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Apolipoprotein B knockdown by AAV-delivered shRNA lowers plasma cholesterol in mice.

Koornneef A, Maczuga P, van Logtenstein R, Borel F, Blits B, Ritsema T, van Deventer S, Petry H, Konstantinova P.

Mol Ther. 2011 Apr;19(4):731-40. doi: 10.1038/mt.2011.6. Epub 2011 Feb 8.


Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver.

Maczuga P, Verheij J, van der Loos C, van Logtenstein R, Hooijer G, Martier R, Borel F, Lubelski J, Koornneef A, Blits B, van Deventer S, Petry H, Konstantinova P.

Gene Ther. 2014 Jan;21(1):60-70. doi: 10.1038/gt.2013.58. Epub 2013 Oct 24.


Embedding siRNA sequences targeting apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy.

Maczuga P, Lubelski J, van Logtenstein R, Borel F, Blits B, Fakkert E, Costessi A, Butler D, van Deventer S, Petry H, Koornneef A, Konstantinova P.

Mol Ther. 2013 Jan;21(1):217-27. doi: 10.1038/mt.2012.160. Epub 2012 Oct 23.


siRNA-induced liver ApoB knockdown lowers serum LDL-cholesterol in a mouse model with human-like serum lipids.

Tadin-Strapps M, Peterson LB, Cumiskey AM, Rosa RL, Mendoza VH, Castro-Perez J, Puig O, Zhang L, Strapps WR, Yendluri S, Andrews L, Pickering V, Rice J, Luo L, Chen Z, Tep S, Ason B, Somers EP, Sachs AB, Bartz SR, Tian J, Chin J, Hubbard BK, Wong KK, Mitnaul LJ.

J Lipid Res. 2011 Jun;52(6):1084-97. doi: 10.1194/jlr.M012872. Epub 2011 Mar 11.


An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis.

Crooke RM, Graham MJ, Lemonidis KM, Whipple CP, Koo S, Perera RJ.

J Lipid Res. 2005 May;46(5):872-84. Epub 2005 Feb 16.


Effective lowering of plasma, LDL, and esterified cholesterol in LDL receptor-knockout mice by adenovirus-mediated gene delivery of ApoB mRNA editing enzyme (Apobec1).

Teng B, Ishida B, Forte TM, Blumenthal S, Song LZ, Gotto AM Jr, Chan L.

Arterioscler Thromb Vasc Biol. 1997 May;17(5):889-97.


Reduction of choroidal neovascularization in mice by adeno-associated virus-delivered anti-vascular endothelial growth factor short hairpin RNA.

Askou AL, Pournaras JA, Pihlmann M, Svalgaard JD, Arsenijevic Y, Kostic C, Bek T, Dagnaes-Hansen F, Mikkelsen JG, Jensen TG, Corydon TJ.

J Gene Med. 2012 Nov;14(11):632-41. doi: 10.1002/jgm.2678.


Effects of dietary fats and cholesterol on liver lipid content and hepatic apolipoprotein A-I, B, and E and LDL receptor mRNA levels in cebus monkeys.

Hennessy LK, Osada J, Ordovas JM, Nicolosi RJ, Stucchi AF, Brousseau ME, Schaefer EJ.

J Lipid Res. 1992 Mar;33(3):351-60.


Safe and sustained overexpression of functional apolipoprotein A-I/high-density lipoprotein in apolipoprotein A-I-null mice by muscular adeno-associated viral serotype 8 vector gene transfer.

Cimmino G, Chen W, Speidl WS, Giannarelli C, Ibanez B, Fuster V, Hajjar R, Walsh CE, Badimon JJ.

J Cardiovasc Pharmacol. 2009 Nov;54(5):405-11. doi: 10.1097/FJC.0b013e3181bad264.


In vivo regulation of low-density lipoprotein receptor and apolipoprotein B gene expressions by dietary fat and cholesterol in inbred strains of mice.

Srivastava RA, Jiao S, Tang JJ, Pfleger BA, Kitchens RT, Schonfeld G.

Biochim Biophys Acta. 1991 Oct 15;1086(1):29-43.


Liver LDL receptor mRNA expression is decreased in human ApoB/CETP double transgenic mice and is regulated by diet as well as the cytokine oncostatin M.

Liu J, Zhang YL, Spence MJ, Vestal RE, Wallace PM, Grass DS.

Arterioscler Thromb Vasc Biol. 1997 Nov;17(11):2948-54.


Development of gene therapy for treatment of age-related macular degeneration.

Askou AL.

Acta Ophthalmol. 2014 Jul;92 Thesis3:1-38. doi: 10.1111/aos.12452.


Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia.

Kassim SH, Li H, Bell P, Somanathan S, Lagor W, Jacobs F, Billheimer J, Wilson JM, Rader DJ.

Hum Gene Ther. 2013 Jan;24(1):19-26. doi: 10.1089/hum.2012.108. Epub 2012 Nov 14.


Phenotype interaction of apobec-1 and CETP, LDLR, and apoE gene expression in mice: role of apoB mRNA editing in lipoprotein phenotype expression.

Nakamuta M, Taniguchi S, Ishida BY, Kobayashi K, Chan L.

Arterioscler Thromb Vasc Biol. 1998 May;18(5):747-55.


ApoB siRNA-induced liver steatosis is resistant to clearance by the loss of fatty acid transport protein 5 (Fatp5).

Ason B, Castro-Perez J, Tep S, Stefanni A, Tadin-Strapps M, Roddy T, Hankemeier T, Hubbard B, Sachs AB, Michael Flanagan W, Kuklin NA, Mitnaul LJ.

Lipids. 2011 Nov;46(11):991-1003. doi: 10.1007/s11745-011-3596-3. Epub 2011 Aug 9.


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