References
- Roche-Molina M Sanz-Rosa D Cruz FM Induction of sustained hyper-cholesterolemia by single adeno-associated virus-mediated gene transfer of mutant hPCSK9 Arterioscler Thromb Vasc Biol 2015 Jan 35 1 50 9
- Lagor WR Johnston JC Lock M Vandenberghe LH Rader DJ Adeno-associated viruses as liver-directed gene delivery vehicles: focus on lipoprotein metabolism Methods Mol Biol 2013 1027 273 307
- Asokan A Schaffer DV Samulski RJ The AAV vector toolkit: poised at the clinical crossroads Mol Ther 2012 Apr 20 4 699 708
- Kitajima K Marchadier DH Miller GC Gao GP Wilson JM Rader DJ Complete prevention of atherosclerosis in apoE-deficient mice by hepatic human apoE gene transfer with adeno-associated virus serotypes 7 and 8 Arterioscler Thromb Vasc Biol 2006 Aug 26 8 1852 7
- Gaudet D Méthot J Déry S Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial Gene Ther 2013 Apr 20 4 361 9
- Dias MF Joo K Kemp JA Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives Prog Retin Eye Res 2018 Mar 63 107 131
- Bauer RC Sasaki M Cohen DM Tribbles-1 regulates hepatic lipogenesis through posttranscriptional regulation of C/EBPα J Clin Invest 2015 Oct 1 125 10 3809 18
- Seidah NG Awan Z Chretien M Mbikay M PCSK9: a key modulator of cardiovascular health Circ Res 2014 Mar 14 114 6 1022 36
- Bjørklund MM Hollensen AK Hagensen MK Induction of atherosclerosis in mice and hamsters without germline genetic engineering Circ Res 2014 May 23 114 11 1684 9
- Gaudet D Brisson D Gene-based therapies in lipidology: current status and future challenges Curr Opin Lipidol 2015 Dec 26 6 553 65
- Gaudet D Stroes ES Méthot J Long-Term Retrospective Analysis of Gene Therapy with Alipogene Tiparvovec and Its Effect on Lipoprotein Lipase Deficiency-Induced Pancreatitis Hum Gene Ther 2016 Nov 27 11 916 25
- Defesche JC Gidding SS Harada-Shiba M Hegele RA Santos RD Wierzbicki AS Familial hypercholesterolaemia Nat Rev Dis Primers 2017 Dec 7 3 17093
- Ajufo E Cuchel M Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia Curr Atheroscler Rep 2016 May 18 5 22
- Grossman M Rader DJ Muller DW A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia Nat Med 1995 Nov 1 11 1148 54
- Lebherz C Gao G Louboutin JP Millar J Rader D Wilson JM Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia J Gene Med 2004 Jun 6 6 663 72
- Kassim SH Li H Bell P 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 Hum Gene Ther 2013 Jan 24 1 19 26
- Chen SJ Sanmiguel J Lock M Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia Hum Gene Ther Clin Dev 2013 Dec 24 4 154 60
- Watts JK Corey DR Silencing disease genes in the laboratory and the clinic J Pathol 2012 Jan 226 2 365 79
- Crooke ST Witztum JL Bennett CF Baker BF RNA-Targeted Therapeutics Cell Metab 2018 Apr 3 27 4 714 39
- Prakash TP Graham MJ Yu J Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice Nucleic Acids Res 2014 Jul 42 13 8796 807
- Wittrup A Lieberman J Knocking down disease: a progress report on siRNA therapeutics Nat Rev Genet 2015 Sep 16 9 543 52
- McGowan MP Tardif JC Ceska R Randomized, placebo-controlled trial of mipomersen in patients with severe hypercholesterolemia receiving maximally tolerated lipid-lowering therapy PLoS One 2012 7 11 e49006
- Raabe M Kim E Véniant M Nielsen LB Young SG Using genetically engineered mice to understand apolipoprotein-B deficiency syndromes in humans Proc Assoc Am Physicians 1998 Nov–Dec 110 6 521 30
- Raal FJ Santos RD Blom DJ Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial Lancet 2010 Mar 20 375 9719 998 1006
- Duell PB Santos RD Kirwan BA Witztum JL Tsimikas S Kastelein JJP Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia J Clin Lipidol 2016 Jul–Aug 10 4 1011 21
- Ray KK Landmesser U Leiter LA Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol N Engl J Med 2017 Apr 13 376 15 1430 40
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) (ORION-2); 2018 Apr 25 [cited 2018 Oct 5]. Available from: https://clinicaltrials.gov/ct2/show/NCT02963311
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 An Extension Trial of Inclisiran Compared to Evolocumab in Participants With Cardiovascular Disease and High Cholesterol; 2017 Dec 22 [cited 2018 Oct 5]. Available from: https://clinicaltrials.gov/ct2/show/NCT03060577
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Inclisiran for Participants With Atherosclerotic Cardiovascular Disease and Elevated Low-density Lipoprotein Cholesterol (ORION-10); 2018 Apr 23 [cited 2018 Oct 5]. Available from: https://clinicaltrials.gov/ct2/show/NCT03399370
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Inclisiran for Subjects With ACSVD or ACSVD-Risk Equivalents and Elevated Low-density Lipoprotein Cholesterol (ORION-11); 2018 Apr 26 [cited 2018 Oct 5]. Available from: https://clinicaltrials.gov/ct2/show/NCT03400800
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Trial to Evaluate the Effect of Inclisiran Treatment on Low Density Lipoprotein Cholesterol (LDL-C) in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH) (ORION-9); 2018 Apr 9 [cited 2018 Oct 5]. Available from: https://clinicaltrials.gov/ct2/show/NCT03397121
- Wang CS McConathy WJ Kloer HU Alaupovic P Modulation of lipoprotein lipase activity by apolipoproteins. Effect of apolipoprotein C-III J Clin Invest 1985 Feb 75 2 384 90
- Gordts PL Nock R Son NH ApoC-III inhibits clearance of triglyceride-rich lipoproteins through LDL family receptors J Clin Invest 2016 Aug 1 126 8 2855 66
- Norata GD Tsimikas S Pirillo A Catapano AL Apolipoprotein C-III: From Pathophysiology to Pharmacology Trends Pharmacol Sci 2015 Oct 36 10 675 87
-
Graham
MJ
Lee
RG
Bell
TA
3rd Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans Circ Res 2013 May 24 112 11 1479 90 - Gaudet D Digenio A Alexander V The approach study: a randomized, double-blind, placebo-controlled, phase 3 study of volanesorsen administered subcutaneously to patients with familial chylomicronemia syndrome (FCS) Atherosclerosis 2017 Aug 263 E10
- Lee EC Desai U Gololobov G Identification of a new functional domain in angiopoietin-like 3 (ANGPTL3) and angiopoietin-like 4 (ANGPTL4) involved in binding and inhibition of lipoprotein lipase (LPL) J Biol Chem 2009 May 15 284 20 13735 45
- Xu YX Redon V Yu H Role of angiopoietin-like 3 (ANGPTL3) in regulating plasma level of low-density lipoprotein cholesterol Atherosclerosis 2018 Jan 268 196 206
- Dewey FE Gusarova V Dunbar RL Genetic and Pharmacologic Inactivation of ANGPTL3 and Cardiovascular Disease N Engl J Med 2017 Jul 20 377 3 211 21
- Graham MJ Lee RG Brandt TA Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides N Engl J Med 2017 Jul 20 377 3 222 32
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL); 2018 Jul 2 {cited 2018 Oct 5]. Available from: https://clinicaltrials.gov/ct2/show/NCT03514420
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH); 2018 Apr 23 [cited 2018 Oct 5]. Available from: https://clinicaltrials.gov/ct2/show/NCT03455777
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Phase 2 Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Familial Chylomicronemia Syndrome (FCS); 2018 Aug 1 [cited 2018 Oct 5]. https://clinicaltrials.gov/ct2/show/NCT03360747
- Clinical Trials [Internet] Bethesda, MD U.S. National Library of Medicine c2018 Study of ISIS 703802 in Subjects With Hypertriglyceridemia, Type 2 Diabetes Mellitus, and Nonalcoholic Fatty Liver Disease; 2018 Sep 26 [cited 2018 Oct 5]. https://clinicaltrials.gov/ct2/show/NCT03371355
- Jinek M Chylinski K Fonfara I Hauer M Doudna JA Charpentier E A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity Science 2012 Aug 17 337 6096 816 21
- Ding Q Strong A Patel KM Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing Circ Res 2014 Aug 15 115 5 488 92
- Ran FA Cong L Yan WX In vivo genome editing using Staphylococcus aureus Cas9 Nature 2015 Apr 9 520 7546 186 91
- Jarrett KE Lee CM Yeh YH Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease Sci Rep 2017 Mar 16 7 44624
- Jarrett KE Lee C De Giorgi M Somatic Editing of Ldlr With Adeno-Associated Viral-CRISPR Is an Efficient Tool for Atherosclerosis Research Arterioscler Thromb Vasc Biol 2018 Sep 38 9 1997 2006
- Wagner DL Amini L Wendering DJ High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population Nat Med. 2018 Oct 29 [Epub ahead of print]
- Komor AC Kim YB Packer MS Zuris JA Liu DR Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage Nature 2016 May 19 533 7603 420 4
- Chadwick AC Evitt NH Lv W Musunuru K Reduced Blood Lipid Levels With In Vivo CRISPR-Cas9 Base Editing of ANGPTL3 Circulation 2018 Feb 27 137 9 975 7
- Rossidis AC Stratigis JD Chadwick AC In utero CRISPR-mediated therapeutic editing of metabolic genes Nat Med 2018 Oct 24 10 1513 8
DOI: https://doi.org/10.14797/mdcj-15-1-62 | Journal eISSN: 1947-6108
Language: English
Page range: 62 - 69
Published on: Jan 1, 2019
Published by: Houston Methodist DeBakey Heart & Vascular Center
In partnership with: Paradigm Publishing Services
Keywords:
© 2019 Marco De Giorgi, William R. Lagor, published by Houston Methodist DeBakey Heart & Vascular Center
This work is licensed under the Creative Commons Attribution-NonCommercial 4.0 License.