메뉴 건너뛰기




Volumn 7, Issue , 2017, Pages

Erratum: Lentiviral vectors can be used for full-length dystrophin gene therapy (Scientific Reports (2017) 7 (44775) DOI: 10.1038/srep44775);Lentiviral vectors can be used for full-length dystrophin gene therapy

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN;

EID: 85015635141     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep46880     Document Type: Erratum
Times cited : (30)

References (53)
  • 1
    • 0023614188 scopus 로고
    • Dystrophin: The protein product of the Duchenne muscular dystrophy locus
    • Hoffman, E. P., Brown, R. H. & Kunkel, L. M. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51, 919-28 (1987).
    • (1987) Cell , vol.51 , pp. 919-928
    • Hoffman, E.P.1    Brown, R.H.2    Kunkel, L.M.3
  • 2
    • 0034783643 scopus 로고    scopus 로고
    • Systematic determination of the packaging limit of lentiviral vectors
    • Kumar, M., Keller, B., Makalou, N. & Sutton, R. E. Systematic determination of the packaging limit of lentiviral vectors. Hum. Gene Ther. 12, 1893-1905 (2001).
    • (2001) Hum. Gene Ther. , vol.12 , pp. 1893-1905
    • Kumar, M.1    Keller, B.2    Makalou, N.3    Sutton, R.E.4
  • 3
    • 74049085498 scopus 로고    scopus 로고
    • Effect of genome size on AAV vector packaging
    • Wu, Z., Yang, H. & Colosi, P. Effect of genome size on AAV vector packaging. Mol. Ther. 18, 80-6 (2010).
    • (2010) Mol. Ther. , vol.18 , pp. 80-86
    • Wu, Z.1    Yang, H.2    Colosi, P.3
  • 4
    • 0029849257 scopus 로고    scopus 로고
    • Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
    • Dong, J. Y., Fan, P. D. & Frizzell, R. A. Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 7, 2101-12 (1996).
    • (1996) Hum. Gene Ther. , vol.7 , pp. 2101-2112
    • Dong, J.Y.1    Fan, P.D.2    Frizzell, R.A.3
  • 5
    • 33745191588 scopus 로고    scopus 로고
    • A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy
    • Li, S. et al. A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy. Hum Mol Genet 15, 1610-1622 (2006).
    • (2006) Hum Mol Genet , vol.15 , pp. 1610-1622
    • Li, S.1
  • 6
    • 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. 23, 98-103 (2012).
    • (2012) Hum. Gene Ther. , vol.23 , pp. 98-103
    • Zhang, Y.1    Duan, D.2
  • 7
    • 0035793047 scopus 로고    scopus 로고
    • Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
    • Mann, C. J. et al. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc. Natl. Acad. Sci. USA 98, 42-7 (2001).
    • (2001) Proc. Natl. Acad. Sci. USA , vol.98 , pp. 42-47
    • Mann, C.J.1
  • 8
    • 0042536463 scopus 로고    scopus 로고
    • Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
    • Lu, Q. L. et al. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat. Med. 9, 1009-14 (2003).
    • (2003) Nat. Med. , vol.9 , pp. 1009-1014
    • Lu, Q.L.1
  • 9
    • 36749032678 scopus 로고    scopus 로고
    • Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice
    • Benchaouir, R. et al. Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice. Cell Stem Cell 1, 646-57 (2007).
    • (2007) Cell Stem Cell , vol.1 , pp. 646-657
    • Benchaouir, R.1
  • 10
    • 84907200149 scopus 로고    scopus 로고
    • Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
    • Long, C. et al. Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science 345, 1184-1188 (2014).
    • (2014) Science , vol.345 , pp. 1184-1188
    • Long, C.1
  • 11
    • 84961291537 scopus 로고    scopus 로고
    • Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
    • Long, C. et al. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science, doi: 10.1126/science.aad5725 (2015).
    • (2015) Science
    • Long, C.1
  • 12
    • 84963940775 scopus 로고    scopus 로고
    • In vivo genome editing improves muscle function in a mouse model of duchenne muscular dystrophy
    • science.aad5143
    • Nelson, C. E. et al. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science science.aad5143, doi: 10.1126/science.aad5143 (2015).
    • (2015) Science
    • Nelson, C.E.1
  • 13
    • 84963985350 scopus 로고    scopus 로고
    • In vivo gene editing in dystrophic mouse muscle and muscle stem cells
    • Tabebordbar, M. et al. In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351, 407-411 (2015).
    • (2015) Science , vol.351 , pp. 407-411
    • Tabebordbar, M.1
  • 14
    • 0025159208 scopus 로고
    • Very mild muscular dystrophy associated with the deletion of 46% of dystrophin
    • England, S. B. et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 343, 180-2 (1990).
    • (1990) Nature , vol.343 , pp. 180-182
    • England, S.B.1
  • 15
    • 0036091665 scopus 로고    scopus 로고
    • Efficient integration of recombinant adeno-associated virus DNA vectors requires a p5-rep sequence in cis
    • Philpott, N. J. et al. Efficient integration of recombinant adeno-associated virus DNA vectors requires a p5-rep sequence in cis. J. Virol. 76, 5411-21 (2002).
    • (2002) J. Virol. , vol.76 , pp. 5411-5421
    • Philpott, N.J.1
  • 16
    • 84949556815 scopus 로고    scopus 로고
    • Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division
    • Dumont, N. A. et al. Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division. Nat. Med. 21, 1455-63 (2015).
    • (2015) Nat. Med. , vol.21 , pp. 1455-1463
    • Dumont, N.A.1
  • 17
    • 33644822908 scopus 로고    scopus 로고
    • Effective gene therapy with nonintegrating lentiviral vectors
    • Yanez-Munoz, R. J. et al. Effective gene therapy with nonintegrating lentiviral vectors. Nat. Med. 12, 348-53 (2006).
    • (2006) Nat. Med. , vol.12 , pp. 348-353
    • Yanez-Munoz, R.J.1
  • 18
    • 34447581119 scopus 로고    scopus 로고
    • Optimized production and concentration of lentiviral vectors containing large inserts
    • Al Yacoub, N., Romanowska, M., Haritonova, N. & Foerster, J. Optimized production and concentration of lentiviral vectors containing large inserts. J. Gene Med. 9, 579-584 (2007).
    • (2007) J. Gene Med. , vol.9 , pp. 579-584
    • Al Yacoub, N.1    Romanowska, M.2    Haritonova, N.3    Foerster, J.4
  • 19
    • 84955513294 scopus 로고    scopus 로고
    • Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy
    • Meng, J. et al. Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy. Sci. Rep. 6, 19750 (2016).
    • (2016) Sci. Rep. , vol.6 , pp. 19750
    • Meng, J.1
  • 20
    • 84975795968 scopus 로고    scopus 로고
    • Lentiviral gene transfer into human and murine hematopoietic stem cells: Size matters
    • Cante-Barrett, K. et al. Lentiviral gene transfer into human and murine hematopoietic stem cells: size matters. BMC Res. Notes 9, 312 (2016).
    • (2016) BMC Res. Notes , vol.9 , pp. 312
    • Cante-Barrett, K.1
  • 21
    • 0025095253 scopus 로고
    • Genetic consequences of packaging two RNA genomes in one retroviral particle: Pseudodiploidy and high rate of genetic recombination
    • Hu, W. S. & Temin, H. M. Genetic consequences of packaging two RNA genomes in one retroviral particle: pseudodiploidy and high rate of genetic recombination. Proc. Natl. Acad. Sci. USA 87, 1556-60 (1990).
    • (1990) Proc. Natl. Acad. Sci. USA , vol.87 , pp. 1556-1560
    • Hu, W.S.1    Temin, H.M.2
  • 22
    • 0028274130 scopus 로고
    • Retrovirus recombination depends on the length of sequence identity and is not error prone
    • Zhang, J. & Temin, H. M. Retrovirus recombination depends on the length of sequence identity and is not error prone. J. Virol. 68, 2409-14 (1994).
    • (1994) J. Virol. , vol.68 , pp. 2409-2414
    • Zhang, J.1    Temin, H.M.2
  • 23
    • 0036822475 scopus 로고    scopus 로고
    • HIV-1 genetic recombination: Experimental approaches and observations
    • An, W. & Telesnitsky, A. HIV-1 genetic recombination: Experimental approaches and observations. AIDS Rev. 4, 195-212 (2002).
    • (2002) AIDS Rev. , vol.4 , pp. 195-212
    • An, W.1    Telesnitsky, A.2
  • 24
    • 0026486193 scopus 로고
    • Mechanism of DNA strand transfer reactions catalyzed by HIV-1 reverse transcriptase
    • Peliska, J. & Benkovic, S. Mechanism of DNA strand transfer reactions catalyzed by HIV-1 reverse transcriptase. Science (80-). 258, 1112-1118 (1992).
    • (1992) Science (80-) , vol.258 , pp. 1112-1118
    • Peliska, J.1    Benkovic, S.2
  • 25
    • 33845917770 scopus 로고    scopus 로고
    • Reduced dNTP interaction of human immunodeficiency virus type 1 reverse transcriptase promotes strand transfer
    • Operario, D. J., Balakrishnan, M., Bambara, R. A. & Kim, B. Reduced dNTP Interaction of Human Immunodeficiency Virus Type 1 Reverse Transcriptase Promotes Strand Transfer. J. Biol. Chem. 281, 32113-32121 (2006).
    • (2006) J. Biol. Chem. , vol.281 , pp. 32113-32121
    • Operario, D.J.1    Balakrishnan, M.2    Bambara, R.A.3    Kim, B.4
  • 26
    • 69449090477 scopus 로고    scopus 로고
    • High efficiency of HIV-1 genomic RNA packaging and heterozygote formation revealed by single virion analysis
    • Chen, J. et al. High efficiency of HIV-1 genomic RNA packaging and heterozygote formation revealed by single virion analysis. Proc. Natl. Acad. Sci. USA 106, 13535-13540 (2009).
    • (2009) Proc. Natl. Acad. Sci. USA , vol.106 , pp. 13535-13540
    • Chen, J.1
  • 27
    • 80054971700 scopus 로고    scopus 로고
    • Determining the frequency and mechanisms of HIV-1 and HIV-2 RNA copackaging by single-virion analysis
    • Dilley, K. a. et al. Determining the Frequency and Mechanisms of HIV-1 and HIV-2 RNA Copackaging by Single-Virion Analysis. J. Virol. 85, 10499-10508 (2011).
    • (2011) J. Virol. , vol.85 , pp. 10499-10508
    • Dilley, K.A.1
  • 28
    • 34247095230 scopus 로고    scopus 로고
    • Dimer initiation signal of human immunodeficiency virus type 1: Its role in partner selection during RNA copackaging and its effects on recombination
    • Moore, M. D. et al. Dimer initiation signal of human immunodeficiency virus type 1: its role in partner selection during RNA copackaging and its effects on recombination. J. Virol. 81, 4002-11 (2007).
    • (2007) J. Virol. , vol.81 , pp. 4002-4011
    • Moore, M.D.1
  • 29
    • 79955707349 scopus 로고    scopus 로고
    • High cleavage efficiency of a 2A peptide derived from porcine teschovirus-1 in human cell lines, zebrafish and mice
    • Kim, J. H. et al. High Cleavage Efficiency of a 2A Peptide Derived from Porcine Teschovirus-1 in Human Cell Lines, Zebrafish and Mice. PLoS One 6, e18556 (2011).
    • (2011) PLoS One , vol.6 , pp. e18556
    • Kim, J.H.1
  • 31
    • 34247125600 scopus 로고    scopus 로고
    • Using RT-prone recombination to promote re-building of complete retroviral vectors from two defective precursors: Low efficiency and sequence specificities
    • Bru, T. et al. Using RT-prone recombination to promote re-building of complete retroviral vectors from two defective precursors: Low efficiency and sequence specificities. J. Virol. Methods 142, 118-126 (2007).
    • (2007) J. Virol. Methods , vol.142 , pp. 118-126
    • Bru, T.1
  • 32
    • 33144454419 scopus 로고    scopus 로고
    • Development of a new bicistronic retroviral vector with strong IRES activity
    • Martin, P., Albagli, O., Poggi, M. C., Boulukos, K. E. & Pognonec, P. Development of a new bicistronic retroviral vector with strong IRES activity. BMC Biotechnol. 6, 4 (2006).
    • (2006) BMC Biotechnol. , vol.6 , pp. 4
    • Martin, P.1    Albagli, O.2    Poggi, M.C.3    Boulukos, K.E.4    Pognonec, P.5
  • 33
    • 47749126221 scopus 로고    scopus 로고
    • Compensatory role of human immunodeficiency virus central polypurine tract sequence in kinetically disrupted reverse transcription
    • Skasko, M. & Kim, B. Compensatory role of human immunodeficiency virus central polypurine tract sequence in kinetically disrupted reverse transcription. J. Virol. 82, 7716-7720 (2008).
    • (2008) J. Virol. , vol.82 , pp. 7716-7720
    • Skasko, M.1    Kim, B.2
  • 34
    • 43049136539 scopus 로고    scopus 로고
    • Strand transfer events during HIV-1 reverse transcription
    • Basu, V. P. et al. Strand transfer events during HIV-1 reverse transcription. Virus Res 134, 19-38 (2008).
    • (2008) Virus Res , vol.134 , pp. 19-38
    • Basu, V.P.1
  • 35
    • 0028803233 scopus 로고
    • Analysis of primer extension and the first template switch during human immunodeficiency virus reverse transcription
    • Arts, E. J., Li, Z. & Wainberg, M. A. Analysis of Primer Extension and the First Template Switch during Human Immunodeficiency Virus Reverse Transcription. J. Biomed. Sci. 2, 314-321 (1995).
    • (1995) J. Biomed. Sci. , vol.2 , pp. 314-321
    • Arts, E.J.1    Li, Z.2    Wainberg, M.A.3
  • 36
    • 0030831874 scopus 로고    scopus 로고
    • RNA secondary structure switching during DNA synthesis catalyzed by HIV-1 reverse transcriptase
    • Suo, Z. & Johnson, K. A. RNA Secondary Structure Switching during DNA Synthesis Catalyzed by HIV-1 Reverse Transcriptase. Biochemistry 36, 14778-14785 (1997).
    • (1997) Biochemistry , vol.36 , pp. 14778-14785
    • Suo, Z.1    Johnson, K.A.2
  • 37
    • 0035259873 scopus 로고    scopus 로고
    • A tertiary structure model of the internal ribosome entry site (IRES) for methionine-independent initiation of translation
    • Kanamori, Y. & Nakashima, N. A tertiary structure model of the internal ribosome entry site (IRES) for methionine-independent initiation of translation. RNA 7, 266-274 (2001).
    • (2001) RNA , vol.7 , pp. 266-274
    • Kanamori, Y.1    Nakashima, N.2
  • 38
    • 0038750886 scopus 로고    scopus 로고
    • Structural elements in the internal ribosome entry site of Plautia stali intestine virus responsible for binding with ribosomes
    • Nishiyama, T. et al. Structural elements in the internal ribosome entry site of Plautia stali intestine virus responsible for binding with ribosomes. Nucleic Acids Res. 31, 2434-2442 (2003).
    • (2003) Nucleic Acids Res. , vol.31 , pp. 2434-2442
    • Nishiyama, T.1
  • 39
    • 0037073485 scopus 로고    scopus 로고
    • Factorless ribosome assembly on the internal ribosome entry site of cricket paralysis virus
    • Jan, E. & Sarnow, P. Factorless Ribosome Assembly on the Internal Ribosome Entry Site of Cricket Paralysis Virus. J. Mol. Biol. 324, 889-902 (2002).
    • (2002) J. Mol. Biol. , vol.324 , pp. 889-902
    • Jan, E.1    Sarnow, P.2
  • 40
    • 0032528327 scopus 로고    scopus 로고
    • Pausing of reverse transcriptase on retroviral RNA templates is influenced by secondary structures both 5′ and 3′ of the catalytic site
    • Harrison, G. P., Mayo, M. S., Hunter, E. & Lever, A. M. L. Pausing of reverse transcriptase on retroviral RNA templates is influenced by secondary structures both 5′ and 3′ of the catalytic site. Nucleic Acids Res. 26, 3433-3442 (1998).
    • (1998) Nucleic Acids Res. , vol.26 , pp. 3433-3442
    • Harrison, G.P.1    Mayo, M.S.2    Hunter, E.3    Lever, A.M.L.4
  • 41
    • 0027238697 scopus 로고
    • Template-directed pausing of DNA synthesis by HIV-1 reverse transcriptase during polymerization of HIV-1 sequences in vitro
    • Klarmann, G. J., Schauber, C. A. & Preston, B. D. Template-directed pausing of DNA synthesis by HIV-1 reverse transcriptase during polymerization of HIV-1 sequences in vitro. J. Biol. Chem. 268, 9793-802 (1993).
    • (1993) J. Biol. Chem. , vol.268 , pp. 9793-9802
    • Klarmann, G.J.1    Schauber, C.A.2    Preston, B.D.3
  • 42
    • 0027215749 scopus 로고
    • Mechanism of HIV-1 reverse transcriptase. Termination of processive synthesis on a natural DNA template is influenced by the sequence of the template-primer stem
    • Abbotts, J., Bebenek, K., Kunkel, T. A. & Wilson, S. H. Mechanism of HIV-1 reverse transcriptase. Termination of processive synthesis on a natural DNA template is influenced by the sequence of the template-primer stem. J. Biol. Chem. 268, 10312-23 (1993).
    • (1993) J. Biol. Chem. , vol.268 , pp. 10312-10323
    • Abbotts, J.1    Bebenek, K.2    Kunkel, T.A.3    Wilson, S.H.4
  • 43
    • 79951936473 scopus 로고    scopus 로고
    • Creating higher titer lentivirus with caffeine
    • Ellis, B. L., Potts, P. R. & Porteus, M. H. Creating Higher Titer Lentivirus with Caffeine. Hum. Gene Ther. 22, 93-100 (2011).
    • (2011) Hum. Gene Ther. , vol.22 , pp. 93-100
    • Ellis, B.L.1    Potts, P.R.2    Porteus, M.H.3
  • 44
    • 84887293605 scopus 로고    scopus 로고
    • Simplified production and concentration of lentiviral vectors to achieve high transduction in primary human T cells
    • Cribbs, A. P., Kennedy, A., Gregory, B. & Brennan, F. M. Simplified production and concentration of lentiviral vectors to achieve high transduction in primary human T cells. BMC Biotechnol. 13, 98 (2013).
    • (2013) BMC Biotechnol. , vol.13 , pp. 98
    • Cribbs, A.P.1    Kennedy, A.2    Gregory, B.3    Brennan, F.M.4
  • 45
    • 0038182574 scopus 로고    scopus 로고
    • Dystrophin-glycoprotein complex: Post-translational processing and dystroglycan function
    • Michele, D. E. & Campbell, K. P. Dystrophin-glycoprotein complex: post-translational processing and dystroglycan function. J. Biol. Chem. 278, 15457-60 (2003).
    • (2003) J. Biol. Chem. , vol.278 , pp. 15457-15460
    • Michele, D.E.1    Campbell, K.P.2
  • 46
    • 0031710033 scopus 로고    scopus 로고
    • A third-generation lentivirus vector with a conditional packaging system
    • Dull, T. et al. A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463-8471 (1998).
    • (1998) J. Virol. , vol.72 , pp. 8463-8471
    • Dull, T.1
  • 48
    • 0030819379 scopus 로고    scopus 로고
    • Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
    • Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L. & Trono, D. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat. Biotechnol. 15, 871-5 (1997).
    • (1997) Nat. Biotechnol. , vol.15 , pp. 871-875
    • Zufferey, R.1    Nagy, D.2    Mandel, R.J.3    Naldini, L.4    Trono, D.5
  • 49
    • 0031743608 scopus 로고    scopus 로고
    • Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
    • Zufferey, R. et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873-9880 (1998).
    • (1998) J. Virol. , vol.72 , pp. 9873-9880
    • Zufferey, R.1
  • 50
    • 79955936790 scopus 로고    scopus 로고
    • Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction
    • Charrier, S. et al. Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction. Gene Ther. 18, 479-87 (2011).
    • (2011) Gene Ther. , vol.18 , pp. 479-487
    • Charrier, S.1
  • 51
    • 15244346492 scopus 로고    scopus 로고
    • Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells
    • De Palma, M. et al. Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood 105, 2307-15 (2005).
    • (2005) Blood , vol.105 , pp. 2307-2315
    • De Palma, M.1
  • 52
    • 84923381487 scopus 로고    scopus 로고
    • Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases
    • Mock, U. et al. Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases. Sci. Rep. 4, 6409 (2014).
    • (2014) Sci. Rep. , vol.4 , pp. 6409
    • Mock, U.1
  • 53
    • 38949175402 scopus 로고    scopus 로고
    • Protocol for nearly full-length sequencing of HIV-1 RNA from plasma
    • Nadai, Y. et al. Protocol for Nearly Full-Length Sequencing of HIV-1 RNA from Plasma. PLoS One 3, e1420 (2008).
    • (2008) PLoS One , vol.3 , pp. e1420
    • Nadai, Y.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.