메뉴 건너뛰기




Volumn 35, Issue 1, 2015, Pages 81-88

Adeno-associated virus-mediated microRNA delivery and therapeutics

Author keywords

adeno associated virus; in vivo gene delivery; microRNA inhibition; microRNA overexpression

Indexed keywords

MICRORNA; PARVOVIRUS VECTOR;

EID: 84925848922     PISSN: 02728087     EISSN: 10988971     Source Type: Journal    
DOI: 10.1055/s-0034-1397352     Document Type: Article
Times cited : (24)

References (79)
  • 1
    • 11844278458 scopus 로고    scopus 로고
    • Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets
    • Lewis B. P., Burge C. B., Bartel D. P. Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets. Cell: 2005; 120 1 15 20
    • (2005) Cell , vol.120 , Issue.1 , pp. 15-20
    • Lewis, B.P.1    Burge, C.B.2    Bartel, D.P.3
  • 2
    • 18344369543 scopus 로고    scopus 로고
    • MicroRNA biogenesis: Coordinated cropping and dicing
    • Kim V. N. MicroRNA biogenesis: coordinated cropping and dicing. Nat Rev Mol Cell Biol: 2005; 6 5 376 385
    • (2005) Nat Rev Mol Cell Biol , vol.6 , Issue.5 , pp. 376-385
    • Kim, V.N.1
  • 3
    • 9344235449 scopus 로고    scopus 로고
    • Human microRNAs are processed from capped, polyadenylated transcripts that can also function as mRNAs
    • Cai X., Hagedorn C. H., Cullen B. R. Human microRNAs are processed from capped, polyadenylated transcripts that can also function as mRNAs. RNA: 2004; 10 12 1957 1966
    • (2004) RNA , vol.10 , Issue.12 , pp. 1957-1966
    • Cai, X.1    Hagedorn, C.H.2    Cullen, B.R.3
  • 4
    • 33845370280 scopus 로고    scopus 로고
    • RNA polymerase III transcribes human microRNAs
    • Borchert G. M., Lanier W., Davidson B. L. RNA polymerase III transcribes human microRNAs. Nat Struct Mol Biol: 2006; 13 12 1097 1101
    • (2006) Nat Struct Mol Biol , vol.13 , Issue.12 , pp. 1097-1101
    • Borchert, G.M.1    Lanier, W.2    Davidson, B.L.3
  • 5
    • 0027751663 scopus 로고
    • The C. Elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14
    • Lee R. C., Feinbaum R. L., Ambros V. The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14. Cell: 1993; 75 5 843 854
    • (1993) Cell , vol.75 , Issue.5 , pp. 843-854
    • Lee, R.C.1    Feinbaum, R.L.2    Ambros, V.3
  • 6
    • 18744396337 scopus 로고    scopus 로고
    • Frequent deletions and down-regulation of micro- RNA genes miR15 and miR16 at 13q14 in chronic lymphocytic leukemia
    • Calin G. A., Dumitru C. D., Shimizu M., et al. Frequent deletions and down-regulation of micro- RNA genes miR15 and miR16 at 13q14 in chronic lymphocytic leukemia. Proc Natl Acad Sci U S A: 2002; 99 24 15524 15529
    • (2002) Proc Natl Acad Sci U S A , vol.99 , Issue.24 , pp. 15524-15529
    • Calin, G.A.1    Dumitru, C.D.2    Shimizu, M.3
  • 7
    • 12144290519 scopus 로고    scopus 로고
    • Human microRNA genes are frequently located at fragile sites and genomic regions involved in cancers
    • Calin G. A., Sevignani C., Dumitru C. D., et al. Human microRNA genes are frequently located at fragile sites and genomic regions involved in cancers. Proc Natl Acad Sci U S A: 2004; 101 9 2999 3004
    • (2004) Proc Natl Acad Sci U S A , vol.101 , Issue.9 , pp. 2999-3004
    • Calin, G.A.1    Sevignani, C.2    Dumitru, C.D.3
  • 8
    • 20044395613 scopus 로고    scopus 로고
    • RAS is regulated by the let-7 microRNA family
    • Johnson S. M., Grosshans H., Shingara J., et al. RAS is regulated by the let-7 microRNA family. Cell: 2005; 120 5 635 647
    • (2005) Cell , vol.120 , Issue.5 , pp. 635-647
    • Johnson, S.M.1    Grosshans, H.2    Shingara, J.3
  • 9
    • 77949658280 scopus 로고    scopus 로고
    • Regression of murine lung tumors by the let-7 microRNA
    • Trang P., Medina P. P., Wiggins J. F., et al. Regression of murine lung tumors by the let-7 microRNA. Oncogene: 2010; 29 11 1580 1587
    • (2010) Oncogene , vol.29 , Issue.11 , pp. 1580-1587
    • Trang, P.1    Medina, P.P.2    Wiggins, J.F.3
  • 10
    • 70349871321 scopus 로고    scopus 로고
    • MicroRNA expression, survival, and response to interferon in liver cancer
    • Ji J., Shi J., Budhu A., et al. MicroRNA expression, survival, and response to interferon in liver cancer. N Engl J Med: 2009; 361 15 1437 1447
    • (2009) N Engl J Med , vol.361 , Issue.15 , pp. 1437-1447
    • Ji, J.1    Shi, J.2    Budhu, A.3
  • 11
    • 0037015049 scopus 로고    scopus 로고
    • Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
    • Gao G. P., Alvira M. R., Wang L., Calcedo R., Johnston J., Wilson J. M. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A: 2002; 99 18 11854 11859
    • (2002) Proc Natl Acad Sci U S A , vol.99 , Issue.18 , pp. 11854-11859
    • Gao, G.P.1    Alvira, M.R.2    Wang, L.3    Calcedo, R.4    Johnston, J.5    Wilson, J.M.6
  • 12
    • 66449136951 scopus 로고    scopus 로고
    • Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model
    • Kota J., Chivukula R. R., O'Donnell K. A., et al. Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model. Cell: 2009; 137 6 1005 1017
    • (2009) Cell , vol.137 , Issue.6 , pp. 1005-1017
    • Kota, J.1    Chivukula, R.R.2    O'Donnell, K.A.3
  • 13
    • 33845317603 scopus 로고    scopus 로고
    • A signature pattern of stress-responsive microRNAs that can evoke cardiac hypertrophy and heart failure
    • van Rooij E., Sutherland L. B., Liu N., et al. A signature pattern of stress-responsive microRNAs that can evoke cardiac hypertrophy and heart failure. Proc Natl Acad Sci U S A: 2006; 103 48 18255 18260
    • (2006) Proc Natl Acad Sci U S A , vol.103 , Issue.48 , pp. 18255-18260
    • Van Rooij, E.1    Sutherland, L.B.2    Liu, N.3
  • 14
    • 57749168828 scopus 로고    scopus 로고
    • MicroRNA-21 contributes to myocardial disease by stimulating MAP kinase signalling in fibroblasts
    • Thum T., Gross C., Fiedler J., et al. MicroRNA-21 contributes to myocardial disease by stimulating MAP kinase signalling in fibroblasts. Nature: 2008; 456 7224 980 984
    • (2008) Nature , vol.456 , Issue.7224 , pp. 980-984
    • Thum, T.1    Gross, C.2    Fiedler, J.3
  • 15
    • 80053567152 scopus 로고    scopus 로고
    • Therapeutic inhibition of miR-208a improves cardiac function and survival during heart failure
    • Montgomery R. L., Hullinger T. G., Semus H. M., et al. Therapeutic inhibition of miR-208a improves cardiac function and survival during heart failure. Circulation: 2011; 124 14 1537 1547
    • (2011) Circulation , vol.124 , Issue.14 , pp. 1537-1547
    • Montgomery, R.L.1    Hullinger, T.G.2    Semus, H.M.3
  • 16
    • 34247589595 scopus 로고    scopus 로고
    • Control of stress-dependent cardiac growth and gene expression by a microRNA
    • van Rooij E., Sutherland L. B., Qi X., Richardson J. A., Hill J., Olson E. N. Control of stress-dependent cardiac growth and gene expression by a microRNA. Science: 2007; 316 5824 575 579
    • (2007) Science , vol.316 , Issue.5824 , pp. 575-579
    • Van Rooij, E.1    Sutherland, L.B.2    Qi, X.3    Richardson, J.A.4    Hill, J.5    Olson, E.N.6
  • 17
    • 84860340270 scopus 로고    scopus 로고
    • A cardiac microRNA governs systemic energy homeostasis by regulation of MED13
    • Grueter C. E., van Rooij E., Johnson B. A., et al. A cardiac microRNA governs systemic energy homeostasis by regulation of MED13. Cell: 2012; 149 3 671 683
    • (2012) Cell , vol.149 , Issue.3 , pp. 671-683
    • Grueter, C.E.1    Van Rooij, E.2    Johnson, B.A.3
  • 18
    • 65249093130 scopus 로고    scopus 로고
    • MiR-375 maintains normal pancreatic alpha- and beta-cell mass
    • Poy M. N., Hausser J., Trajkovski M., et al. miR-375 maintains normal pancreatic alpha- and beta-cell mass. Proc Natl Acad Sci U S A: 2009; 106 14 5813 5818
    • (2009) Proc Natl Acad Sci U S A , vol.106 , Issue.14 , pp. 5813-5818
    • Poy, M.N.1    Hausser, J.2    Trajkovski, M.3
  • 19
    • 77953787211 scopus 로고    scopus 로고
    • MiR-33 contributes to the regulation of cholesterol homeostasis
    • Rayner K. J., Suárez Y., Dávalos A., et al. MiR-33 contributes to the regulation of cholesterol homeostasis. Science: 2010; 328 5985 1570 1573
    • (2010) Science , vol.328 , Issue.5985 , pp. 1570-1573
    • Rayner, K.J.1    Suárez, Y.2    Dávalos, A.3
  • 20
    • 79960015327 scopus 로고    scopus 로고
    • Antagonism of miR-33 in mice promotes reverse cholesterol transport and regression of atherosclerosis
    • Rayner K. J., Sheedy F. J., Esau C. C., et al. Antagonism of miR-33 in mice promotes reverse cholesterol transport and regression of atherosclerosis. J Clin Invest: 2011; 121 7 2921 2931
    • (2011) J Clin Invest , vol.121 , Issue.7 , pp. 2921-2931
    • Rayner, K.J.1    Sheedy, F.J.2    Esau, C.C.3
  • 21
    • 80054971110 scopus 로고    scopus 로고
    • Inhibition of miR-33a/b in non-human primates raises plasma HDL and lowers VLDL triglycerides
    • Rayner K. J., Esau C. C., Hussain F. N., et al. Inhibition of miR-33a/b in non-human primates raises plasma HDL and lowers VLDL triglycerides. Nature: 2011; 478 7369 404 407
    • (2011) Nature , vol.478 , Issue.7369 , pp. 404-407
    • Rayner, K.J.1    Esau, C.C.2    Hussain, F.N.3
  • 22
    • 23844523406 scopus 로고    scopus 로고
    • MiR-122, a mammalian liver-specific microRNA, is processed from hcr mRNA and may downregulate the high affinity cationic amino acid transporter CAT-1
    • Chang J., Nicolas E., Marks D., et al. miR-122, a mammalian liver-specific microRNA, is processed from hcr mRNA and may downregulate the high affinity cationic amino acid transporter CAT-1. RNA Biol: 2004; 1 2 106 113
    • (2004) RNA Biol , vol.1 , Issue.2 , pp. 106-113
    • Chang, J.1    Nicolas, E.2    Marks, D.3
  • 23
    • 24644483623 scopus 로고    scopus 로고
    • Modulation of hepatitis C virus RNA abundance by a liver-specific MicroRNA
    • Jopling C. L., Yi M., Lancaster A. M., Lemon S. M., Sarnow P. Modulation of hepatitis C virus RNA abundance by a liver-specific MicroRNA. Science: 2005; 309 5740 1577 1581
    • (2005) Science , vol.309 , Issue.5740 , pp. 1577-1581
    • Jopling, C.L.1    Yi, M.2    Lancaster, A.M.3    Lemon, S.M.4    Sarnow, P.5
  • 24
    • 84873149944 scopus 로고    scopus 로고
    • Competing and noncompeting activities of miR-122 and the 5′ exonuclease Xrn1 in regulation of hepatitis C virus replication
    • Li Y., Masaki T., Yamane D., McGivern D. R., Lemon S. M. Competing and noncompeting activities of miR-122 and the 5′ exonuclease Xrn1 in regulation of hepatitis C virus replication. Proc Natl Acad Sci U S A: 2013; 110 5 1881 1886
    • (2013) Proc Natl Acad Sci U S A , vol.110 , Issue.5 , pp. 1881-1886
    • Li, Y.1    Masaki, T.2    Yamane, D.3    McGivern, D.R.4    Lemon, S.M.5
  • 25
    • 84877258007 scopus 로고    scopus 로고
    • Treatment of HCV infection by targeting microRNA
    • Janssen H. L., Reesink H. W., Lawitz E. J., et al. Treatment of HCV infection by targeting microRNA. N Engl J Med: 2013; 368 18 1685 1694
    • (2013) N Engl J Med , vol.368 , Issue.18 , pp. 1685-1694
    • Janssen, H.L.1    Reesink, H.W.2    Lawitz, E.J.3
  • 26
    • 39749143354 scopus 로고    scopus 로고
    • Targeted deletion reveals essential and overlapping functions of the miR-17 through 92 family of miRNA clusters
    • Ventura A., Young A. G., Winslow M. M., et al. Targeted deletion reveals essential and overlapping functions of the miR-17 through 92 family of miRNA clusters. Cell: 2008; 132 5 875 886
    • (2008) Cell , vol.132 , Issue.5 , pp. 875-886
    • Ventura, A.1    Young, A.G.2    Winslow, M.M.3
  • 27
    • 60849106298 scopus 로고    scopus 로고
    • Attribution of vascular phenotypes of the murine Egfl7 locus to the microRNA miR-126
    • Kuhnert F., Mancuso M. R., Hampton J., et al. Attribution of vascular phenotypes of the murine Egfl7 locus to the microRNA miR-126. Development: 2008; 135 24 3989 3993
    • (2008) Development , vol.135 , Issue.24 , pp. 3989-3993
    • Kuhnert, F.1    Mancuso, M.R.2    Hampton, J.3
  • 28
    • 33645075443 scopus 로고    scopus 로고
    • MiR-122 regulation of lipid metabolism revealed by in vivo antisense targeting
    • Esau C., Davis S., Murray S. F., et al. miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting. Cell Metab: 2006; 3 2 87 98
    • (2006) Cell Metab , vol.3 , Issue.2 , pp. 87-98
    • Esau, C.1    Davis, S.2    Murray, S.F.3
  • 29
    • 74249112787 scopus 로고    scopus 로고
    • Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection
    • Lanford R. E., Hildebrandt-Eriksen E. S., Petri A., et al. Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science: 2010; 327 5962 198 201
    • (2010) Science , vol.327 , Issue.5962 , pp. 198-201
    • Lanford, R.E.1    Hildebrandt-Eriksen, E.S.2    Petri, A.3
  • 30
    • 42249093319 scopus 로고    scopus 로고
    • LNA-mediated microRNA silencing in non-human primates
    • Elmén J., Lindow M., Schütz S., et al. LNA-mediated microRNA silencing in non-human primates. Nature: 2008; 452 7189 896 899
    • (2008) Nature , vol.452 , Issue.7189 , pp. 896-899
    • Elmén, J.1    Lindow, M.2    Schütz, S.3
  • 31
    • 34548316982 scopus 로고    scopus 로고
    • MicroRNA sponges: Competitive inhibitors of small RNAs in mammalian cells
    • Ebert M. S., Neilson J. R., Sharp P. A. MicroRNA sponges: competitive inhibitors of small RNAs in mammalian cells. Nat Methods: 2007; 4 9 721 726
    • (2007) Nat Methods , vol.4 , Issue.9 , pp. 721-726
    • Ebert, M.S.1    Neilson, J.R.2    Sharp, P.A.3
  • 32
    • 64549109671 scopus 로고    scopus 로고
    • Vectors expressing efficient RNA decoys achieve the long-term suppression of specific microRNA activity in mammalian cells
    • Haraguchi T., Ozaki Y., Iba H. Vectors expressing efficient RNA decoys achieve the long-term suppression of specific microRNA activity in mammalian cells. Nucleic Acids Res: 2009; 37 6 e43
    • (2009) Nucleic Acids Res , vol.37 , Issue.6 , pp. e43
    • Haraguchi, T.1    Ozaki, Y.2    Iba, H.3
  • 33
    • 58149252151 scopus 로고    scopus 로고
    • Stable knockdown of microRNA in vivo by lentiviral vectors
    • Gentner B., Schira G., Giustacchini A., et al. Stable knockdown of microRNA in vivo by lentiviral vectors. Nat Methods: 2009; 6 1 63 66
    • (2009) Nat Methods , vol.6 , Issue.1 , pp. 63-66
    • Gentner, B.1    Schira, G.2    Giustacchini, A.3
  • 34
    • 84862805889 scopus 로고    scopus 로고
    • Long-term, efficient inhibition of microRNA function in mice using rAAV vectors
    • Xie J., Ameres S. L., Friedline R., et al. Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. Nat Methods: 2012; 9 4 403 409
    • (2012) Nat Methods , vol.9 , Issue.4 , pp. 403-409
    • Xie, J.1    Ameres, S.L.2    Friedline, R.3
  • 35
    • 0028338651 scopus 로고
    • Interaction of the adeno-associated virus Rep protein with a sequence within the A palindrome of the viral terminal repeat
    • McCarty D. M., Ryan J. H., Zolotukhin S., Zhou X., Muzyczka N. Interaction of the adeno-associated virus Rep protein with a sequence within the A palindrome of the viral terminal repeat. J Virol: 1994; 68 8 4998 5006
    • (1994) J Virol , vol.68 , Issue.8 , pp. 4998-5006
    • McCarty, D.M.1    Ryan, J.H.2    Zolotukhin, S.3    Zhou, X.4    Muzyczka, N.5
  • 36
    • 0027170411 scopus 로고
    • Features of the adeno-associated virus origin involved in substrate recognition by the viral Rep protein
    • Snyder R. O., Im D. S., Ni T., Xiao X., Samulski R. J., Muzyczka N. Features of the adeno-associated virus origin involved in substrate recognition by the viral Rep protein. J Virol: 1993; 67 10 6096 6104
    • (1993) J Virol , vol.67 , Issue.10 , pp. 6096-6104
    • Snyder, R.O.1    Im, D.S.2    Ni, T.3    Xiao, X.4    Samulski, R.J.5    Muzyczka, N.6
  • 37
    • 0032826095 scopus 로고    scopus 로고
    • Adeno-associated virus type 2 protein interactions: Formation of pre-encapsidation complexes
    • Dubielzig R., King J. A., Weger S., Kern A., Kleinschmidt J. A. Adeno-associated virus type 2 protein interactions: formation of pre-encapsidation complexes. J Virol: 1999; 73 11 8989 8998
    • (1999) J Virol , vol.73 , Issue.11 , pp. 8989-8998
    • Dubielzig, R.1    King, J.A.2    Weger, S.3    Kern, A.4    Kleinschmidt, J.A.5
  • 38
    • 0028331815 scopus 로고
    • Analysis of adeno-associated virus (AAV) wild-type and mutant Rep proteins for their abilities to negatively regulate AAV p5 and p19 mRNA levels
    • Kyöstiö S. R., Owens R. A., Weitzman M. D., Antoni B. A., Chejanovsky N., Carter B. J. Analysis of adeno-associated virus (AAV) wild-type and mutant Rep proteins for their abilities to negatively regulate AAV p5 and p19 mRNA levels. J Virol: 1994; 68 5 2947 2957
    • (1994) J Virol , vol.68 , Issue.5 , pp. 2947-2957
    • Kyöstiö, S.R.1    Owens, R.A.2    Weitzman, M.D.3    Antoni, B.A.4    Chejanovsky, N.5    Carter, B.J.6
  • 39
    • 0013914098 scopus 로고
    • Studies of small DNA viruses found in various adenovirus preparations: Physical, biological, and immunological characteristics
    • Hoggan M. D., Blacklow N. R., Rowe W. P. Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics. Proc Natl Acad Sci U S A: 1966; 55 6 1467 1474
    • (1966) Proc Natl Acad Sci U S A , vol.55 , Issue.6 , pp. 1467-1474
    • Hoggan, M.D.1    Blacklow, N.R.2    Rowe, W.P.3
  • 40
    • 0019802153 scopus 로고
    • Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication
    • Buller R. M., Janik J. E., Sebring E. D., Rose J. A. Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J Virol: 1981; 40 1 241 247
    • (1981) J Virol , vol.40 , Issue.1 , pp. 241-247
    • Buller, R.M.1    Janik, J.E.2    Sebring, E.D.3    Rose, J.A.4
  • 41
    • 0034531297 scopus 로고    scopus 로고
    • Building a better vector: The manipulation of AAV virions
    • Rabinowitz J. E., Samulski R. J. Building a better vector: the manipulation of AAV virions. Virology: 2000; 278 2 301 308
    • (2000) Virology , vol.278 , Issue.2 , pp. 301-308
    • Rabinowitz, J.E.1    Samulski, R.J.2
  • 42
    • 84857602982 scopus 로고    scopus 로고
    • Adeno-associated virus vectorology, manufacturing, and clinical applications
    • Grieger J. C., Samulski R. J. Adeno-associated virus vectorology, manufacturing, and clinical applications. Methods Enzymol: 2012; 507 229 254
    • (2012) Methods Enzymol , vol.507 , pp. 229-254
    • Grieger, J.C.1    Samulski, R.J.2
  • 43
    • 84859440283 scopus 로고    scopus 로고
    • The AAV vector toolkit: Poised at the clinical crossroads
    • Asokan A., Schaffer D. V., Samulski R. J. The AAV vector toolkit: poised at the clinical crossroads. Mol Ther: 2012; 20 4 699 708
    • (2012) Mol Ther , vol.20 , Issue.4 , pp. 699-708
    • Asokan, A.1    Schaffer, D.V.2    Samulski, R.J.3
  • 44
    • 79954622209 scopus 로고    scopus 로고
    • Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
    • Mingozzi F., High K. A. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet: 2011; 12 5 341 355
    • (2011) Nat Rev Genet , vol.12 , Issue.5 , pp. 341-355
    • Mingozzi, F.1    High, K.A.2
  • 45
    • 48949120141 scopus 로고    scopus 로고
    • Recent developments in adeno-associated virus vector technology
    • Büning H., Perabo L., Coutelle O., Quadt-Humme S., Hallek M. Recent developments in adeno-associated virus vector technology. J Gene Med: 2008; 10 7 717 733
    • (2008) J Gene Med , vol.10 , Issue.7 , pp. 717-733
    • Büning, H.1    Perabo, L.2    Coutelle, O.3    Quadt-Humme, S.4    Hallek, M.5
  • 46
    • 0025214094 scopus 로고
    • Site-specific integration by adeno-associated virus
    • Kotin R. M., Siniscalco M., Samulski R. J., et al. Site-specific integration by adeno-associated virus. Proc Natl Acad Sci U S A: 1990; 87 6 2211 2215
    • (1990) Proc Natl Acad Sci U S A , vol.87 , Issue.6 , pp. 2211-2215
    • Kotin, R.M.1    Siniscalco, M.2    Samulski, R.J.3
  • 47
    • 0026070440 scopus 로고
    • Targeted integration of adeno-associated virus (AAV) into human chromosome 19
    • Samulski R. J., Zhu X., Xiao X., et al. Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J: 1991; 10 12 3941 3950
    • (1991) EMBO J , vol.10 , Issue.12 , pp. 3941-3950
    • Samulski, R.J.1    Zhu, X.2    Xiao, X.3
  • 48
    • 49149113894 scopus 로고    scopus 로고
    • Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle
    • Penaud-Budloo M., Le Guiner C., Nowrouzi A., et al. Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J Virol: 2008; 82 16 7875 7885
    • (2008) J Virol , vol.82 , Issue.16 , pp. 7875-7885
    • Penaud-Budloo, M.1    Le Guiner, C.2    Nowrouzi, A.3
  • 49
    • 79953088521 scopus 로고    scopus 로고
    • Assessing the potential for AAV vector genotoxicity in a murine model
    • Li H., Malani N., Hamilton S. R., et al. Assessing the potential for AAV vector genotoxicity in a murine model. Blood: 2011; 117 12 3311 3319
    • (2011) Blood , vol.117 , Issue.12 , pp. 3311-3319
    • Li, H.1    Malani, N.2    Hamilton, S.R.3
  • 50
    • 84880289352 scopus 로고    scopus 로고
    • A largely random AAV integration profile after LPLD gene therapy
    • Kaeppel C., Beattie S. G., Fronza R., et al. A largely random AAV integration profile after LPLD gene therapy. Nat Med: 2013; 19 7 889 891
    • (2013) Nat Med , vol.19 , Issue.7 , pp. 889-891
    • Kaeppel, C.1    Beattie, S.G.2    Fronza, R.3
  • 51
    • 84877865566 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction
    • Zhong L., Malani N., Li M., et al. Recombinant adeno-associated virus integration sites in murine liver after ornithine transcarbamylase gene correction. Hum Gene Ther: 2013; 24 5 520 525
    • (2013) Hum Gene Ther , vol.24 , Issue.5 , pp. 520-525
    • Zhong, L.1    Malani, N.2    Li, M.3
  • 52
    • 84882394773 scopus 로고    scopus 로고
    • Lessons learned from the clinical development and market authorization of Glybera
    • Bryant L. M., Christopher D. M., Giles A. R., et al. Lessons learned from the clinical development and market authorization of Glybera. Hum Gene Ther Clin Dev: 2013; 24 2 55 64
    • (2013) Hum Gene Ther Clin Dev , vol.24 , Issue.2 , pp. 55-64
    • Bryant, L.M.1    Christopher, D.M.2    Giles, A.R.3
  • 53
    • 0348038748 scopus 로고    scopus 로고
    • Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo
    • Wang Z., Ma H. I., Li J., Sun L., Zhang J., Xiao X. Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther: 2003; 10 26 2105 2111
    • (2003) Gene Ther , vol.10 , Issue.26 , pp. 2105-2111
    • Wang, Z.1    Ma, H.I.2    Li, J.3    Sun, L.4    Zhang, J.5    Xiao, X.6
  • 54
    • 0346777307 scopus 로고    scopus 로고
    • Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
    • McCarty D. M., Fu H., Monahan P. E., Toulson C. E., Naik P., Samulski R. J. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther: 2003; 10 26 2112 2118
    • (2003) Gene Ther , vol.10 , Issue.26 , pp. 2112-2118
    • McCarty, D.M.1    Fu, H.2    Monahan, P.E.3    Toulson, C.E.4    Naik, P.5    Samulski, R.J.6
  • 55
    • 41649114199 scopus 로고    scopus 로고
    • Suppression of non-small cell lung tumor development by the let-7 microRNA family
    • Kumar M. S., Erkeland S. J., Pester R. E., et al. Suppression of non-small cell lung tumor development by the let-7 microRNA family. Proc Natl Acad Sci U S A: 2008; 105 10 3903 3908
    • (2008) Proc Natl Acad Sci U S A , vol.105 , Issue.10 , pp. 3903-3908
    • Kumar, M.S.1    Erkeland, S.J.2    Pester, R.E.3
  • 56
    • 84880288761 scopus 로고    scopus 로고
    • MicroRNA-30c reduces hyperlipidemia and atherosclerosis in mice by decreasing lipid synthesis and lipoprotein secretion
    • Soh J., Iqbal J., Queiroz J., Fernandez-Hernando C., Hussain M. M. MicroRNA-30c reduces hyperlipidemia and atherosclerosis in mice by decreasing lipid synthesis and lipoprotein secretion. Nat Med: 2013; 19 7 892 900
    • (2013) Nat Med , vol.19 , Issue.7 , pp. 892-900
    • Soh, J.1    Iqbal, J.2    Queiroz, J.3    Fernandez-Hernando, C.4    Hussain, M.M.5
  • 57
    • 84872556705 scopus 로고    scopus 로고
    • Potent microRNA suppression by RNA Pol II-transcribed 'Tough Decoy' inhibitors
    • Bak R. O., Hollensen A. K., Primo M. N., Sorensen C. D., Mikkelsen J. G. Potent microRNA suppression by RNA Pol II-transcribed 'Tough Decoy' inhibitors. RNA: 2013; 19 2 280 293
    • (2013) RNA , vol.19 , Issue.2 , pp. 280-293
    • Bak, R.O.1    Hollensen, A.K.2    Primo, M.N.3    Sorensen, C.D.4    Mikkelsen, J.G.5
  • 58
    • 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 R. J. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol: 1998; 72 3 2224 2232
    • (1998) J Virol , vol.72 , Issue.3 , pp. 2224-2232
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 59
    • 34347252689 scopus 로고    scopus 로고
    • Production and characterization of adeno-associated viral vectors
    • Grieger J. C., Choi V. W., Samulski R. J. Production and characterization of adeno-associated viral vectors. Nat Protoc: 2006; 1 3 1412 1428
    • (2006) Nat Protoc , vol.1 , Issue.3 , pp. 1412-1428
    • Grieger, J.C.1    Choi, V.W.2    Samulski, R.J.3
  • 61
    • 79961032418 scopus 로고    scopus 로고
    • Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
    • Zhang H., Yang B., Mu X., et al. Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol Ther: 2011; 19 8 1440 1448
    • (2011) Mol Ther , vol.19 , Issue.8 , pp. 1440-1448
    • Zhang, H.1    Yang, B.2    Mu, X.3
  • 62
    • 84903760379 scopus 로고    scopus 로고
    • Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10
    • Yang B., Li S., Wang H., et al. Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther: 2014; 22 7 1299 1309
    • (2014) Mol Ther , vol.22 , Issue.7 , pp. 1299-1309
    • Yang, B.1    Li, S.2    Wang, H.3
  • 64
    • 84863714144 scopus 로고    scopus 로고
    • Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2
    • Miyazaki Y., Adachi H., Katsuno M., et al. Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2. Nat Med: 2012; 18 7 1136 1141
    • (2012) Nat Med , vol.18 , Issue.7 , pp. 1136-1141
    • Miyazaki, Y.1    Adachi, H.2    Katsuno, M.3
  • 65
    • 84864761391 scopus 로고    scopus 로고
    • Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver
    • Hsu S. H., Wang B., Kota J., et al. Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver. J Clin Invest: 2012; 122 8 2871 2883
    • (2012) J Clin Invest , vol.122 , Issue.8 , pp. 2871-2883
    • Hsu, S.H.1    Wang, B.2    Kota, J.3
  • 66
    • 84864773072 scopus 로고    scopus 로고
    • MicroRNA-122 plays a critical role in liver homeostasis and hepatocarcinogenesis
    • Tsai W. C., Hsu S. D., Hsu C. S., et al. MicroRNA-122 plays a critical role in liver homeostasis and hepatocarcinogenesis. J Clin Invest: 2012; 122 8 2884 2897
    • (2012) J Clin Invest , vol.122 , Issue.8 , pp. 2884-2897
    • Tsai, W.C.1    Hsu, S.D.2    Hsu, C.S.3
  • 67
    • 84867170366 scopus 로고    scopus 로고
    • MiR-26a is required for skeletal muscle differentiation and regeneration in mice
    • Dey B. K., Gagan J., Yan Z., Dutta A. miR-26a is required for skeletal muscle differentiation and regeneration in mice. Genes Dev: 2012; 26 19 2180 2191
    • (2012) Genes Dev , vol.26 , Issue.19 , pp. 2180-2191
    • Dey, B.K.1    Gagan, J.2    Yan, Z.3    Dutta, A.4
  • 68
    • 84868475728 scopus 로고    scopus 로고
    • MicroRNA therapeutics for cardiovascular disease: Opportunities and obstacles
    • van Rooij E., Olson E. N. MicroRNA therapeutics for cardiovascular disease: opportunities and obstacles. Nat Rev Drug Discov: 2012; 11 11 860 872
    • (2012) Nat Rev Drug Discov , vol.11 , Issue.11 , pp. 860-872
    • Van Rooij, E.1    Olson, E.N.2
  • 69
    • 33749479908 scopus 로고    scopus 로고
    • Downregulation of miR-122 in the rodent and human hepatocellular carcinomas
    • Kutay H., Bai S., Datta J., et al. Downregulation of miR-122 in the rodent and human hepatocellular carcinomas. J Cell Biochem: 2006; 99 3 671 678
    • (2006) J Cell Biochem , vol.99 , Issue.3 , pp. 671-678
    • Kutay, H.1    Bai, S.2    Datta, J.3
  • 70
    • 70349976819 scopus 로고    scopus 로고
    • Loss of miR-122 expression in liver cancer correlates with suppression of the hepatic phenotype and gain of metastatic properties
    • Coulouarn C., Factor V. M., Andersen J. B., Durkin M. E., Thorgeirsson S. S. Loss of miR-122 expression in liver cancer correlates with suppression of the hepatic phenotype and gain of metastatic properties. Oncogene: 2009; 28 40 3526 3536
    • (2009) Oncogene , vol.28 , Issue.40 , pp. 3526-3536
    • Coulouarn, C.1    Factor, V.M.2    Andersen, J.B.3    Durkin, M.E.4    Thorgeirsson, S.S.5
  • 71
    • 66149114383 scopus 로고    scopus 로고
    • MicroRNA-122, a tumor suppressor microRNA that regulates intrahepatic metastasis of hepatocellular carcinoma
    • Tsai W. C., Hsu P. W., Lai T. C., et al. MicroRNA-122, a tumor suppressor microRNA that regulates intrahepatic metastasis of hepatocellular carcinoma. Hepatology: 2009; 49 5 1571 1582
    • (2009) Hepatology , vol.49 , Issue.5 , pp. 1571-1582
    • Tsai, W.C.1    Hsu, P.W.2    Lai, T.C.3
  • 72
    • 84862762321 scopus 로고    scopus 로고
    • MicroRNA-26a promotes cholangiocarcinoma growth by activating β-catenin
    • Zhang J., Han C., Wu T. MicroRNA-26a promotes cholangiocarcinoma growth by activating β-catenin. Gastroenterology: 2012; 143 1 246 56.e8
    • (2012) Gastroenterology , vol.143 , Issue.1 , pp. 246-56e8
    • Zhang, J.1    Han, C.2    Wu, T.3
  • 73
    • 83155181655 scopus 로고    scopus 로고
    • The Wnt/β-catenin pathway as a therapeutic target in human hepatocellular carcinoma
    • Dahmani R., Just P. A., Perret C. The Wnt/β-catenin pathway as a therapeutic target in human hepatocellular carcinoma. Clin Res Hepatol Gastroenterol: 2011; 35 11 709 713
    • (2011) Clin Res Hepatol Gastroenterol , vol.35 , Issue.11 , pp. 709-713
    • Dahmani, R.1    Just, P.A.2    Perret, C.3
  • 74
    • 66149173789 scopus 로고    scopus 로고
    • The PTEN-regulating microRNA miR-26a is amplified in high-grade glioma and facilitates gliomagenesis in vivo
    • Huse J. T., Brennan C., Hambardzumyan D., et al. The PTEN-regulating microRNA miR-26a is amplified in high-grade glioma and facilitates gliomagenesis in vivo. Genes Dev: 2009; 23 11 1327 1337
    • (2009) Genes Dev , vol.23 , Issue.11 , pp. 1327-1337
    • Huse, J.T.1    Brennan, C.2    Hambardzumyan, D.3
  • 75
    • 84862217953 scopus 로고    scopus 로고
    • The dose can make the poison: Lessons learned from adverse in vivo toxicities caused by RNAi overexpression
    • Grimm D. The dose can make the poison: lessons learned from adverse in vivo toxicities caused by RNAi overexpression. Silence: 2011; 2 8
    • (2011) Silence , vol.2 , pp. 8
    • Grimm, D.1
  • 76
    • 33745299723 scopus 로고    scopus 로고
    • Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
    • Grimm D., Streetz K. L., Jopling C. L., et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature: 2006; 441 7092 537 541
    • (2006) Nature , vol.441 , Issue.7092 , pp. 537-541
    • Grimm, D.1    Streetz, K.L.2    Jopling, C.L.3
  • 77
    • 77956355233 scopus 로고    scopus 로고
    • Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver
    • Grimm D., Wang L., Lee J. S., et al. Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest: 2010; 120 9 3106 3119
    • (2010) J Clin Invest , vol.120 , Issue.9 , pp. 3106-3119
    • Grimm, D.1    Wang, L.2    Lee, J.S.3
  • 78
    • 58149232358 scopus 로고    scopus 로고
    • Artificial microRNAs as siRNA shuttles: Improved safety as compared to shRNAs in vitro and in vivo
    • Boudreau R. L., Martins I., Davidson B. L. Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol Ther: 2009; 17 1 169 175
    • (2009) Mol Ther , vol.17 , Issue.1 , pp. 169-175
    • Boudreau, R.L.1    Martins, I.2    Davidson, B.L.3
  • 79
    • 44449121785 scopus 로고    scopus 로고
    • Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
    • McBride J. L., Boudreau R. L., Harper S. Q., et al. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A: 2008; 105 15 5868 5873
    • (2008) Proc Natl Acad Sci U S A , vol.105 , Issue.15 , pp. 5868-5873


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.