-
1
-
-
0028610531
-
Prevalence of hepatocellular carcinoma in alpha-1-antitrypsin deficiency
-
Propst, T, Propst, A, Dietze, O, Judmaier, G, Braunsteiner, H and Vogel, W (1994). Prevalence of hepatocellular carcinoma in alpha-1-antitrypsin deficiency. J Hepatol 21: 1006-1011.
-
(1994)
J Hepatol
, vol.21
, pp. 1006-1011
-
-
Propst, T.1
Propst, A.2
Dietze, O.3
Judmaier, G.4
Braunsteiner, H.5
Vogel, W.6
-
2
-
-
0034721790
-
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
-
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
-
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
-
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
-
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
-
7
-
-
33845807816
-
1-antitrypsin (AAT) vector in AAT-deficient adults
-
DOI 10.1089/hum.2006.17.1177
-
Brantly, ML, Spencer, LT, Humphries, M, Conlon, TJ, Spencer, CT, Poirier, A et al. (2006). Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Hum Gene Ther 17: 1177-1186. (Pubitemid 44975135)
-
(2006)
Human Gene Therapy
, vol.17
, Issue.12
, pp. 1177-1186
-
-
Brantly, M.L.1
Spencer, L.T.2
Humphries, M.3
Conlon, T.J.4
Spencer, C.T.5
Poirier, A.6
Garlington, W.7
Baker, D.8
Song, S.9
Berns, K.I.10
Muzyczka, N.11
Snyder, R.O.12
Byrne, B.J.13
Flotte, T.R.14
-
8
-
-
0742307402
-
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
-
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
-
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
-
11
-
-
34547943486
-
In vivo post-transcriptional gene silencing of α-1 antitrypsin by adeno-associated virus vectors expressing siRNA
-
DOI 10.1038/labinvest.3700629, PII 3700629
-
Cruz, PE, Mueller, C, Cossette, TL, Golant, A, Tang, Q, Beattie, SG et al. (2007). In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA. Lab Invest 87: 893-902. (Pubitemid 47267814)
-
(2007)
Laboratory Investigation
, vol.87
, Issue.9
, pp. 893-902
-
-
Cruz, P.E.1
Mueller, C.2
Cossette, T.L.3
Golant, A.4
Tang, Q.5
Beattie, S.G.6
Brantly, M.7
Campbell-Thompson, M.8
Blomenkamp, K.S.9
Teckman, J.H.10
Flotte, T.R.11
-
12
-
-
33745299723
-
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
-
DOI 10.1038/nature04791, PII NATURE04791
-
Grimm, D, Streetz, KL, Jopling, CL, Storm, TA, Pandey, K, Davis, CR et al. (2006). Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 441: 537-541. (Pubitemid 44050158)
-
(2006)
Nature
, vol.441
, Issue.7092
, pp. 537-541
-
-
Grimm, D.1
Streetz, K.L.2
Jopling, C.L.3
Storm, T.A.4
Pandey, K.5
Davis, C.R.6
Marion, P.7
Salazar, F.8
Kay, M.A.9
-
13
-
-
44449121785
-
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
-
DOI 10.1073/pnas.0801775105
-
McBride, JL, Boudreau, RL, Harper, SQ, Staber, PD, Monteys, AM, Martins, I et al. (2008). Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci USA 105: 5868-5873. (Pubitemid 351758464)
-
(2008)
Proceedings of the National Academy of Sciences of the United States of America
, vol.105
, Issue.15
, pp. 5868-5873
-
-
McBride, J.L.1
Boudreau, R.L.2
Harper, S.Q.3
Staber, P.D.4
Monteys, A.M.5
Martins, I.6
Gilmore, B.L.7
Burstein, H.8
Peluso, R.W.9
Polisky, B.10
Carter, B.J.11
Davidson, B.L.12
-
14
-
-
58149232358
-
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
-
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
-
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
-
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
-
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
-
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
-
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
-
21
-
-
34548548614
-
Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC
-
DOI 10.1093/nar/gkm543
-
Castanotto, D, Sakurai, K, Lingeman, R, Li, H, Shively, L, Aagaard, L et al. (2007). Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC. Nucleic Acids Res 35: 5154-5164. (Pubitemid 47394199)
-
(2007)
Nucleic Acids Research
, vol.35
, Issue.15
, pp. 5154-5164
-
-
Castanotto, D.1
Sakurai, K.2
Lingeman, R.3
Li, H.4
Shively, L.5
Aagaard, L.6
Soifer, H.7
Gatignol, A.8
Riggs, A.9
Rossi, J.J.10
-
22
-
-
77956355233
-
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
-
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
-
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
-
26
-
-
34547955952
-
A Slicer-independent role for Argonaute 2 in hematopoiesis and the microRNA pathway
-
DOI 10.1101/gad.1565607
-
O'Carroll, D, Mecklenbrauker, I, Das, PP, Santana, A, Koenig, U, Enright, AJ et al. (2007). A Slicer-independent role for Argonaute 2 in hematopoiesis and the microRNA pathway. Genes Dev 21: 1999-2004. (Pubitemid 47267286)
-
(2007)
Genes and Development
, vol.21
, Issue.16
, pp. 1999-2004
-
-
O'Carroll, D.1
Mecklenbrauker, I.2
Das, P.P.3
Santana, A.4
Koenig, U.5
Enright, A.J.6
Miska, E.A.7
Tarakhovsky, A.8
-
27
-
-
36849048778
-
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
-
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
-
29
-
-
75749147413
-
Essential role for Argonaute2 protein in mouse oogenesis
-
Kaneda, M, Tang, F, O'Carroll, D, Lao, K and Surani, MA (2009). Essential role for Argonaute2 protein in mouse oogenesis. Epigenetics Chromatin 2: 9.
-
(2009)
Epigenetics Chromatin
, vol.2
, pp. 9
-
-
Kaneda, M.1
Tang, F.2
O'Carroll, D.3
Lao, K.4
Surani, M.A.5
-
30
-
-
67149115119
-
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
-
31
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
DOI 10.1073/pnas.182412299
-
Gao, GP, Alvira, MR, Wang, L, Calcedo, R, Johnston, J and Wilson, JM (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99: 11854-11859. (Pubitemid 34994488)
-
(2002)
Proceedings of the National Academy of Sciences of the United States of America
, 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
-
32
-
-
1842683164
-
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
-
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
|