메뉴 건너뛰기




Volumn 20, Issue 3, 2012, Pages 590-600

Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 ANTITRYPSIN; MICRORNA; PARVOVIRUS VECTOR; SHORT HAIRPIN RNA;

EID: 84857787568     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.292     Document Type: Article
Times cited : (94)

References (33)
  • 1
  • 2
    • 0034721790 scopus 로고    scopus 로고
    • Pathogenic alpha 1-antitrypsin polymers are formed by reactive loop-beta-sheet A linkage
    • Sivasothy, P, Dafforn, TR, Gettins, PG and Lomas, DA (2000). Pathogenic alpha 1-antitrypsin polymers are formed by reactive loop-beta-sheet A linkage. J Biol Chem 275: 33663-33668.
    • (2000) J Biol Chem , vol.275 , pp. 33663-33668
    • Sivasothy, P.1    Dafforn, T.R.2    Gettins, P.G.3    Lomas, D.A.4
  • 3
    • 0026755363 scopus 로고
    • The mechanism of Z alpha 1-antitrypsin accumulation in the liver
    • Lomas, DA, Evans, DL, Finch, JT and Carrell, RW (1992). The mechanism of Z alpha 1-antitrypsin accumulation in the liver. Nature 357: 605-607.
    • (1992) Nature , vol.357 , pp. 605-607
    • Lomas, D.A.1    Evans, D.L.2    Finch, J.T.3    Carrell, R.W.4
  • 4
    • 79959703757 scopus 로고    scopus 로고
    • Z a-1 antitrypsin deficiency and the endoplasmic reticulum stress response
    • Greene, CM and McElvaney, NG (2010). Z a-1 antitrypsin deficiency and the endoplasmic reticulum stress response. World J Gastrointest Pharmacol Ther 1: 94-101.
    • (2010) World J Gastrointest Pharmacol Ther , vol.1 , pp. 94-101
    • Greene, C.M.1    McElvaney, N.G.2
  • 5
    • 36348957434 scopus 로고    scopus 로고
    • Alpha-1-antitrypsin mutant Z protein content in individual hepatocytes correlates with cell death in a mouse model
    • DOI 10.1002/hep.21822
    • Lindblad, D, Blomenkamp, K and Teckman, J (2007). Alpha-1-antitrypsin mutant Z protein content in individual hepatocytes correlates with cell death in a mouse model. Hepatology 46: 1228-1235. (Pubitemid 350144787)
    • (2007) Hepatology , vol.46 , Issue.4 , pp. 1228-1235
    • Lindblad, D.1    Blomenkamp, K.2    Teckman, J.3
  • 6
    • 79551604651 scopus 로고    scopus 로고
    • Alpha-1-antitrypsin deficiency: Importance of proteasomal and autophagic degradative pathways in disposal of liver disease-associated protein aggregates
    • Perlmutter, DH (2011). Alpha-1-antitrypsin deficiency: importance of proteasomal and autophagic degradative pathways in disposal of liver disease-associated protein aggregates. Annu Rev Med 62: 333-345.
    • (2011) Annu Rev Med , vol.62 , pp. 333-345
    • Perlmutter, D.H.1
  • 8
    • 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
    • DOI 10.1089/10430340460732490
    • Flotte, TR, Brantly, ML, Spencer, LT, Byrne, BJ, Spencer, CT, Baker, DJ et al. (2004). 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 15: 93-128. (Pubitemid 38147192)
    • (2004) Human Gene Therapy , vol.15 , Issue.1 , pp. 93-128
    • Flotte, T.R.1    Brantly, M.L.2    Spencer, L.T.3    Byrne, B.J.4    Spencer, C.T.5    Baker, D.J.6    Humphries, M.7
  • 9
    • 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
    • DOI 10.1038/sj.gt.3300793
    • Zern, MA, Ozaki, I, Duan, L, Pomerantz, R, Liu, SL and Strayer, DS (1999). A novel SV40-based vector successfully transduces and expresses an alpha 1-antitrypsin ribozyme in a human hepatoma-derived cell line. Gene Ther 6: 114-120. (Pubitemid 29043701)
    • (1999) Gene Therapy , vol.6 , Issue.1 , pp. 114-120
    • Zern, M.A.1    Ozaki, I.2    Duan, L.3    Pomerantz, R.4    Liu, S.-L.5    Strayer, D.S.6
  • 10
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in caenorhabditis elegans
    • DOI 10.1038/35888
    • Fire, A, Xu, S, Montgomery, MK, Kostas, SA, Driver, SE and Mello, CC (1998). Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391: 806-811. (Pubitemid 28099681)
    • (1998) Nature , vol.391 , Issue.6669 , pp. 806-811
    • Fire, A.1    Xu, S.2    Montgomery, M.K.3    Kostas, S.A.4    Driver, S.E.5    Mello, C.C.6
  • 14
    • 58149232358 scopus 로고    scopus 로고
    • Artificial microRNAs as siRNA shuttles: Improved safety as compared to shRNAs in vitro and in vivo
    • Boudreau, RL, Martins, I and Davidson, BL (2009). Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol Ther 17: 169-175.
    • (2009) Mol Ther , vol.17 , pp. 169-175
    • Boudreau, R.L.1    Martins, I.2    Davidson, B.L.3
  • 15
    • 80052137678 scopus 로고    scopus 로고
    • Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins
    • Li, C, Xiao, P, Gray, SJ, Weinberg, MS and Samulski, RJ (2011). Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins. Proc Natl Acad Sci USA 108: 14258-14263.
    • (2011) Proc Natl Acad Sci USA , vol.108 , pp. 14258-14263
    • Li, C.1    Xiao, P.2    Gray, S.J.3    Weinberg, M.S.4    Samulski, R.J.5
  • 16
    • 9144224451 scopus 로고    scopus 로고
    • Processing of primary microRNAs by the Microprocessor complex
    • DOI 10.1038/nature03049
    • Denli, AM, Tops, BB, Plasterk, RH, Ketting, RF and Hannon, GJ (2004). Processing of primary microRNAs by the Microprocessor complex. Nature 432: 231-235. (Pubitemid 39545854)
    • (2004) Nature , vol.432 , Issue.7014 , pp. 231-235
    • Denli, A.M.1    Tops, B.B.J.2    Plasterk, R.H.A.3    Ketting, R.F.4    Hannon, G.J.5
  • 17
    • 2442650370 scopus 로고    scopus 로고
    • The action of ARGONAUTE1 in the miRNA pathway and its regulation by the miRNA pathway are crucial for plant development
    • DOI 10.1101/gad.1201404
    • Vaucheret, H, Vazquez, F, Crété, P and Bartel, DP (2004). The action of ARGONAUTE1 in the miRNA pathway and its regulation by the miRNA pathway are crucial for plant development. Genes Dev 18: 1187-1197. (Pubitemid 38669039)
    • (2004) Genes and Development , vol.18 , Issue.10 , pp. 1187-1197
    • Vaucheret, H.1    Vazquez, F.2    Crete, P.3    Bartel, D.P.4
  • 18
    • 0023091532 scopus 로고
    • Tissue specific expression of the human alpha-1-antitrypsin gene in transgenic mice
    • Sifers, RN, Carlson, JA, Clift, SM, DeMayo, FJ, Bullock, DW and Woo, SL (1987). Tissue specific expression of the human alpha-1-antitrypsin gene in transgenic mice. Nucleic Acids Res 15: 1459-1475. (Pubitemid 17010428)
    • (1987) Nucleic Acids Research , vol.15 , Issue.4 , pp. 1459-1475
    • Sifers, R.N.1    Carlson, J.A.2    Clift, S.M.3
  • 19
    • 25144464388 scopus 로고    scopus 로고
    • Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice
    • DOI 10.1016/j.ymthe.2005.05.006, PII S1525001605002005
    • Rodriguez-Lebron, E, Denovan-Wright, EM, Nash, K, Lewin, AS and Mandel, RJ (2005). Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther 12: 618-633. (Pubitemid 41350143)
    • (2005) Molecular Therapy , vol.12 , Issue.4 , pp. 618-633
    • Rodriguez-Lebron, E.1    Denovan-Wright, E.M.2    Nash, K.3    Lewin, A.S.4    Mandel, R.J.5
  • 20
    • 79960148982 scopus 로고    scopus 로고
    • Lethal toxicity caused by expression of shRNA in the mouse striatum: Implications for therapeutic design
    • Martin, JN, Wolken, N, Brown, T, Dauer, WT, Ehrlich, ME and Gonzalez-Alegre, P (2011). Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design. Gene Ther 18: 666-673.
    • (2011) Gene Ther , vol.18 , pp. 666-673
    • Martin, J.N.1    Wolken, N.2    Brown, T.3    Dauer, W.T.4    Ehrlich, M.E.5    Gonzalez-Alegre, P.6
  • 22
    • 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, JS, Schürmann, N, Gu, S, Börner, K et al. (2010). Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest 120: 3106-3119.
    • (2010) J Clin Invest , vol.120 , pp. 3106-3119
    • Grimm, D.1    Wang, L.2    Lee, J.S.3    Schürmann, N.4    Gu, S.5    Börner, K.6
  • 24
    • 65549118489 scopus 로고    scopus 로고
    • Collapse of germline piRNAs in the absence of Argonaute3 reveals somatic piRNAs in flies
    • Li, C, Vagin, VV, Lee, S, Xu, J, Ma, S, Xi, H et al. (2009). Collapse of germline piRNAs in the absence of Argonaute3 reveals somatic piRNAs in flies. Cell 137: 509-521.
    • (2009) Cell , vol.137 , pp. 509-521
    • Li, C.1    Vagin, V.V.2    Lee, S.3    Xu, J.4    Ma, S.5    Xi, H.6
  • 25
    • 77953183812 scopus 로고    scopus 로고
    • A dicer-independent miRNA biogenesis pathway that requires Ago catalysis
    • Cheloufi, S, Dos Santos, CO, Chong, MM and Hannon, GJ (2010). A dicer-independent miRNA biogenesis pathway that requires Ago catalysis. Nature 465: 584-589.
    • (2010) Nature , vol.465 , pp. 584-589
    • Cheloufi, S.1    Santos, D.2    Chong, M.M.C.O.3    Hannon, G.J.4
  • 27
    • 36849048778 scopus 로고    scopus 로고
    • Dual Role for Argonautes in MicroRNA Processing and Posttranscriptional Regulation of MicroRNA Expression
    • DOI 10.1016/j.cell.2007.10.032, PII S0092867407013517
    • Diederichs, S and Haber, DA (2007). Dual role for argonautes in microRNA processing and posttranscriptional regulation of microRNA expression. Cell 131: 1097-1108. (Pubitemid 350235019)
    • (2007) Cell , vol.131 , Issue.6 , pp. 1097-1108
    • Diederichs, S.1    Haber, D.A.2
  • 28
    • 70449574252 scopus 로고    scopus 로고
    • Argonaute2 suppresses Drosophila fragile X expression preventing neurogenesis and oogenesis defects
    • Pepper, AS, Beerman, RW, Bhogal, B and Jongens, TA (2009). Argonaute2 suppresses Drosophila fragile X expression preventing neurogenesis and oogenesis defects. PLoS ONE 4: e7618.
    • (2009) PLoS ONE , vol.4
    • Pepper, A.S.1    Beerman, R.W.2    Bhogal, B.3    Jongens, T.A.4
  • 30
    • 67149115119 scopus 로고    scopus 로고
    • Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs
    • Khan, AA, Betel, D, Miller, ML, Sander, C, Leslie, CS and Marks, DS (2009). Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs. Nat Biotechnol 27: 549-555.
    • (2009) Nat Biotechnol , vol.27 , pp. 549-555
    • Khan, A.A.1    Betel, D.2    Miller, M.L.3    Sander, C.4    Leslie, C.S.5    Marks, D.S.6
  • 32
    • 1842683164 scopus 로고    scopus 로고
    • In Situ Synthesis of Oligonucleotide Microarrays
    • DOI 10.1002/bip.20005
    • Gao, X, Gulari, E and Zhou, X (2004). In situ synthesis of oligonucleotide microarrays. Biopolymers 73: 579-596. (Pubitemid 38480705)
    • (2004) Biopolymers , vol.73 , Issue.5 , pp. 579-596
    • Gao, X.1    Gulari, E.2    Zhou, X.3
  • 33
    • 0037316303 scopus 로고    scopus 로고
    • A comparison of normalization methods for high density oligonucleotide array data based on variance and bias
    • DOI 10.1093/bioinformatics/19.2.185
    • Bolstad, BM, Irizarry, RA, Astrand, M and Speed, TP (2003). A comparison of normalization methods for high density oligonucleotide array data based on variance and bias. Bioinformatics 19: 185-193. (Pubitemid 36181903)
    • (2003) Bioinformatics , vol.19 , Issue.2 , pp. 185-193
    • Bolstad, B.M.1    Irizarry, R.A.2    Astrand, M.3    Speed, T.P.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.