNAS 106:10770-74
    • (2009) PNAS , vol.106 , pp. 10770-10774
    • Li, C.1    Goudy, K.2    Hirsch, M.3    Asokan, A.4    Fan, Y.5
  • 55
    • 67449093069 scopus 로고    scopus 로고
    • 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, et al. 2009. Cytotoxic-T-lymphocyte-mediated elimination of target cells transduced with engineered adeno-associated virus type 2 vector in vivo. J. Virol. 83:6817-24
    • (2009) J. Virol , vol.83 , pp. 6817-6824
    • Li, C.1    Hirsch, M.2    DiPrimio, N.3    Asokan, A.4    Goudy, K.5
  • 56
    • 34247200575 scopus 로고    scopus 로고
    • Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets
    • Wang L, Figueredo J, Calcedo R, Lin J,Wilson JM. 2007. Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. Hum. Gene Ther. 18:185-94
    • (2007) Hum. Gene Ther , vol.18 , pp. 185-194
    • Wang, L.1    Figueredo, J.2    Calcedo, R.3    Lin, J.4    Wilson, J.M.5
  • 57
    • 74049120342 scopus 로고    scopus 로고
    • Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
    • Asokan A, Conway JC, Phillips JL, Li C, Hegge J, et al. 2010. Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle. Nat. Biotechnol. 28:79-82
    • (2010) Nat. Biotechnol , vol.28 , pp. 79-82
    • Asokan, A.1    Conway, J.C.2    Phillips, J.L.3    Li, C.4    Hegge, J.5
  • 58
    • 34447281279 scopus 로고    scopus 로고
    • Adeno-associated virus type 2 (AAV2) capsidspecific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo
    • Li C, Hirsch M, Asokan A, Zeithaml B,Ma H, et al. 2007. Adeno-associated virus type 2 (AAV2) capsidspecific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J. Virol. 81:7540-47
    • (2007) J. Virol , vol.81 , pp. 7540-7547
    • Li, C.1    Hirsch, M.2    Asokan, A.3    Zeithaml, B.4    Ma, H.5
  • 59
    • 84877930020 scopus 로고    scopus 로고
    • Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
    • Martino AT, Basner-Tschakarjan E, Markusic DM, Finn JD, Hinderer C, et al. 2013. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood 121:2224-33
    • (2013) Blood , vol.121 , pp. 2224-2233
    • Martino, A.T.1    Basner-Tschakarjan, E.2    Markusic, D.M.3    Finn, J.D.4    Hinderer, C.5
  • 60
    • 84891837069 scopus 로고    scopus 로고
    • CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome
    • Wu TL, Li H, Faust SM, Chi E, Zhou S, et al. 2014. CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome. Mol. Ther. 22:42-51
    • (2014) Mol. Ther , vol.22 , pp. 42-51
    • Wu, T.L.1    Li, H.2    Faust, S.M.3    Chi, E.4    Zhou, S.5
  • 61
    • 33646734488 scopus 로고    scopus 로고
    • Loss of Siglec expression on T lymphocytes during human evolution
    • Nguyen DH, Hurtado-Ziola N, Gagneux P, Varki A. 2006. Loss of Siglec expression on T lymphocytes during human evolution. PNAS 103:7765-70
    • (2006) PNAS , vol.103 , pp. 7765-7770
    • Nguyen, D.H.1    Hurtado-Ziola, N.2    Gagneux, P.3    Varki, A.4
  • 62
    • 80455174048 scopus 로고    scopus 로고
    • Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates
    • Li H, Lasaro MO, Jia B, Lin SW, Haut LH, et al. 2011. Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates. Mol. Ther. 19:2021-30
    • (2011) Mol. Ther , vol.19 , pp. 2021-2030
    • Li, H.1    Lasaro, M.O.2    Jia, B.3    Lin, S.W.4    Haut, L.H.5
  • 63
    • 67651006589 scopus 로고    scopus 로고
    • Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
    • Pien GC, Basner-Tschakarjan E, Hui DJ, Mentlik AN, Finn JD, et al. 2009. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J. Clin. Investig. 119:1688-95
    • (2009) J. Clin. Investig , vol.119 , pp. 1688-1695
    • Pien, G.C.1    Basner-Tschakarjan, E.2    Hui, D.J.3    Mentlik, A.N.4    Finn, J.D.5
  • 64
    • 11444268812 scopus 로고    scopus 로고
    • Preservation and redirection of HPV16E7-specific T cell receptors for immunotherapy of cervical cancer
    • Scholten KBJ, Schreurs MWJ, Ruizendaal JJ, Kueter EWM, Kramer D, et al. 2005. Preservation and redirection of HPV16E7-specific T cell receptors for immunotherapy of cervical cancer. Clin. Immunol. 114:119-29
    • (2005) Clin. Immunol , vol.114 , pp. 119-129
    • Scholten, K.B.J.1    Schreurs, M.W.J.2    Ruizendaal, J.J.3    Kueter, E.W.M.4    Kramer, D.5
  • 65
    • 84879683527 scopus 로고    scopus 로고
    • CpG-depleted adeno-associated virus vectors evade immune detection
    • Faust SM, Bell P, Cutler BJ, Ashley SN, Zhu Y, et al. 2013. CpG-depleted adeno-associated virus vectors evade immune detection. J. Clin. Investig. 123:2994-3001
    • (2013) J. Clin. Investig , vol.123 , pp. 2994-3001
    • Faust, S.M.1    Bell, P.2    Cutler, B.J.3    Ashley, S.N.4    Zhu, Y.5
  • 67
    • 84994875763 scopus 로고    scopus 로고
    • Neurological disease as a failure of brain-immune crosstalk: The multiple faces of neuroinflammation
    • Schwartz M, Deczkowska A. 2016. Neurological disease as a failure of brain-immune crosstalk: the multiple faces of neuroinflammation. Trends Immunol. 37:668-79
    • (2016) Trends Immunol , vol.37 , pp. 668-679
    • Schwartz, M.1    Deczkowska, A.2
  • 70
    • 84985994414 scopus 로고    scopus 로고
    • Postarrest stalling rather than crawling favors CD8+ over CD4+ T-cell migration across the blood-brain barrier under flow in vitro
    • Rudolph H, Klopstein A, Gruber I, Blatti C, Lyck R, Engelhardt B. 2016. Postarrest stalling rather than crawling favors CD8+ over CD4+ T-cell migration across the blood-brain barrier under flow in vitro. Eur. J. Immunol. 46:2187-203
    • (2016) Eur. J. Immunol , vol.46 , pp. 2187-2203
    • Rudolph, H.1    Klopstein, A.2    Gruber, I.3    Blatti, C.4    Lyck, R.5    Engelhardt, B.6
  • 71
    • 84988521082 scopus 로고    scopus 로고
    • Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: An ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
    • Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, et al. 2016. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet 388:476-87
    • (2016) Lancet , vol.388 , pp. 476-487
    • Sessa, M.1    Lorioli, L.2    Fumagalli, F.3    Acquati, S.4    Redaelli, D.5
  • 72
    • 84879873039 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
    • Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, et al. 2013. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341:1233158
    • (2013) Science , vol.341 , pp. 1233158
    • Biffi, A.1    Montini, E.2    Lorioli, L.3    Cesani, M.4    Fumagalli, F.5
  • 74
    • 34250683023 scopus 로고    scopus 로고
    • Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase i trial
    • Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, et al. 2007. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369:2097-105
    • (2007) Lancet , vol.369 , pp. 2097-2105
    • Kaplitt, M.G.1    Feigin, A.2    Tang, C.3    Fitzsimons, H.L.4    Mattis, P.5
  • 77
    • 44349136847 scopus 로고    scopus 로고
    • Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA
    • Worgall S, Sondhi D, Hackett NR, Kosofsky B, Kekatpure MV, et al. 2008. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum. Gene Ther. 19:463-74
    • (2008) Hum. Gene Ther , vol.19 , pp. 463-474
    • Worgall, S.1    Sondhi, D.2    Hackett, N.R.3    Kosofsky, B.4    Kekatpure, M.V.5
  • 79
    • 79551632875 scopus 로고    scopus 로고
    • Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes
    • Ellinwood NM, Ausseil J, Desmaris N, Bigou S, Liu S, et al. 2011. Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes. Mol. Ther. 19:251-59
    • (2011) Mol. Ther , vol.19 , pp. 251-259
    • Ellinwood, N.M.1    Ausseil, J.2    Desmaris, N.3    Bigou, S.4    Liu, S.5
  • 80
    • 84881228888 scopus 로고    scopus 로고
    • Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
    • Haurigot V, Marćo S, Ribera A, Garcia M, Ruzo A, et al. 2013. Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. J. Clin. Investig. 123:3254-71
    • (2013) J. Clin. Investig , vol.123 , pp. 3254-3271
    • Haurigot, V.1    Marćo, S.2    Ribera, A.3    Garcia, M.4    Ruzo, A.5
  • 81
    • 32944459983 scopus 로고    scopus 로고
    • Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
    • Cearley CN, Wolfe JH. 2006. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther. 13:528-37
    • (2006) Mol Ther , vol.13 , pp. 528-537
    • Cearley, C.N.1    Wolfe, J.H.2
  • 83
    • 67649861393 scopus 로고    scopus 로고
    • Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
    • Duque S, Joussemet B, Rivière C, Marais T, Dubreil L, et al. 2009. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol. Ther. 17:1187-96
    • (2009) Mol. Ther , vol.17 , pp. 1187-1196
    • Duque, S.1    Joussemet, B.2    Rivière, C.3    Marais, T.4    Dubreil, L.5
  • 84
    • 84957588611 scopus 로고    scopus 로고
    • Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
    • Deverman BE, Pravdo PL, Simpson BP, Kumar SR, Chan KY, et al. 2016. Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain. Nat. Biotechnol. 34:204-9
    • (2016) Nat. Biotechnol , vol.34 , pp. 204-209
    • Deverman, B.E.1    Pravdo, P.L.2    Simpson, B.P.3    Kumar, S.R.4    Chan, K.Y.5
  • 85
    • 84959091629 scopus 로고    scopus 로고
    • Exosome-associated AAV vector as a robust and convenient neuroscience tool
    • Hudry E, Martin C, Gandhi S, György B, Scheffer DI, et al. 2016. Exosome-associated AAV vector as a robust and convenient neuroscience tool. Gene Ther. 23:380-92
    • (2016) Gene Ther , vol.23 , pp. 380-392
    • Hudry, E.1    Martin, C.2    Gandhi, S.3    György, B.4    Scheffer, D.I.5
  • 86
    • 85030321404 scopus 로고    scopus 로고
    • Deleted in proof
    • Deleted in proof
  • 87
    • 54949097323 scopus 로고    scopus 로고
    • Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer
    • Foster H, Sharp PS, Athanasopoulos T, Trollet C, Graham IR, et al. 2008. Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer. Mol. Ther. 16:1825-32
    • (2008) Mol. Ther , vol.16 , pp. 1825-1832
    • Foster, H.1    Sharp, P.S.2    Athanasopoulos, T.3    Trollet, C.4    Graham, I.R.5
  • 88
    • 0036099319 scopus 로고    scopus 로고
    • Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors
    • Fraites TJ Jr., Schleissing MR, Shanely RA, Walter GA, Cloutier DA, et al. 2002. Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors. Mol. Ther. 5:571-78
    • (2002) Mol. Ther , vol.5 , pp. 571-578
    • Fraites, T.J.1    Schleissing, M.R.2    Shanely, R.A.3    Walter, G.A.4    Cloutier, D.A.5
  • 89
    • 27944479711 scopus 로고    scopus 로고
    • Efficient in vivo gene expression by transsplicing adeno-associated viral vectors
    • Lai Y, Yue Y, Liu M, Ghosh A, Engelhardt JF, et al. 2005. Efficient in vivo gene expression by transsplicing adeno-associated viral vectors. Nat. Biotechnol. 23:1435-39
    • (2005) Nat. Biotechnol , vol.23 , pp. 1435-1439
    • Lai, Y.1    Yue, Y.2    Liu, M.3    Ghosh, A.4    Engelhardt, J.F.5
  • 90
    • 84964313862 scopus 로고    scopus 로고
    • Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients
    • Le Guiner C, Montus M, Servais L, Chérel Y, Francois V, et al. 2014. Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients. Mol. Ther. 22:1923-35
    • (2014) Mol. Ther , vol.22 , pp. 1923-1935
    • Le Guiner, C.1    Montus, M.2    Servais, L.3    Chérel, Y.4    Francois, V.5
  • 91
    • 77952502008 scopus 로고    scopus 로고
    • Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer
    • LostalW,BartoliM,BourgN,Roudaut C, Bentaib A, et al. 2010. Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer. Hum. Mol. Genet. 19:1897-907
    • (2010) Hum. Mol. Genet , vol.19 , pp. 1897-1907
    • Lostal, W.1    Bartoli, M.2    Bourg, N.3    Roudaut, C.4    Bentaib, A.5
  • 92
    • 17544383607 scopus 로고    scopus 로고
    • Gene transfer into skeletal muscle using novel AAV serotypes
    • Louboutin JP,Wang L,Wilson JM. 2005. Gene transfer into skeletal muscle using novel AAV serotypes. J. Gene Med. 7:442-51
    • (2005) J. Gene Med , vol.7 , pp. 442-451
    • Louboutin, J.P.1    Wang, L.2    Wilson, J.M.3
  • 93
    • 33646933211 scopus 로고    scopus 로고
    • Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation
    • Ross CJD, Twisk J, Bakker AC, Miao F, Verbart D, et al. 2006. Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation. Hum. Gene Ther. 17:487-99
    • (2006) Hum. Gene Ther , vol.17 , pp. 487-499
    • Ross, C.J.D.1    Twisk, J.2    Bakker, A.C.3    Miao, F.4    Verbart, D.5
  • 94
    • 13144305062 scopus 로고    scopus 로고
    • Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors
    • Song S, Morgan M, Ellis T, Poirier A, Chesnut K, et al. 1998. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. PNAS 95:14384-88
    • (1998) PNAS , vol.95 , pp. 14384-14388
    • Song, S.1    Morgan, M.2    Ellis, T.3    Poirier, A.4    Chesnut, K.5
  • 95
    • 0034610364 scopus 로고    scopus 로고
    • Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
    • Wang B, Li J, Xiao X. 2000. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. PNAS 97:13714-19
    • (2000) PNAS , vol.97 , pp. 13714-13719
    • Wang, B.1    Li, J.2    Xiao, X.3
  • 96
    • 33745894322 scopus 로고    scopus 로고
    • RAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
    • Gregorevic P, Allen JM, Minami E, BlankinshipMJ, HaraguchiM, et al. 2006. RAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat. Med. 12:787-89
    • (2006) Nat. Med , vol.12 , pp. 787-789
    • Gregorevic, P.1    Allen, J.M.2    Minami, E.3    Blankinship, M.J.4    Haraguchi, M.5
  • 97
    • 0030451214 scopus 로고    scopus 로고
    • Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein
    • Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, et al. 1996. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. PNAS 93:14082-87
    • (1996) PNAS , vol.93 , pp. 14082-14087
    • Kessler, P.D.1    Podsakoff, G.M.2    Chen, X.3    McQuiston, S.A.4    Colosi, P.C.5
  • 98
    • 0029843596 scopus 로고    scopus 로고
    • Efficient long-term gene transfer intomuscle tissue of immunocompetent mice by adeno-associated virus vector
    • Xiao X, Li J, Samulski RJ. 1996. Efficient long-term gene transfer intomuscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70:8098-108
    • (1996) J. Virol , vol.70 , pp. 8098-8108
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 99
    • 0030902985 scopus 로고    scopus 로고
    • Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
    • Herzog RW,Hagstrom JN, Kung SH,Tai SJ,Wilson JM, et al. 1997. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. PNAS 94:5804-9
    • (1997) PNAS , vol.94 , pp. 5804-5809
    • Herzog, R.W.1    Hagstrom, J.N.2    Kung, S.H.3    Tai, S.J.4    Wilson, J.M.5
  • 100
    • 84997294339 scopus 로고    scopus 로고
    • Long-term retrospective analysis of gene therapywith alipogene tiparvovec and its effect on lipoprotein lipase deficiency-induced pancreatitis
    • Gaudet D, Stroes ES, Methot J, Brisson D, Tremblay K, et al. 2016. Long-term retrospective analysis of gene therapywith alipogene tiparvovec and its effect on lipoprotein lipase deficiency-induced pancreatitis. Hum. Gene Ther. 27:916-25
    • (2016) Hum. Gene Ther , vol.27 , pp. 916-925
    • Gaudet, D.1    Stroes, E.S.2    Methot, J.3    Brisson, D.4    Tremblay, K.5
  • 101
    • 84890054617 scopus 로고    scopus 로고
    • Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression
    • Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, et al. 2013. Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression. J. Clin. Investig. 123:5310-18
    • (2013) J. Clin. Investig , vol.123 , pp. 5310-5318
    • Mueller, C.1    Chulay, J.D.2    Trapnell, B.C.3    Humphries, M.4    Carey, B.5
  • 102
    • 84859198455 scopus 로고    scopus 로고
    • Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
    • Buchlis G, Podsakoff GM, Radu A, Hawk SM, Flake AW, et al. 2012. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer. Blood 119:3038-41
    • (2012) Blood , vol.119 , pp. 3038-3041
    • Buchlis, G.1    Podsakoff, G.M.2    Radu, A.3    Hawk, S.M.4    Flake, A.W.5
  • 103
    • 85016950473 scopus 로고    scopus 로고
    • Systemic AAV8-mediated gene therapy drives whole-body correction of myotubular myopathy in dogs
    • Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, et al. 2017. Systemic AAV8-mediated gene therapy drives whole-body correction of myotubular myopathy in dogs. Mol. Ther. 25:839-54
    • (2017) Mol. Ther , vol.25 , pp. 839-854
    • Mack, D.L.1    Poulard, K.2    Goddard, M.A.3    Latournerie, V.4    Snyder, J.M.5
  • 105
    • 85030311944 scopus 로고    scopus 로고
    • Adeno-associated virus vector (AAV)microdystrophin gene therapy prolongs survival and restores muscle function in the caninemodel of Duchenne muscular dystrophy (DMD)
    • Adeno-associated virus vector (AAV)microdystrophin gene therapy prolongs survival and restores muscle function in the caninemodel of Duchenne muscular dystrophy (DMD). Mol. Ther. 24:S200
    • Mol. Ther , vol.24 , pp. S200
  • 106
    • 0037066427 scopus 로고    scopus 로고
    • The danger model: A renewed sense of self
    • Matzinger P. 2002. The danger model: a renewed sense of self. Science 296:301-5
    • (2002) Science , vol.296 , pp. 301-305
    • Matzinger, P.1
  • 108
    • 77954244683 scopus 로고    scopus 로고
    • Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs
    • Haurigot V, Mingozzi F, Buchlis G, Hui DJ, Chen Y, et al. 2010. Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. Mol. Ther. 18:1318-29
    • (2010) Mol. Ther , vol.18 , pp. 1318-1329
    • Haurigot, V.1    Mingozzi, F.2    Buchlis, G.3    Hui, D.J.4    Chen, Y.5
  • 109
    • 74149084765 scopus 로고    scopus 로고
    • Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle
    • Toromanoff A, Adjali O, Larcher T, Hill M, Guigand L, et al. 2010. Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle. Mol. Ther. 18:151-60
    • (2010) Mol. Ther , vol.18 , pp. 151-160
    • Toromanoff, A.1    Adjali, O.2    Larcher, T.3    Hill, M.4    Guigand, L.5
  • 110
    • 0036376642 scopus 로고    scopus 로고
    • Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy
    • Herzog RW, Fields PA, Arruda VR, Brubaker JO, Armstrong E, et al. 2002. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum. Gene Ther. 13:1281-91
    • (2002) Hum. Gene Ther , vol.13 , pp. 1281-1291
    • Herzog, R.W.1    Fields, P.A.2    Arruda, V.R.3    Brubaker, J.O.4    Armstrong, E.5
  • 111
    • 84892726816 scopus 로고    scopus 로고
    • Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B
    • Rogers GL, Martino AT, Zolotukhin I, Ertl HC, Herzog RW. 2014. Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B. J. Transl. Med. 12:25
    • (2014) J. Transl. Med , vol.12 , pp. 25
    • Rogers, G.L.1    Martino, A.T.2    Zolotukhin, I.3    Ertl, H.C.4    Herzog, R.W.5
  • 112
    • 71649099089 scopus 로고    scopus 로고
    • Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants
    • Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, et al. 2010. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol. Genet. Metab. 99:26-33
    • (2010) Mol. Genet. Metab , vol.99 , pp. 26-33
    • Kishnani, P.S.1    Goldenberg, P.C.2    DeArmey, S.L.3    Heller, J.4    Benjamin, D.5
  • 113
    • 85015239107 scopus 로고    scopus 로고
    • Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice
    • Falk DJ, Soustek MS, Todd AG, Mah CS, Cloutier DA, et al. 2015. Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice. Mol. Ther.Methods Clin. Dev. 2:15007
    • (2015) Mol. Ther.Methods Clin. Dev , vol.2 , pp. 15007
    • Falk, D.J.1    Soustek, M.S.2    Todd, A.G.3    Mah, C.S.4    Cloutier, D.A.5
  • 114
    • 69949160511 scopus 로고    scopus 로고
    • AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsidspecific T cells
    • Mingozzi F, Meulenberg JJ, Hui DJ, Basner-Tschakarjan E, Hasbrouck NC, et al. 2009. AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsidspecific T cells. Blood 114:2077-86
    • (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
  • 115
    • 84920677763 scopus 로고    scopus 로고
    • A phase 1/2a follistatin gene therapy trial for Becker muscular dystrophy
    • Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, et al. 2015. A phase 1/2a follistatin gene therapy trial for Becker muscular dystrophy. Mol. Ther. 23:192-201
    • (2015) Mol. Ther , vol.23 , pp. 192-201
    • Mendell, J.R.1    Sahenk, Z.2    Malik, V.3    Gomez, A.M.4    Flanigan, K.M.5
  • 116
    • 84896320924 scopus 로고    scopus 로고
    • Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL S447X) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy
    • Ferreira V, Twisk J, Kwikkers K, Aronica E, Brisson D, et al. 2014. Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL S447X) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy. Hum. Gene Ther. 25:180-88
    • (2014) Hum. Gene Ther , vol.25 , pp. 180-188
    • Ferreira, V.1    Twisk, J.2    Kwikkers, K.3    Aronica, E.4    Brisson, D.5
  • 117
    • 84897932793 scopus 로고    scopus 로고
    • Immune responses to AAV-vectors, the Glybera example from bench to bedside
    • Ferreira V, Petry H, Salmon F. 2014. Immune responses to AAV-vectors, the Glybera example from bench to bedside. Front. Immunol. 5:82
    • (2014) Front. Immunol , vol.5 , pp. 82
    • Ferreira, V.1    Petry, H.2    Salmon, F.3
  • 118
    • 70349481529 scopus 로고    scopus 로고
    • Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
    • BrantlyML, Chulay JD, Wang L, Mueller C,Humphries M, et al. 2009. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. PNAS 106:16363-68
    • (2009) PNAS , vol.106 , pp. 16363-16368
    • Brantly, M.L.1    Chulay, J.D.2    Wang, L.3    Mueller, C.4    Humphries, M.5
  • 119
    • 80155145248 scopus 로고    scopus 로고
    • Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing 1-antitrypsin: Interim results
    • Flotte TR, Trapnell BC, Humphries M, Carey B, Calcedo R, et al. 2011. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing 1-antitrypsin: interim results. Hum. Gene Ther. 22:1239-47
    • (2011) Hum. Gene Ther , vol.22 , pp. 1239-1247
    • Flotte, T.R.1    Trapnell, B.C.2    Humphries, M.3    Carey, B.4    Calcedo, R.5
  • 120
    • 85013074473 scopus 로고    scopus 로고
    • Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for 1-antitrypsin deficiency
    • Calcedo R, Somanathan S, Qin Q, Betts MR, Rech AJ, et al. 2017. Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for 1-antitrypsin deficiency. PNAS 114:1655-59
    • (2017) PNAS , vol.114 , pp. 1655-1659
    • Calcedo, R.1    Somanathan, S.2    Qin, Q.3    Betts, M.R.4    Rech, A.J.5
  • 121
    • 60249095128 scopus 로고    scopus 로고
    • Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy
    • Velazquez VM, Bowen DG, Walker CM. 2009. Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy. Blood 113:538-45
    • (2009) Blood , vol.113 , pp. 538-545
    • Velazquez, V.M.1    Bowen, D.G.2    Walker, C.M.3
  • 122
    • 80052497157 scopus 로고    scopus 로고
    • Adeno-associated virus antibody profiles in newborns, children, and adolescents
    • Calcedo R, Morizono H, Wang L, McCarter R, He J, et al. 2011. Adeno-associated virus antibody profiles in newborns, children, and adolescents. Clin. Vaccine Immunol. 18:1586-88
    • (2011) Clin. Vaccine Immunol , vol.18 , pp. 1586-1588
    • Calcedo, R.1    Morizono, H.2    Wang, L.3    McCarter, R.4    He, J.5
  • 123
    • 0345411588 scopus 로고    scopus 로고
    • 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. 59:406-11
    • (1999) J. Med. Virol , vol.59 , pp. 406-411
    • Erles, K.1    Sebokova, P.2    Schlehofer, J.R.3
  • 124
    • 84858005636 scopus 로고    scopus 로고
    • Neutralizing antibodies against adenoassociated virus examined prospectively in pediatric patients with hemophilia
    • Li C, NarkbunnamN, Samulski RJ, Asokan A,Hu G, et al. 2011. Neutralizing antibodies against adenoassociated virus examined prospectively in pediatric patients with hemophilia. Gene Ther. 19:288-94
    • (2011) Gene Ther , vol.19 , pp. 288-294
    • Li, C.1    Narkbunnam, N.2    Samulski, R.J.3    Asokan, A.4    Hu, G.5
  • 125
    • 0037015049 scopus 로고    scopus 로고
    • 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. PNAS 99:11854-59
    • (2002) PNAS , 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
  • 126
    • 77954976233 scopus 로고    scopus 로고
    • 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, et al. 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. 21:704-12
    • (2010) Hum. Gene Ther , vol.21 , pp. 704-712
    • Boutin, S.1    Monteilhet, V.2    Veron, P.3    Leborgne, C.4    Benveniste, O.5
  • 127
    • 33751181882 scopus 로고    scopus 로고
    • 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, et al. 2006. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 108:3321-28
    • (2006) Blood , vol.108 , pp. 3321-3328
    • Jiang, H.1    Couto, L.B.2    Patarroyo-White, S.3    Liu, T.4    Nagy, D.5
  • 128
    • 33344466244 scopus 로고    scopus 로고
    • Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCIDmice
    • Scallan CD, Jiang H, Liu T, Patarroyo-White S, Sommer JM, et al. 2006. Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCIDmice. Blood 107:1810-17
    • (2006) Blood , vol.107 , pp. 1810-1817
    • Scallan, C.D.1    Jiang, H.2    Liu, T.3    Patarroyo-White, S.4    Sommer, J.M.5
  • 129
    • 84928168850 scopus 로고    scopus 로고
    • Determination of antiadeno-associated virus vector neutralizing antibody titer with an in vitro reporter system
    • Meliani A, Leborgne C, Triffault S, Jeanson-Leh L, Veron P, Mingozzi F. 2015. Determination of antiadeno-associated virus vector neutralizing antibody titer with an in vitro reporter system. Hum. Gene Ther. Methods 26:45-53
    • (2015) Hum. Gene Ther. Methods , vol.26 , pp. 45-53
    • Meliani, A.1    Leborgne, C.2    Triffault, S.3    Jeanson-Leh, L.4    Veron, P.5    Mingozzi, F.6
  • 130
    • 0013442744 scopus 로고    scopus 로고
    • 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, et al. 2002. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101:2963-72
    • (2002) 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
  • 131
    • 84931466179 scopus 로고    scopus 로고
    • Gene therapy in an era of emerging treatment options for hemophilia B
    • Monahan PE. 2015. Gene therapy in an era of emerging treatment options for hemophilia B. J. Thromb. Haemost. 13(Suppl. 1):S151-60
    • (2015) J. Thromb. Haemost , vol.13 , pp. S151-S160
    • Monahan, P.E.1
  • 132
    • 85006791028 scopus 로고    scopus 로고
    • Updated results from a dose escalating study in adult patients with haemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy
    • Miesbach W, Tangelder M, Klamroth R, Schutgens R, Coppens M, et al. 2016. Updated results from a dose escalating study in adult patients with haemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy. Haemophilia 22(Suppl. 4):151-52
    • (2016) Haemophilia , vol.22 , pp. 151-152
    • Miesbach, W.1    Tangelder, M.2    Klamroth, R.3    Schutgens, R.4    Coppens, M.5
  • 134
    • 85011356650 scopus 로고    scopus 로고
    • Interim results of an open-label, phase 1/2 study of BMN 270, an AAV5-FVIII gene transfer in severe hemophilia A
    • Pasi J, Wong W, Rangarajan S. 2016. Interim results of an open-label, phase 1/2 study of BMN 270, an AAV5-FVIII gene transfer in severe hemophilia A. Haemophilia 22(Suppl. 4):151-52
    • (2016) Haemophilia , vol.22 , pp. 151-152
    • Pasi, J.1    Wong, W.2    Rangarajan, S.3
  • 135
    • 84895910861 scopus 로고    scopus 로고
    • Plasmapheresis eliminates the negative impact of aav antibodies on microdystrophin gene expression following vascular delivery
    • Chicoine LG, Montgomery CL, Bremer WG, Shontz KM, Griffin DA, et al. 2014. Plasmapheresis eliminates the negative impact of aav antibodies on microdystrophin gene expression following vascular delivery. Mol. Ther. 22:338-47
    • (2014) Mol. Ther , vol.22 , pp. 338-347
    • Chicoine, L.G.1    Montgomery, C.L.2    Bremer, W.G.3    Shontz, K.M.4    Griffin, D.A.5
  • 136
    • 80455155171 scopus 로고    scopus 로고
    • A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8
    • Monteilhet V, Saheb S, Boutin S, Leborgne C, Veron P, et al. 2011. A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8. Mol. Ther. 19:2084-91
    • (2011) Mol. Ther , vol.19 , pp. 2084-2091
    • Monteilhet, V.1    Saheb, S.2    Boutin, S.3    Leborgne, C.4    Veron, P.5
  • 138
    • 84863480768 scopus 로고    scopus 로고
    • Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B
    • Mingozzi F, Chen Y, Murphy SL, Edmonson SC, Tai A, et al. 2012. Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. Mol. Ther. 20:1410-16
    • (2012) Mol. Ther , vol.20 , pp. 1410-1416
    • Mingozzi, F.1    Chen, Y.2    Murphy, S.L.3    Edmonson, S.C.4    Tai, A.5
  • 139
    • 84862158942 scopus 로고    scopus 로고
    • Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates
    • Unzu C, Hervás-Stubbs S, Sampedro A, Mauleon I, Mancheno U, et al. 2012. Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates. J. Transl. Med. 10:122
    • (2012) J. Transl. Med , vol.10 , pp. 122
    • Unzu, C.1    Hervás-Stubbs, S.2    Sampedro, A.3    Mauleon, I.4    Mancheno, U.5
  • 141
    • 84875912760 scopus 로고    scopus 로고
    • Prevalence and pharmacological modulation of humoral immunity toAAVvectors in gene transfer to synovial tissue
    • Mingozzi F, Chen Y, Edmonson SC, Zhou S,Thurlings RM, et al. 2013. Prevalence and pharmacological modulation of humoral immunity toAAVvectors in gene transfer to synovial tissue. Gene Ther. 20:417-24
    • (2013) Gene Ther , vol.20 , pp. 417-424
    • Mingozzi, F.1    Chen, Y.2    Edmonson, S.C.3    Zhou, S.4    Thurlings, R.M.5
  • 142
    • 84873337445 scopus 로고    scopus 로고
    • 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, et al. 2013. Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors. Mol. Ther. 21:318-23
    • (2013) Mol. Ther , vol.21 , pp. 318-323
    • Mimuro, J.1    Mizukami, H.2    Hishikawa, S.3    Ikemoto, T.4    Ishiwata, A.5


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