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Volumn 87, Issue 9, 2007, Pages 893-902

In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA

Author keywords

1 antitrypsin; PI; rAAV; siRNA; Z

Indexed keywords

ADENOVIRUS VECTOR; ALPHA 1 ANTITRYPSIN; RECOMBINANT ALPHA 1 ANTITRYPSIN; RECOMBINANT PROTEIN; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

EID: 34547943486     PISSN: 00236837     EISSN: 15300307     Source Type: Journal    
DOI: 10.1038/labinvest.3700629     Document Type: Article
Times cited : (59)

References (45)
  • 1
    • 0024842004 scopus 로고
    • The alpha 1-antitrypsin gene and its deficiency states
    • Crystal RG. The alpha 1-antitrypsin gene and its deficiency states. Trends Genet 1989;5:411-417.
    • (1989) Trends Genet , vol.5 , pp. 411-417
    • Crystal, R.G.1
  • 2
    • 0025350658 scopus 로고    scopus 로고
    • Crystal RG. Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy. J Clin Invest 1990;85:1343-1352.
    • Crystal RG. Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy. J Clin Invest 1990;85:1343-1352.
  • 4
    • 0029794112 scopus 로고    scopus 로고
    • Perlmutter DH. Alpha-1-antitrypsin deficiency: biochemistry and clinical manifestations. Ann Med 1996;28:385-394.
    • Perlmutter DH. Alpha-1-antitrypsin deficiency: biochemistry and clinical manifestations. Ann Med 1996;28:385-394.
  • 5
    • 0029859978 scopus 로고    scopus 로고
    • Molecular pathogenesis of liver disease in alpha1-antitrypsin deficiency
    • Teckman JH, Qu D, Perlmutter DH. Molecular pathogenesis of liver disease in alpha1-antitrypsin deficiency. Hepatology 1996;24:1504-1516.
    • (1996) Hepatology , vol.24 , pp. 1504-1516
    • Teckman, J.H.1    Qu, D.2    Perlmutter, D.H.3
  • 6
    • 0025300409 scopus 로고
    • Histopathology of alpha 1-antitrypsin liver disease in a transgenic mouse model
    • Geller SA, Nichols WS, Dycaico MJ, et al. Histopathology of alpha 1-antitrypsin liver disease in a transgenic mouse model. Hepatology 1990;12:40-47.
    • (1990) Hepatology , vol.12 , pp. 40-47
    • Geller, S.A.1    Nichols, W.S.2    Dycaico, M.J.3
  • 7
    • 0027473016 scopus 로고
    • Effect of the Z mutation on the physical and inhibitory properties of alpha 1-antitrypsin
    • Lomas DA, Evans DL, Stone SR, et al. Effect of the Z mutation on the physical and inhibitory properties of alpha 1-antitrypsin. Biochemistry 1993;32:500-508.
    • (1993) Biochemistry , vol.32 , pp. 500-508
    • Lomas, D.A.1    Evans, D.L.2    Stone, S.R.3
  • 8
    • 0028260565 scopus 로고
    • Loop-sheet polymerization: The structural basis of Z alpha 1-antitrypsin accumulation in the liver
    • Lomas DA. Loop-sheet polymerization: the structural basis of Z alpha 1-antitrypsin accumulation in the liver. Clin Sci Lond 1994;86:489-495.
    • (1994) Clin Sci Lond , vol.86 , pp. 489-495
    • Lomas, D.A.1
  • 9
    • 0033867065 scopus 로고    scopus 로고
    • Loop-sheet polymerization: The mechanism of alpha1-antitrypsin deficiency
    • Lomas DA. Loop-sheet polymerization: the mechanism of alpha1-antitrypsin deficiency. Respir Med 2000;94(Suppl C):S3-S6.
    • (2000) Respir Med , vol.94 , Issue.SUPPL. C
    • Lomas, D.A.1
  • 10
    • 85047684827 scopus 로고    scopus 로고
    • Alpha1-antitrypsin polymerization and the serpinopathies: Pathobiology and prospects for therapy
    • Lomas DA, Mahadeva R. Alpha1-antitrypsin polymerization and the serpinopathies: pathobiology and prospects for therapy. J Clin Invest 2002;110:1585-1590.
    • (2002) J Clin Invest , vol.110 , pp. 1585-1590
    • Lomas, D.A.1    Mahadeva, R.2
  • 11
    • 0036547631 scopus 로고    scopus 로고
    • Hypersensitive mousetraps, alpha1-antitrypsin deficiency and dementia
    • Lomas DA, Lourbakos A, Cumming SA, et al. Hypersensitive mousetraps, alpha1-antitrypsin deficiency and dementia. Biochem Soc Trans 2002;30:89-92.
    • (2002) Biochem Soc Trans , vol.30 , pp. 89-92
    • Lomas, D.A.1    Lourbakos, A.2    Cumming, S.A.3
  • 12
    • 2942638034 scopus 로고    scopus 로고
    • Alpha1-antitrypsin deficiency, 4: Molecular pathophysiology
    • Lomas DA, Parfrey H. Alpha1-antitrypsin deficiency, 4: molecular pathophysiology. Thorax 2004;59:529-535.
    • (2004) Thorax , vol.59 , pp. 529-535
    • Lomas, D.A.1    Parfrey, H.2
  • 13
    • 33645057629 scopus 로고    scopus 로고
    • Polymerisation underlies alpha1-antitrypsin deficiency, dementia and other serpinopathies
    • Lomas DA, Belorgey D, Mallya M, et al. Polymerisation underlies alpha1-antitrypsin deficiency, dementia and other serpinopathies. Front Biosci 2004;9:2873-2891.
    • (2004) Front Biosci , vol.9 , pp. 2873-2891
    • Lomas, D.A.1    Belorgey, D.2    Mallya, M.3
  • 14
    • 21744457450 scopus 로고    scopus 로고
    • Molecular mousetraps, alpha1-antitrypsin deficiency and the serpinopathies
    • Lomas DA. Molecular mousetraps, alpha1-antitrypsin deficiency and the serpinopathies. Clin Med 2005;5:249-257.
    • (2005) Clin Med , vol.5 , pp. 249-257
    • Lomas, D.A.1
  • 15
    • 0037011795 scopus 로고    scopus 로고
    • Alpha1-antitrypsin deficiency - a model for conformational diseases
    • Carrell RW, Lomas DA. Alpha1-antitrypsin deficiency - a model for conformational diseases. N Engl J Med 2002;346:45-53.
    • (2002) N Engl J Med , vol.346 , pp. 45-53
    • Carrell, R.W.1    Lomas, D.A.2
  • 16
    • 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, et al. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci USA 1998;95:14384-14388.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 14384-14388
    • Song, S.1    Morgan, M.2    Ellis, T.3
  • 17
    • 0034828903 scopus 로고    scopus 로고
    • Stable therapeutic serum levels of human alpha-1 antitrypsin AAT; after portal vein injection of recombinant adeno-associated virus rAAV; vectors
    • Song S, Embury J, Laipis PJ, et al. Stable therapeutic serum levels of human alpha-1 antitrypsin AAT; after portal vein injection of recombinant adeno-associated virus rAAV; vectors. Gene Therapy 2001;8:1299-1306.
    • (2001) Gene Therapy , vol.8 , pp. 1299-1306
    • Song, S.1    Embury, J.2    Laipis, P.J.3
  • 18
    • 0036764793 scopus 로고    scopus 로고
    • Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin rAAV-SERPINA1; vectors in a nonhuman primate model: Safety and immunologic aspects
    • Song S, Scott-Jorgensen M, Wang J, et al. Intramuscular administration of recombinant adeno-associated virus 2 alpha-1 antitrypsin rAAV-SERPINA1; vectors in a nonhuman primate model: safety and immunologic aspects. Mol Ther 2002;6:329-335.
    • (2002) Mol Ther , vol.6 , pp. 329-335
    • Song, S.1    Scott-Jorgensen, M.2    Wang, J.3
  • 19
    • 27744533048 scopus 로고    scopus 로고
    • Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector
    • Conlon TJ, Cossette T, Erger K, et al. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. Mol Ther 2005;12:867-875.
    • (2005) Mol Ther , vol.12 , pp. 867-875
    • Conlon, T.J.1    Cossette, T.2    Erger, K.3
  • 20
    • 33745241652 scopus 로고    scopus 로고
    • Therapeutic level of functional human alpha 1 antitrypsin hAAT; secreted from murine muscle transduced by adeno-associated virus rAAV1; vector
    • Lu Y, Choi YK, Campbell-Thompson M, et al. Therapeutic level of functional human alpha 1 antitrypsin hAAT; secreted from murine muscle transduced by adeno-associated virus rAAV1; vector. J Gene Med 2006;8:730-735.
    • (2006) J Gene Med , vol.8 , pp. 730-735
    • Lu, Y.1    Choi, Y.K.2    Campbell-Thompson, M.3
  • 21
    • 0742307402 scopus 로고    scopus 로고
    • Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin rAAV2-CB-hAAT; gene vector to AAT-deficient adults
    • Flotte TR, Brantly ML, Spencer LT, et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin rAAV2-CB-hAAT; gene vector to AAT-deficient adults. Hum Gene Ther 2004;15:93-128.
    • (2004) Hum Gene Ther , vol.15 , pp. 93-128
    • Flotte, T.R.1    Brantly, M.L.2    Spencer, L.T.3
  • 22
    • 33845807816 scopus 로고    scopus 로고
    • Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alpha1;antitrypsin AAT; vector in AAT-deficient adults
    • Brantly ML, Spencer LT, Humphries M, et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alpha1;antitrypsin AAT; vector in AAT-deficient adults. Hum Gene Ther 2006;17:1177-1186.
    • (2006) Hum Gene Ther , vol.17 , pp. 1177-1186
    • Brantly, M.L.1    Spencer, L.T.2    Humphries, M.3
  • 23
    • 0032922168 scopus 로고    scopus 로고
    • A novel SV40-based vector successfully transduces and expresses an alpha 1-antitrypsin ribozyme in a human hepatoma-derived cell line
    • Zern MA, Ozaki I, Duan L, et al. A novel SV40-based vector successfully transduces and expresses an alpha 1-antitrypsin ribozyme in a human hepatoma-derived cell line. Gene Therapy 1999;6:114-120.
    • (1999) Gene Therapy , vol.6 , pp. 114-120
    • Zern, M.A.1    Ozaki, I.2    Duan, L.3
  • 24
    • 0031775494 scopus 로고    scopus 로고
    • Adeno-associated virus as a vector for liver-directed gene therapy
    • Xiao W, Berta SC, Lu MM, et al. Adeno-associated virus as a vector for liver-directed gene therapy. J Virol 1998;72:10222-10226.
    • (1998) J Virol , vol.72 , pp. 10222-10226
    • Xiao, W.1    Berta, S.C.2    Lu, M.M.3
  • 25
    • 0034182612 scopus 로고    scopus 로고
    • In vivo selection of hepatocytes transduced with adeno-associated viral vectors
    • Chen SJ, Tazelaar J, Moscioni AD, et al. In vivo selection of hepatocytes transduced with adeno-associated viral vectors. Mol Ther 2000;1:414-422.
    • (2000) Mol Ther , vol.1 , pp. 414-422
    • Chen, S.J.1    Tazelaar, J.2    Moscioni, A.D.3
  • 27
    • 33744900338 scopus 로고    scopus 로고
    • High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes
    • Gao GP, Lu Y, Sun X, et al. High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes. J Virol 2006;80:6192-6194.
    • (2006) J Virol , vol.80 , pp. 6192-6194
    • Gao, G.P.1    Lu, Y.2    Sun, X.3
  • 28
    • 33645992763 scopus 로고    scopus 로고
    • Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype
    • Grimm D, Pandey K, Nakai HS, et al. Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype. J Virol 2006;80:426-439.
    • (2006) J Virol , vol.80 , pp. 426-439
    • Grimm, D.1    Pandey, K.2    Nakai, H.S.3
  • 29
    • 0035803583 scopus 로고    scopus 로고
    • Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate
    • Elbashir SM, Martinez J, Patkaniowska A, et al. Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate. EMBO J 2001;20:6877-6888.
    • (2001) EMBO J , vol.20 , pp. 6877-6888
    • Elbashir, S.M.1    Martinez, J.2    Patkaniowska, A.3
  • 30
    • 0035942736 scopus 로고    scopus 로고
    • Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    • Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001;411:494-498.
    • (2001) Nature , vol.411 , pp. 494-498
    • Elbashir, S.M.1    Harborth, J.2    Lendeckel, W.3
  • 31
    • 0035863097 scopus 로고    scopus 로고
    • RNA interference is mediated by 21- and 22-nucleotide RNAs
    • Elbashir SM, Lendeckel W, Tuschl T. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev 2001;15:188-200.
    • (2001) Genes Dev , vol.15 , pp. 188-200
    • Elbashir, S.M.1    Lendeckel, W.2    Tuschl, T.3
  • 32
    • 0036428801 scopus 로고    scopus 로고
    • Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
    • Zolotukhin S, Potter M, Zolotukhin I, et al. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 2002;28:158-167.
    • (2002) Methods , vol.28 , pp. 158-167
    • Zolotukhin, S.1    Potter, M.2    Zolotukhin, I.3
  • 33
    • 0041375553 scopus 로고    scopus 로고
    • Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver
    • Loiler SA, Conlon TJ, Song S, et al. Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver. Gene Therapy 2003;10:1551-1558.
    • (2003) Gene Therapy , vol.10 , pp. 1551-1558
    • Loiler, S.A.1    Conlon, T.J.2    Song, S.3
  • 34
    • 0023689993 scopus 로고
    • Multiple tissues express alpha 1-antitrypsin in transgenic mice and man
    • Carlson JA, Rogers BB, Sifers RN, et al. Multiple tissues express alpha 1-antitrypsin in transgenic mice and man. J Clin Invest 1988;82:26-36.
    • (1988) J Clin Invest , vol.82 , pp. 26-36
    • Carlson, J.A.1    Rogers, B.B.2    Sifers, R.N.3
  • 35
    • 0024547734 scopus 로고
    • Accumulation of PiZ alpha 1-antitrypsin causes liver damage in transgenic mice
    • Carlson JA, Rogers BB, Sifers RN, et al. Accumulation of PiZ alpha 1-antitrypsin causes liver damage in transgenic mice. J Clin Invest 1989;83:1183-1190.
    • (1989) J Clin Invest , vol.83 , pp. 1183-1190
    • Carlson, J.A.1    Rogers, B.B.2    Sifers, R.N.3
  • 36
    • 0024239161 scopus 로고
    • Neonatal hepatitis induced by alpha 1-antitrypsin: A transgenic mouse model
    • Dycaico MJ, Grant SG, Felts K, et al. Neonatal hepatitis induced by alpha 1-antitrypsin: a transgenic mouse model. Science 1988;242:1409-1412.
    • (1988) Science , vol.242 , pp. 1409-1412
    • Dycaico, M.J.1    Grant, S.G.2    Felts, K.3
  • 37
    • 0024802557 scopus 로고
    • Neonatal growth delay in alpha-1-antitrypsin disease. Influence of genetic background
    • Dycaico MJ, Felts K, Nichols SW, et al. Neonatal growth delay in alpha-1-antitrypsin disease. Influence of genetic background. Mol Biol Med 1989;6:137-141.
    • (1989) Mol Biol Med , vol.6 , pp. 137-141
    • Dycaico, M.J.1    Felts, K.2    Nichols, S.W.3
  • 38
    • 11244346111 scopus 로고    scopus 로고
    • Quantitative isolation of alphalAT mutant Z protein polymers from human and mouse livers and the effect of heat
    • An JK, Blomenkamp K, Lindblad D, et al. Quantitative isolation of alphalAT mutant Z protein polymers from human and mouse livers and the effect of heat. Hepatology 2005;41:160-167.
    • (2005) Hepatology , vol.41 , pp. 160-167
    • An, J.K.1    Blomenkamp, K.2    Lindblad, D.3
  • 39
    • 1842532017 scopus 로고    scopus 로고
    • Analyses of hepatocellular proliferation in a mouse model of alpha-1-antitrypsin deficiency
    • Rudnick DA, Liao Y, An JK, et al. Analyses of hepatocellular proliferation in a mouse model of alpha-1-antitrypsin deficiency. Hepatology 2004;39:1048-1055.
    • (2004) Hepatology , vol.39 , pp. 1048-1055
    • Rudnick, D.A.1    Liao, Y.2    An, J.K.3
  • 40
    • 33750611304 scopus 로고    scopus 로고
    • Indomethacin increases liver damage in a murine model of liver injury from alpha-1-antitrypsin deficiency
    • Rudnick DA, Shikapwashya O, Blomenkamp K, et al. Indomethacin increases liver damage in a murine model of liver injury from alpha-1-antitrypsin deficiency. Hepatology 2006;44:976-982.
    • (2006) Hepatology , vol.44 , pp. 976-982
    • Rudnick, D.A.1    Shikapwashya, O.2    Blomenkamp, K.3
  • 41
    • 0037015049 scopus 로고    scopus 로고
    • Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
    • Gao GP, Alvira MR, Wang L, et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 2002;99:11854-11859.
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 11854-11859
    • Gao, G.P.1    Alvira, M.R.2    Wang, L.3
  • 42
    • 0141482003 scopus 로고    scopus 로고
    • Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy
    • Grimm D, Zhou S, Nakai H, et al. Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 2003;102:2412-2419.
    • (2003) Blood , vol.102 , pp. 2412-2419
    • Grimm, D.1    Zhou, S.2    Nakai, H.3
  • 43
    • 0034030443 scopus 로고    scopus 로고
    • Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: Cell cycling does not influence hepatocyte transduction
    • Miao CH, Nakai H, Thompson AR, et al. Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction. J Virol 2000;74:3793-3803.
    • (2000) J Virol , vol.74 , pp. 3793-3803
    • Miao, C.H.1    Nakai, H.2    Thompson, A.R.3
  • 44
    • 0036828203 scopus 로고    scopus 로고
    • A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction
    • Nakai H, Thomas CE, Storm TA, et al. A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. J Virol 2002;76:11343-11349.
    • (2002) J Virol , vol.76 , pp. 11343-11349
    • Nakai, H.1    Thomas, C.E.2    Storm, T.A.3
  • 45
    • 33745299723 scopus 로고    scopus 로고
    • Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
    • Grimm D, Streetz KL, Jopling CL, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006;441:537-541.
    • (2006) Nature , vol.441 , pp. 537-541
    • Grimm, D.1    Streetz, K.L.2    Jopling, C.L.3


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