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Volumn 27, Issue 1, 2016, Pages 1-12

Expressing transgenes that exceed the packaging capacity of adeno-associated virus capsids

Author keywords

[No Author keywords available]

Indexed keywords

PARVOVIRUS VECTOR; PROTEIN; CAPSID PROTEIN; VIRUS DNA;

EID: 84959127834     PISSN: 19466536     EISSN: 19466544     Source Type: Journal    
DOI: 10.1089/hgtb.2015.140     Document Type: Review
Times cited : (108)

References (72)
  • 1
    • 84871991715 scopus 로고    scopus 로고
    • Gene therapy enters the pharma market: The short story of a long journey
    • Buning H. Gene therapy enters the pharma market: The short story of a long journey. EMBO Mol Med 2013; 5: 1-3
    • (2013) EMBO Mol Med , vol.5 , pp. 1-3
    • Buning, H.1
  • 2
    • 0013914098 scopus 로고
    • Studies of small DNA viruses found in various adenovirus preparations: Physical, biological, and immunological characteristics
    • Hoggan MD, Blacklow NR, Rowe WP. Studies of small DNA viruses found in various adenovirus preparations: Physical, biological, and immunological characteristics. Proc Natl Acad Sci U S A 1966; 55: 1467-1474
    • (1966) Proc Natl Acad Sci U S A , vol.55 , pp. 1467-1474
    • Hoggan, M.D.1    Blacklow, N.R.2    Rowe, W.P.3
  • 3
    • 84942905508 scopus 로고    scopus 로고
    • Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
    • Nault JC, Datta S, Imbeaud S, et al. Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas. Nat Genet 2015; 47: 1187-1193
    • (2015) Nat Genet , vol.47 , pp. 1187-1193
    • Nault, J.C.1    Datta, S.2    Imbeaud, S.3
  • 4
    • 0001592496 scopus 로고
    • Adenovirus-Associated defective virus particles
    • Atchison RW, Casto BC, Hammon WM. Adenovirus-Associated defective virus particles. Science 1965; 149: 754
    • (1965) Science , vol.149 , pp. 754
    • Atchison, R.W.1    Casto, B.C.2    Hammon, W.M.3
  • 5
    • 85016968998 scopus 로고    scopus 로고
    • AAV-mediated gene therapy for research and therapeutic purposes
    • Samulski RJ, Muzyczka N. AAV-mediated gene therapy for research and therapeutic purposes. Ann Rev Virol 2014; 1: 427-451
    • (2014) Ann Rev Virol , vol.1 , pp. 427-451
    • Samulski, R.J.1    Muzyczka, N.2
  • 6
    • 0015288898 scopus 로고
    • Separation of two types of adeno-Associated virus particles containing complementary polynucleotide chains
    • Berns KI, Adler S. Separation of two types of adeno-Associated virus particles containing complementary polynucleotide chains. J Virol 1972; 9: 394-396
    • (1972) J Virol , vol.9 , pp. 394-396
    • Berns, K.I.1    Adler, S.2
  • 7
    • 0014886349 scopus 로고
    • Adenovirus associated viruses
    • Hoggan MD. Adenovirus associated viruses. Prog Med Virol 1970; 12: 211-239
    • (1970) Prog Med Virol , vol.12 , pp. 211-239
    • Hoggan, M.D.1
  • 8
    • 0014601124 scopus 로고
    • Evidence for a single-stranded adenovirus-Associated virus genome: Formation of a DNA density hybrid on release of viral DNA
    • Rose JA, Berns KI, Hoggan MD, et al. Evidence for a single-stranded adenovirus-Associated virus genome: Formation of a DNA density hybrid on release of viral DNA. Proc Natl Acad Sci U S A 1969; 64: 863-869
    • (1969) Proc Natl Acad Sci U S A , vol.64 , pp. 863-869
    • Rose, J.A.1    Berns, K.I.2    Hoggan, M.D.3
  • 10
    • 0029858907 scopus 로고    scopus 로고
    • Site-specific integration by adeno-Associated virus
    • Linden RM, Ward P, Giraud C, et al. Site-specific integration by adeno-Associated virus. Proc Natl Acad Sci U S A 1996; 93: 11288-11294
    • (1996) Proc Natl Acad Sci U S A , vol.93 , pp. 11288-11294
    • Linden, R.M.1    Ward, P.2    Giraud, C.3
  • 11
    • 0026070440 scopus 로고
    • Targeted integration of adeno-Associated virus (AAV) into human chromosome 19
    • Samulski RJ, Zhu X, Xiao X, et al. Targeted integration of adeno-Associated virus (AAV) into human chromosome 19. EMBO J 1991; 10: 3941-3950
    • (1991) EMBO J , vol.10 , pp. 3941-3950
    • Samulski, R.J.1    Zhu, X.2    Xiao, X.3
  • 12
    • 0021228018 scopus 로고
    • Genetic analysis of adeno-Associated virus: Properties of deletion mutants constructed in vitro and evidence for an adeno-Associated virus replication function
    • Tratschin JD, Miller IL, Carter BJ. Genetic analysis of adeno-Associated virus: Properties of deletion mutants constructed in vitro and evidence for an adeno-Associated virus replication function. J Virol 1984; 51: 611-619
    • (1984) J Virol , vol.51 , pp. 611-619
    • Tratschin, J.D.1    Miller, I.L.2    Carter, B.J.3
  • 13
    • 0030970938 scopus 로고    scopus 로고
    • Recombinant adeno-Associated viral vectors mediate long-Term transgene expression in muscle
    • Clark KR, Sferra TJ, Johnson PR. Recombinant adeno-Associated viral vectors mediate long-Term transgene expression in muscle. Hum Gene Ther 1997; 8: 659-669
    • (1997) Hum Gene Ther , vol.8 , pp. 659-669
    • Clark, K.R.1    Sferra, T.J.2    Johnson, P.R.3
  • 14
    • 0031691257 scopus 로고    scopus 로고
    • Circular intermediates of recombinant adeno-Associated virus have defined structural characteristics responsible for long-Term episomal persistence in muscle tissue
    • Duan D, Sharma P, Yang J, et al. Circular intermediates of recombinant adeno-Associated virus have defined structural characteristics responsible for long-Term episomal persistence in muscle tissue. J Virol 1998; 72: 8568-8577
    • (1998) J Virol , vol.72 , pp. 8568-8577
    • Duan, D.1    Sharma, P.2    Yang, J.3
  • 15
    • 0033814178 scopus 로고    scopus 로고
    • Recruitment of single-stranded recombinant adeno-Associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo
    • Nakai H, Storm TA, Kay MA. Recruitment of single-stranded recombinant adeno-Associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J Virol 2000; 74: 9451-9463
    • (2000) J Virol , vol.74 , pp. 9451-9463
    • Nakai, H.1    Storm, T.A.2    Kay, M.A.3
  • 16
    • 27744584507 scopus 로고    scopus 로고
    • Characterization of adeno-Associated virus genomes isolated from human tissues
    • Schnepp BC, Jensen RL, Chen CL, et al. Characterization of adeno-Associated virus genomes isolated from human tissues. J Virol 2005; 79: 14793-14803
    • (2005) J Virol , vol.79 , pp. 14793-14803
    • Schnepp, B.C.1    Jensen, R.L.2    Chen, C.L.3
  • 17
    • 84861998784 scopus 로고    scopus 로고
    • Intracellular transport of recombinant adeno-Associated virus vectors
    • Nonnenmacher M, Weber T. Intracellular transport of recombinant adeno-Associated virus vectors. Gene Ther 2012; 19: 649-658
    • (2012) Gene Ther , vol.19 , pp. 649-658
    • Nonnenmacher, M.1    Weber, T.2
  • 18
    • 0018848777 scopus 로고
    • Molecular structure of adeno-Associated virus variant DNA
    • de la Maza LM, Carter BJ. Molecular structure of adeno-Associated virus variant DNA. J Biol Chem 1980; 255: 3194-3203
    • (1980) J Biol Chem , vol.255 , pp. 3194-3203
    • De La Maza, L.M.1    Carter, B.J.2
  • 19
    • 0020684648 scopus 로고
    • Nucleotide sequence and organization of the adenoassociated virus 2 genome
    • Srivastava A, Lusby EW, Berns KI. Nucleotide sequence and organization of the adenoassociated virus 2 genome. J Virol 1983; 45: 555-564
    • (1983) J Virol , vol.45 , pp. 555-564
    • Srivastava, A.1    Lusby, E.W.2    Berns, K.I.3
  • 20
    • 0015909360 scopus 로고
    • Arrangement of nucleotide sequences in adeno-Associated virus DNA
    • Gerry HW, Kelly TJ Jr., Berns KI. Arrangement of nucleotide sequences in adeno-Associated virus DNA. J Mol Biol 1973; 79: 207-225
    • (1973) J Mol Biol , vol.79 , pp. 207-225
    • Gerry, H.W.1    Kelly, T.J.2    Berns, K.I.3
  • 21
    • 84906309321 scopus 로고    scopus 로고
    • Isolation of a goose parvovirus from swan and its molecular characteristics
    • Shao H, Lv Y, Ye J, et al. Isolation of a goose parvovirus from swan and its molecular characteristics. Acta Virol 2014; 58: 194-198
    • (2014) Acta Virol , vol.58 , pp. 194-198
    • Shao, H.1    Lv, Y.2    Ye, J.3
  • 22
    • 0021989577 scopus 로고
    • Equine parvovirus: Initial isolation and partial characterization
    • Wong FC, Spearman JG, Smolenski MA, et al. Equine parvovirus: Initial isolation and partial characterization. Can J Comp Med 1985; 49: 50-54
    • (1985) Can J Comp Med , vol.49 , pp. 50-54
    • Wong, F.C.1    Spearman, J.G.2    Smolenski, M.A.3
  • 23
    • 0027224024 scopus 로고
    • The complete nucleotide sequence of parvovirus LuIII and localization of a unique sequence possibly responsible for its encapsidation pattern
    • Diffoot N, Chen KC, Bates RC, et al. The complete nucleotide sequence of parvovirus LuIII and localization of a unique sequence possibly responsible for its encapsidation pattern. Virology 1993; 192: 339-345
    • (1993) Virology , vol.192 , pp. 339-345
    • Diffoot, N.1    Chen, K.C.2    Bates, R.C.3
  • 24
    • 34250435812 scopus 로고
    • Physicochemical characteristics of the DNA of parvovirus Lu 3
    • Siegl G. Physicochemical characteristics of the DNA of parvovirus Lu 3. Arch Gesamte Virusforsch 1973; 43: 334-344
    • (1973) Arch Gesamte Virusforsch , vol.43 , pp. 334-344
    • Siegl, G.1
  • 25
    • 0031582213 scopus 로고    scopus 로고
    • The packaging capacity of adeno-Associated virus (AAV) and the potential for wild-Type-plus AAV gene therapy vectors
    • Hermonat PL, Quirk JG, Bishop BM, et al. The packaging capacity of adeno-Associated virus (AAV) and the potential for wild-Type-plus AAV gene therapy vectors. FEBS Lett 1997; 407: 78-84
    • (1997) FEBS Lett , vol.407 , pp. 78-84
    • Hermonat, P.L.1    Quirk, J.G.2    Bishop, B.M.3
  • 26
    • 0029849257 scopus 로고    scopus 로고
    • Quantitative analysis of the packaging capacity of recombinant adenoassociated virus
    • Dong JY, Fan PD, Frizzell RA. Quantitative analysis of the packaging capacity of recombinant adenoassociated virus. Hum Gene Ther 1996; 7: 2101-2112
    • (1996) Hum Gene Ther , vol.7 , pp. 2101-2112
    • Dong, J.Y.1    Fan, P.D.2    Frizzell, R.A.3
  • 27
    • 20444453307 scopus 로고    scopus 로고
    • Protein length in eukaryotic and prokaryotic proteomes
    • Brocchieri L, Karlin S. Protein length in eukaryotic and prokaryotic proteomes. Nucleic Acids Res 2005; 33: 3390-3400
    • (2005) Nucleic Acids Res , vol.33 , pp. 3390-3400
    • Brocchieri, L.1    Karlin, S.2
  • 28
    • 84865219047 scopus 로고    scopus 로고
    • Efficacy of codelivery of dual AAV2/5 vectors in the murine retina and hippocampus
    • Palfi A, Chadderton N, McKee AG, et al. Efficacy of codelivery of dual AAV2/5 vectors in the murine retina and hippocampus. Hum Gene Ther 2012; 23: 847-858
    • (2012) Hum Gene Ther , vol.23 , pp. 847-858
    • Palfi, A.1    Chadderton, N.2    McKee, A.G.3
  • 29
    • 43049119982 scopus 로고    scopus 로고
    • Serotypedependent packaging of large genes in adenoassociated viral vectors results in effective gene delivery in mice
    • Allocca M, Doria M, Petrillo M, et al. Serotypedependent packaging of large genes in adenoassociated viral vectors results in effective gene delivery in mice. J Clin Invest 2008; 118: 1955-1964
    • (2008) J Clin Invest , vol.118 , pp. 1955-1964
    • Allocca, M.1    Doria, M.2    Petrillo, M.3
  • 30
    • 74149093851 scopus 로고    scopus 로고
    • Evidence for the failure of adeno-Associated virus serotype 5 to package a viral genome or = 8.2 kb
    • Lai Y, Yue Y, Duan D. Evidence for the failure of adeno-Associated virus serotype 5 to package a viral genome or = 8.2 kb. Mol Ther 2010; 18: 75-79
    • (2010) Mol Ther , vol.18 , pp. 75-79
    • Lai, Y.1    Yue, Y.2    Duan, D.3
  • 31
    • 74049092844 scopus 로고    scopus 로고
    • Characterization of genome integrity for oversized recombinant AAV vector
    • Dong B, Nakai H, Xiao W. Characterization of genome integrity for oversized recombinant AAV vector. Mol Ther 2010; 18: 87-92
    • (2010) Mol Ther , vol.18 , pp. 87-92
    • Dong, B.1    Nakai, H.2    Xiao, W.3
  • 32
    • 74049085498 scopus 로고    scopus 로고
    • Effect of genome size on AAV vector packaging
    • Wu ZJ, Yang HY, Colosi P. Effect of genome size on AAV vector packaging. Mol Ther 2010; 18: 80-86
    • (2010) Mol Ther , vol.18 , pp. 80-86
    • Wu, Z.J.1    Yang, H.Y.2    Colosi, P.3
  • 33
    • 74049143439 scopus 로고    scopus 로고
    • Little vector, big gene transduction: Fragmented genome reassembly of adeno-Associated virus
    • Hirsch ML, Agbandje-McKenna M, Samulski RJ. Little vector, big gene transduction: Fragmented genome reassembly of adeno-Associated virus. Mol Ther 2010; 18: 6-8
    • (2010) Mol Ther , vol.18 , pp. 6-8
    • Hirsch, M.L.1    Agbandje-McKenna, M.2    Samulski, R.J.3
  • 34
    • 84890119936 scopus 로고    scopus 로고
    • Oversized AAV transductifon is mediated via a DNA-PKcsindependent Rad51C-dependent repair pathway
    • Hirsch ML, Li C, Bellon I, et al. Oversized AAV transductifon is mediated via a DNA-PKcsindependent, Rad51C-dependent repair pathway. Mol Ther 2013; 21: 2205-2216
    • (2013) Mol Ther , vol.21 , pp. 2205-2216
    • Hirsch, M.L.1    Li, C.2    Bellon, I.3
  • 35
    • 20044381100 scopus 로고    scopus 로고
    • A shortened adeno-Associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia
    • Ostedgaard LS, Rokhlina T, Karp PH, et al. A shortened adeno-Associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia. Proc Natl Acad Sci U S A 2005; 102: 2952-2957
    • (2005) Proc Natl Acad Sci U S A , vol.102 , pp. 2952-2957
    • Ostedgaard, L.S.1    Rokhlina, T.2    Karp, P.H.3
  • 36
    • 33646513032 scopus 로고    scopus 로고
    • Evaluation of a novel short polyadenylation signal as an alternative to the SV40 polyadenylation signal
    • McFarland TJ, Zhang Y, Atchaneeyaskul LO, et al. Evaluation of a novel short polyadenylation signal as an alternative to the SV40 polyadenylation signal. Plasmid 2006; 56: 62-67
    • (2006) Plasmid , vol.56 , pp. 62-67
    • McFarland, T.J.1    Zhang, Y.2    Atchaneeyaskul, L.O.3
  • 37
    • 0023918719 scopus 로고
    • Syn thesis, processing, and secretion of recombinant human factor-VIII expressed in mammalian-cells
    • Kaufman RJ, Wasley LC, Dorner AJ. Synthesis, processing, and secretion of recombinant human factor-VIII expressed in mammalian-cells. J Biol Chem 1988; 263: 6352-6362
    • (1988) J Biol Chem , vol.263 , pp. 6352-6362
    • Kaufman, R.J.1    Wasley, L.C.2    Dorner, A.J.3
  • 38
    • 0033607305 scopus 로고    scopus 로고
    • Coexpression of factor VIII heavy and light chain adeno-Associated viral vectors produces biologically active protein
    • Burton M, Nakai H, Colosi P, et al. Coexpression of factor VIII heavy and light chain adeno-Associated viral vectors produces biologically active protein. Proc Natl Acad Sci U S A 1999; 96: 12725-12730
    • (1999) Proc Natl Acad Sci U S A , vol.96 , pp. 12725-12730
    • Burton, M.1    Nakai, H.2    Colosi, P.3
  • 39
    • 84870828091 scopus 로고    scopus 로고
    • AAV-based neonatal gene therapy for hemophilia A: Long-Term correction and avoidance of immune responses in mice
    • Hu C, Lipshutz GS. AAV-based neonatal gene therapy for hemophilia A: Long-Term correction and avoidance of immune responses in mice. Gene Ther 2012; 19: 1166-1176
    • (2012) Gene Ther , vol.19 , pp. 1166-1176
    • Hu, C.1    Lipshutz, G.S.2
  • 40
    • 80053576108 scopus 로고    scopus 로고
    • AAVmicrodystrophin therapy improves cardiac performance in aged female mdx mice
    • Bostick B, Shin JH, Yue Y, et al. AAVmicrodystrophin therapy improves cardiac performance in aged female mdx mice. Mol Ther 2011; 19: 1826-1832
    • (2011) Mol Ther , vol.19 , pp. 1826-1832
    • Bostick, B.1    Shin, J.H.2    Yue, Y.3
  • 41
    • 41149172666 scopus 로고    scopus 로고
    • Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice
    • Gregorevic P, Blankinship MJ, Allen JM, et al. Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. Mol Ther 2008; 16: 657-664
    • (2008) Mol Ther , vol.16 , pp. 657-664
    • Gregorevic, P.1    Blankinship, M.J.2    Allen, J.M.3
  • 42
    • 78650903850 scopus 로고    scopus 로고
    • Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6
    • Odom GL, Gregorevic P, Allen JM, et al. Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol Ther 2011; 19: 36-45
    • (2011) Mol Ther , vol.19 , pp. 36-45
    • Odom, G.L.1    Gregorevic, P.2    Allen, J.M.3
  • 43
    • 0035989639 scopus 로고    scopus 로고
    • Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene
    • Halbert CL, Allen JM, Miller AD. Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol 2002; 20: 697-701
    • (2002) Nat Biotechnol , vol.20 , pp. 697-701
    • Halbert, C.L.1    Allen, J.M.2    Miller, A.D.3
  • 44
    • 85017702957 scopus 로고    scopus 로고
    • AAV.Dysferlin overlap vectors restore function in dysferlinopathy animal models
    • Sondergaard PC, Griffin DA, Pozsgai ER, et al. AAV.Dysferlin overlap vectors restore function in dysferlinopathy animal models. Ann Clin Transl Neurol 2015; 2: 256-270
    • (2015) Ann Clin Transl Neurol , vol.2 , pp. 256-270
    • Sondergaard, P.C.1    Griffin, D.A.2    Pozsgai, E.R.3
  • 45
    • 85015170546 scopus 로고    scopus 로고
    • A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence
    • Pryadkina M, Lostal W, Bourg N, et al. A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence. Mol Ther Methods Clin Dev 2015; 2: 15009
    • (2015) Mol Ther Methods Clin Dev , vol.2 , pp. 15009
    • Pryadkina, M.1    Lostal, W.2    Bourg, N.3
  • 46
    • 84893741157 scopus 로고    scopus 로고
    • Effective delivery of large genes to the retina by dual AAV vectors
    • Trapani I, Colella P, Sommella A, et al. Effective delivery of large genes to the retina by dual AAV vectors. EMBO Mol Med 2014; 6: 194-211
    • (2014) EMBO Mol Med , vol.6 , pp. 194-211
    • Trapani, I.1    Colella, P.2    Sommella, A.3
  • 47
    • 84898722805 scopus 로고    scopus 로고
    • Dual adenoassociated virus vectors result in efficient in vitro and in vivo expression of an oversized gene MYO7A
    • Dyka FM, Boye SL, Chiodo VA, et al. Dual adenoassociated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. Hum Gene Ther Methods 2014; 25: 166-177
    • (2014) Hum Gene Ther Methods , vol.25 , pp. 166-177
    • Dyka, F.M.1    Boye, S.L.2    Chiodo, V.A.3
  • 48
    • 84881477796 scopus 로고    scopus 로고
    • Retinal gene therapy with a large MYO7A cDNA using adenoassociated virus
    • Lopes VS, Boye SE, Louie CM, et al. Retinal gene therapy with a large MYO7A cDNA using adenoassociated virus. Gene Ther 2013; 20: 824-833
    • (2013) Gene Ther , vol.20 , pp. 824-833
    • Lopes, V.S.1    Boye, S.E.2    Louie, C.M.3
  • 49
    • 10644271519 scopus 로고    scopus 로고
    • Inverted terminal repeat sequences are important for intermolecular recombination and circularization of adenoassociated virus genomes
    • Yan Z, Zak R, Zhang Y, et al. Inverted terminal repeat sequences are important for intermolecular recombination and circularization of adenoassociated virus genomes. J Virol 2005; 79: 364-379
    • (2005) J Virol , vol.79 , pp. 364-379
    • Yan, Z.1    Zak, R.2    Zhang, Y.3
  • 50
    • 0033567129 scopus 로고    scopus 로고
    • Structural analysis of adeno-Associated virus transduction circular intermediates
    • Duan D, Yan Z, Yue Y, et al. Structural analysis of adeno-Associated virus transduction circular intermediates. Virology 1999; 261: 8-14
    • (1999) Virology , vol.261 , pp. 8-14
    • Duan, D.1    Yan, Z.2    Yue, Y.3
  • 51
    • 0017295050 scopus 로고
    • Concatemers of alternating plus and minus strands are intermediates in adenovirus-Associated virus DNA synthesis
    • Straus SE, Sebring ED, Rose JA. Concatemers of alternating plus and minus strands are intermediates in adenovirus-Associated virus DNA synthesis. Proc Natl Acad Sci U S A 1976; 73: 742-746
    • (1976) Proc Natl Acad Sci U S A , vol.73 , pp. 742-746
    • Straus, S.E.1    Sebring, E.D.2    Rose, J.A.3
  • 52
    • 0034121397 scopus 로고    scopus 로고
    • A new dual-vector approach to enhance recombinant adenoassociated virus-mediated gene expression through intermolecular cis activation
    • Duan D, Yue Y, Yan Z, et al. A new dual-vector approach to enhance recombinant adenoassociated virus-mediated gene expression through intermolecular cis activation. Nat Med 2000; 6: 595-598
    • (2000) Nat Med , vol.6 , pp. 595-598
    • Duan, D.1    Yue, Y.2    Yan, Z.3
  • 53
    • 0034612248 scopus 로고    scopus 로고
    • Trans-splicing vectors expand the utility of adeno-Associated virus for gene therapy
    • Yan Z, Zhang Y, Duan D, et al. Trans-splicing vectors expand the utility of adeno-Associated virus for gene therapy. Proc Natl Acad Sci U S A 2000; 97: 6716-6721
    • (2000) Proc Natl Acad Sci U S A , vol.97 , pp. 6716-6721
    • Yan, Z.1    Zhang, Y.2    Duan, D.3
  • 54
    • 0035988996 scopus 로고    scopus 로고
    • Expression of human factor VIII by splicing between dimerized AAV vectors
    • Chao H, Sun L, Bruce A, et al. Expression of human factor VIII by splicing between dimerized AAV vectors. Mol Ther 2002; 5: 716-722
    • (2002) Mol Ther , vol.5 , pp. 716-722
    • Chao, H.1    Sun, L.2    Bruce, A.3
  • 55
    • 0034756451 scopus 로고    scopus 로고
    • Expanding AAV packaging capacity with trans-splicing or overlapping vectors: A quantitative comparison
    • Duan D, Yue Y, Engelhardt JF. Expanding AAV packaging capacity with trans-splicing or overlapping vectors: A quantitative comparison. Mol Ther 2001; 4: 383-391
    • (2001) Mol Ther , vol.4 , pp. 383-391
    • Duan, D.1    Yue, Y.2    Engelhardt, J.F.3
  • 56
    • 12244311258 scopus 로고    scopus 로고
    • Efficient trans-splicing in the retina expands the utility of adeno-Associated virus as a vector for gene therapy
    • Reich SJ, Auricchio A, Hildinger M, et al. Efficient trans-splicing in the retina expands the utility of adeno-Associated virus as a vector for gene therapy. Hum Gene Ther 2003; 14: 37-44
    • (2003) Hum Gene Ther , vol.14 , pp. 37-44
    • Reich, S.J.1    Auricchio, A.2    Hildinger, M.3
  • 57
    • 0034121579 scopus 로고    scopus 로고
    • Overcoming adeno-Associated virus vector size limitation through viral DNA heterodimerization
    • Sun L, Li J, Xiao X. Overcoming adeno-Associated virus vector size limitation through viral DNA heterodimerization. Nat Med 2000; 6: 599-602
    • (2000) Nat Med , vol.6 , pp. 599-602
    • Sun, L.1    Li, J.2    Xiao, X.3
  • 58
    • 33750357800 scopus 로고    scopus 로고
    • Host cell DNA repair pathways in adeno-Associated viral genome processing
    • Choi VW, McCarty DM, Samulski RJ. Host cell DNA repair pathways in adeno-Associated viral genome processing. J Virol 2006; 80: 10346-10356
    • (2006) J Virol , vol.80 , pp. 10346-10356
    • Choi, V.W.1    McCarty, D.M.2    Samulski, R.J.3
  • 59
    • 33846192000 scopus 로고    scopus 로고
    • Hybrid adeno-Associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo
    • Yan Z, Lei-Butters DC, Zhang Y, et al. Hybrid adeno-Associated virus bearing nonhomologous inverted terminal repeats enhances dual-vector reconstruction of minigenes in vivo. Hum Gene Ther 2007; 18: 81-87
    • (2007) Hum Gene Ther , vol.18 , pp. 81-87
    • Yan, Z.1    Lei-Butters, D.C.2    Zhang, Y.3
  • 60
    • 84956706085 scopus 로고    scopus 로고
    • Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease
    • Trapani I, Toriello E, de Simone S, et al. Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease. Hum Mol Genet 2015; 24: 6811-6825
    • (2015) Hum Mol Genet , vol.24 , pp. 6811-6825
    • Trapani, I.1    Toriello, E.2    De Simone, S.3
  • 61
    • 27944479711 scopus 로고    scopus 로고
    • Efficient in vivo gene expression by trans-splicing adeno-Associated viral vectors
    • Lai Y, Yue Y, Liu M, et al. Efficient in vivo gene expression by trans-splicing adeno-Associated viral vectors. Nat Biotechnol 2005; 23: 1435-1439
    • (2005) Nat Biotechnol , vol.23 , pp. 1435-1439
    • Lai, Y.1    Yue, Y.2    Liu, M.3
  • 62
    • 33846171622 scopus 로고    scopus 로고
    • Synthetic intron improves transduction efficiency of trans-splicing adeno-Associated viral vectors
    • Lai Y, Yue Y, Liu M, et al. Synthetic intron improves transduction efficiency of trans-splicing adeno-Associated viral vectors. Hum Gene Ther 2006; 17: 1036-1042
    • (2006) Hum Gene Ther , vol.17 , pp. 1036-1042
    • Lai, Y.1    Yue, Y.2    Liu, M.3
  • 63
    • 77952502008 scopus 로고    scopus 로고
    • Efficient recovery of dysferlin deficiency by dual adenoassociated vector-mediated gene transfer
    • Lostal W, Bartoli M, Bourg N, et al. Efficient recovery of dysferlin deficiency by dual adenoassociated vector-mediated gene transfer. Hum Mol Genet 2010; 19: 1897-1907
    • (2010) Hum Mol Genet , vol.19 , pp. 1897-1907
    • Lostal, W.1    Bartoli, M.2    Bourg, N.3
  • 64
    • 5744230326 scopus 로고    scopus 로고
    • Disruption of muscle membrane and phenotype divergence in two novel mouse models of dysferlin deficiency
    • Ho M, Post CM, Donahue LR, et al. Disruption of muscle membrane and phenotype divergence in two novel mouse models of dysferlin deficiency. Hum Mol Genet 2004; 13: 1999-2010
    • (2004) Hum Mol Genet , vol.13 , pp. 1999-2010
    • Ho, M.1    Post, C.M.2    Donahue, L.R.3
  • 65
    • 37549053613 scopus 로고    scopus 로고
    • A hybrid vector system expands adeno-Associated viral vector packaging capacity in a transgene-independent manner
    • Ghosh A, Yue Y, Lai Y, et al. A hybrid vector system expands adeno-Associated viral vector packaging capacity in a transgene-independent manner. Mol Ther 2008; 16: 124-130
    • (2008) Mol Ther , vol.16 , pp. 124-130
    • Ghosh, A.1    Yue, Y.2    Lai, Y.3
  • 66
    • 33645223689 scopus 로고    scopus 로고
    • Viral serotype and the transgene sequence influence overlapping adenoassociated viral (AAV) vector-mediated gene transfer in skeletal muscle
    • Ghosh A, Yue Y, Duan D. Viral serotype and the transgene sequence influence overlapping adenoassociated viral (AAV) vector-mediated gene transfer in skeletal muscle. J Gene Med 2006; 8: 298-305
    • (2006) J Gene Med , vol.8 , pp. 298-305
    • Ghosh, A.1    Yue, Y.2    Duan, D.3
  • 67
    • 79951904478 scopus 로고    scopus 로고
    • Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequences
    • Ghosh A, Yue Y, Duan D. Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequences. Hum Gene Ther 2011; 22: 77-83
    • (2011) Hum Gene Ther , vol.22 , pp. 77-83
    • Ghosh, A.1    Yue, Y.2    Duan, D.3
  • 68
    • 84862908306 scopus 로고    scopus 로고
    • Novel mini-dystrophin gene dual adeno-Associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma
    • Zhang Y, Duan D. Novel mini-dystrophin gene dual adeno-Associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma. Hum Gene Ther 2012; 23: 98-103
    • (2012) Hum Gene Ther , vol.23 , pp. 98-103
    • Zhang, Y.1    Duan, D.2
  • 69
    • 65649111197 scopus 로고    scopus 로고
    • Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy
    • Lai Y, Thomas GD, Yue Y, et al. Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy. J Clin Invest 2009; 119: 624-635
    • (2009) J Clin Invest , vol.119 , pp. 624-635
    • Lai, Y.1    Thomas, G.D.2    Yue, Y.3
  • 70
    • 84893698363 scopus 로고    scopus 로고
    • Triple trans-splicing adeno-Associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice
    • Koo T, Popplewell L, Athanasopoulos T, et al. Triple trans-splicing adeno-Associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice. Hum Gene Ther 2014; 25: 98-108
    • (2014) Hum Gene Ther , vol.25 , pp. 98-108
    • Koo, T.1    Popplewell, L.2    Athanasopoulos, T.3
  • 71
    • 28444453994 scopus 로고    scopus 로고
    • Spliceosome-mediated RNA trans-splicing
    • Yang Y, Walsh CE. Spliceosome-mediated RNA trans-splicing. Mol Ther 2005; 12: 1006-1012
    • (2005) Mol Ther , vol.12 , pp. 1006-1012
    • Yang, Y.1    Walsh, C.E.2
  • 72
    • 84876695347 scopus 로고    scopus 로고
    • Protein trans-splicing as a protein ligation tool to study protein structure and function
    • Aranko AS, Volkmann G. Protein trans-splicing as a protein ligation tool to study protein structure and function. Biomol Concepts 2011; 2: 183-198
    • (2011) Biomol Concepts , vol.2 , pp. 183-198
    • Aranko, A.S.1    Volkmann, G.2


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