Brett P Monia

Brett P Monia

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Steric Inhibition of 5' UTR Regulatory Elements Results in Upregulation of Human CFTR.

Mol Ther 2019 10 12;27(10):1749-1757. Epub 2019 Jul 12.

Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA. Electronic address:

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http://dx.doi.org/10.1016/j.ymthe.2019.06.016DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6822282PMC
October 2019

Antisense oligonucleotide targeting of mRNAs encoding ENaC subunits α, β, and γ improves cystic fibrosis-like disease in mice.

J Cyst Fibros 2019 05 10;18(3):334-341. Epub 2018 Aug 10.

Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

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http://dx.doi.org/10.1016/j.jcf.2018.07.006DOI Listing
May 2019

Inotersen (transthyretin-specific antisense oligonucleotide) for treatment of transthyretin amyloidosis.

Neurodegener Dis Manag 2019 02 18;9(1):25-30. Epub 2018 Dec 18.

Ionis Pharmaceuticals, Carlsbad, CA, USA.

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https://www.futuremedicine.com/doi/10.2217/nmt-2018-0037
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http://dx.doi.org/10.2217/nmt-2018-0037DOI Listing
February 2019

Reducing dynamin 2 (DNM2) rescues -related dominant centronuclear myopathy.

Proc Natl Acad Sci U S A 2018 10 5;115(43):11066-11071. Epub 2018 Oct 5.

Institut de Génétique et de Biologie Moléculaire et Cellulaire, 67404 Illkirch, France;

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http://dx.doi.org/10.1073/pnas.1808170115DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6205463PMC
October 2018

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.

N Engl J Med 2018 Jul;379(1):22-31

From the Indiana University School of Medicine, Indianapolis (M.D.B.); Centro de Estudos em Paramiloidose Antônio Rodrigues de Mello, National Amyloidosis Referral Center, University Hospital, Federal University of Rio de Janeiro, Rio de Janeiro (M.W.-C.); Amyloidosis Center, Boston University School of Medicine (J.L.B.) and Brigham and Women's Hospital, Harvard Medical School (A.M.S., S.D.S.), Boston; Johns Hopkins University, Baltimore (M.P.); Mayo Clinic, Rochester, MN (P.J.D., W.J.L., M.A.G.); University of California, Irvine, Irvine (A.K.W.); Amyloid Network-Hospital Henri Mondor-Assistance Publique-Hôpitaux de Paris (AP-HP)-Université Paris Est, Créteil, France (V.P.-B.); Institute for Neurologic Research Raúl Carrea, FLENI, Buenos Aires (F.A.B.); Amyloidosis Research and Treatment Center, Fondazione IRCCS Policlinico San Matteo, University of Pavia, Pavia (G.M., L.O.), and Unit of Neurology, University Hospital, Messina (G.V.) - both in Italy; Hospital AACD (Associação de Assistência à Criança Deficiente), São Paulo (M.S.); Columbia University Medical Center (T.H.B.) and Mount Sinai Medical Center (P.D.G.), New York; University College London-National Amyloidosis Centre, London (C.W.); Penn Presbyterian Medical Center, University of Pennsylvania Health System, Philadelphia (B.M.D.); Centre Hospitaliere Universitaire Bicêtre, AP-HP, Unité 1195, INSERM, Université Paris-Sud, Paris (D.A.); Oregon Health and Science University, Portland (S.B.H.); Centro Hospitalar Lisboa Norte-Hospital de Santa Maria, Lisbon (I.C.), and Centro Hospitalar do Porto, Porto (T.C.) - both in Portugal; Universitätsklinikum Münster, Münster, Germany (H.H.S.); Hospital Clínic, Universitat de Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer (J.M.C.), and Hospital Universitari Vall d'Hebron (J.G.), Barcelona; Auckland City Hospital, Auckland, New Zealand (E.G.); and Ionis Pharmaceuticals, Carlsbad, CA (B.P.M., S.G.H., T.J.K., B.W.M., S.W.J., B.F.B., E.J.A.).

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http://dx.doi.org/10.1056/NEJMoa1716793DOI Listing
July 2018

Knockdown of Z Mutant Alpha-1 Antitrypsin In Vivo Using Modified DNA Antisense Oligonucleotides.

Methods Mol Biol 2017 ;1639:127-138

Department of Antisense Drug Discovery, IONIS Pharmaceuticals, 2855 Gazelle Court, Carlsbad, 92010, CA, USA.

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http://dx.doi.org/10.1007/978-1-4939-7163-3_12DOI Listing
April 2018

Inhaled ENaC antisense oligonucleotide ameliorates cystic fibrosis-like lung disease in mice.

J Cyst Fibros 2017 Nov 20;16(6):671-680. Epub 2017 May 20.

Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

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http://dx.doi.org/10.1016/j.jcf.2017.05.003DOI Listing
November 2017

Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice.

Nat Commun 2017 06 7;8:15661. Epub 2017 Jun 7.

Department of Translational Medicine and Neurogenetics, Institut de Génétique et de Biologie Moléculaire et Cellulaire (IGBMC), Illkirch 67404, France.

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http://dx.doi.org/10.1038/ncomms15661DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5467247PMC
June 2017

Treatment of transthyretin cardiomyopathy with a TTR-specific antisense oligonucleotide (IONIS-TTR).

Amyloid 2017 Mar;24(sup1):134-135

b Ionis Pharmaceuticals , Carlsbad , CA , USA.

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http://dx.doi.org/10.1080/13506129.2017.1280015DOI Listing
March 2017

Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines.

Hum Mol Genet 2016 12;25(23):5178-5187

Molecular Medicine Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.

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http://dx.doi.org/10.1093/hmg/ddw338DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC6078641PMC
December 2016

Reversing Antisense Oligonucleotide Activity with a Sense Oligonucleotide Antidote: Proof of Concept Targeting Prothrombin.

Nucleic Acid Ther 2015 Dec 21;25(6):297-305. Epub 2015 Sep 21.

1 Drug Discovery and Corporate Development, Isis Pharmaceuticals, Inc. , Carlsbad, California.

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http://dx.doi.org/10.1089/nat.2015.0560DOI Listing
December 2015

Genetic diminution of circulating prothrombin ameliorates multiorgan pathologies in sickle cell disease mice.

Blood 2015 Oct 18;126(15):1844-55. Epub 2015 Aug 18.

Division of Experimental Hematology and Cancer Biology, Division of Hematology, Cancer and Blood Diseases Institute, and.

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http://dx.doi.org/10.1182/blood-2015-01-625707DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4600020PMC
October 2015

Transthyretin Antisense Oligonucleotides Lower Circulating RBP4 Levels and Improve Insulin Sensitivity in Obese Mice.

Diabetes 2015 May 18;64(5):1603-14. Epub 2014 Dec 18.

Division of Endocrinology, Diabetes and Metabolism, Department of Medicine, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA

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http://dx.doi.org/10.2337/db14-0970DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4407860PMC
May 2015

Arterial thrombosis is accelerated in mice deficient in histidine-rich glycoprotein.

Blood 2015 Apr 17;125(17):2712-9. Epub 2015 Feb 17.

Thrombosis and Atherosclerosis Research Institute, Department of Medical Sciences, Department of Medicine, and Department of Biochemistry and Biomedical Sciences, McMaster University, Hamilton, Ontario, Canada;

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http://dx.doi.org/10.1182/blood-2014-11-611319DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4416941PMC
April 2015

Factor XI antisense oligonucleotide for prevention of venous thrombosis.

N Engl J Med 2015 Jan 7;372(3):232-40. Epub 2014 Dec 7.

From the Department of Vascular Medicine, Academic Medical Center, University of Amsterdam (H.R.B.), and International Trial Expertise Advisory and Services (ITREAS) (A.S.) - both in Amsterdam; Isis Pharmaceuticals, Carlsbad, CA (C.B., S.B., B.P.M.); Department of Pathology, Microbiology and Immunology, Vanderbilt University, Nashville (D.G.); University of Oklahoma Health Sciences Center, College of Public Health, Oklahoma City (G.E.R.); KU Leuven Department of Cardiovascular Sciences, Vascular Medicine and Hemostasis, Leuven, Belgium (P.V.); and the Thrombosis and Atherosclerosis Research Institute and McMaster University, Hamilton, ON, Canada (J.I.W.).

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http://www.nejm.org/doi/10.1056/NEJMoa1405760
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http://dx.doi.org/10.1056/NEJMoa1405760DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4367537PMC
January 2015

Factor XI regulates pathological thrombus formation on acutely ruptured atherosclerotic plaques.

Arterioscler Thromb Vasc Biol 2014 Aug 19;34(8):1668-73. Epub 2014 Jun 19.

From the Departments of Experimental Vascular Medicine (M.L.v.M., V.L.K., J.C.M.M.) and Pathology (J.J.T.H.R.), Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands; Department of Biochemistry, Cardiovascular Research Institute Maastricht (CARIM), Maastricht University, Maastricht, The Netherlands (M.J.E.K., J.W.M.H.); Department of Antisense Drug Discovery, Isis Pharmaceuticals Inc, Carlsbad, CA (S.B., B.P.M.); and Department of Plasma Proteins, Sanquin Research, Amsterdam, The Netherlands (J.C.M.M.).

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http://atvb.ahajournals.org/content/34/8/1668.full.pdf
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http://atvb.ahajournals.org/cgi/doi/10.1161/ATVBAHA.114.3032
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http://dx.doi.org/10.1161/ATVBAHA.114.303209DOI Listing
August 2014

Nicotinamide N-methyltransferase knockdown protects against diet-induced obesity.

Nature 2014 Apr;508(7495):258-62

Division of Endocrinology, Diabetes, and Metabolism, Department of Medicine, Beth Israel Deaconess Medical Center and Harvard Medical School, 330 Brookline Avenue, Boston, Massachusetts 02215, USA.

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http://dx.doi.org/10.1038/nature13198DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4107212PMC
April 2014

Identification of REST-regulated genes and pathways using a REST-targeted antisense approach.

Nucleic Acid Ther 2013 Dec;23(6):389-400

1 Department of Molecular Neurobiology, Evotec AG , Hamburg, Germany .

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http://dx.doi.org/10.1089/nat.2013.0445DOI Listing
December 2013

Inhibition of vascular permeability by antisense-mediated inhibition of plasma kallikrein and coagulation factor 12.

Nucleic Acid Ther 2013 Jun 12;23(3):175-87. Epub 2013 Apr 12.

Department of Antisense Drug Discovery, Isis Pharmaceuticals, Carlsbad, California 92010, USA.

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http://dx.doi.org/10.1089/nat.2013.0417DOI Listing
June 2013

Genomic analysis of wig-1 pathways.

PLoS One 2012 7;7(2):e29429. Epub 2012 Feb 7.

Department of Drug Discovery, Isis Pharmaceuticals Inc, Carlsbad, California, United States of America.

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http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0029429PLOS
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3274543PMC
July 2012

The use of stable isotope-labeled glycerol and oleic acid to differentiate the hepatic functions of DGAT1 and -2.

J Lipid Res 2012 Jun 3;53(6):1106-16. Epub 2012 Apr 3.

Cardiovascular and Metabolic Disease Research and Community of Research Excellence and Advanced Technology, Janssen Pharmaceutical Companies of Johnson and Johnson, Spring House, PA 19477, USA.

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http://dx.doi.org/10.1194/jlr.M020156DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3351817PMC
June 2012

Clinical development of an antisense therapy for the treatment of transthyretin-associated polyneuropathy.

Amyloid 2012 Jun 12;19 Suppl 1:43-4. Epub 2012 Apr 12.

Department of Antisense Drug Discovery and Clinical Development, Isis Pharmaceuticals, Carlsbad, CA, USA.

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http://dx.doi.org/10.3109/13506129.2012.673140DOI Listing
June 2012

Antisense oligonucleotide suppression of serum amyloid A reduces amyloid deposition in mice with AA amyloidosis.

Amyloid 2011 Sep 10;18(3):136-46. Epub 2011 Aug 10.

Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, IN, USA.

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http://www.tandfonline.com/doi/full/10.3109/13506129.2011.59
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http://dx.doi.org/10.3109/13506129.2011.597464DOI Listing
September 2011

Suppression of choroid plexus transthyretin levels by antisense oligonucleotide treatment.

Amyloid 2010 Jun;17(2):43-9

Department of Pathology and Laboratory Medicine, Indiana University School of Medicine, Indianapolis, IN 46202-5126, USA.

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http://www.tandfonline.com/doi/full/10.3109/13506129.2010.48
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http://dx.doi.org/10.3109/13506129.2010.483121DOI Listing
June 2010

Antisense oligonucleotide targeting of insulin-like growth factor-1 receptor (IGF-1R) in prostate cancer.

Prostate 2010 Feb;70(2):206-18

Vancouver Prostate Centre, University of British Columbia, Vancouver, BC, Canada.

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http://dx.doi.org/10.1002/pros.21054DOI Listing
February 2010

PKCepsilon plays a causal role in acute ethanol-induced steatosis.

Arch Biochem Biophys 2009 Feb 11;482(1-2):104-11. Epub 2008 Nov 11.

Department of Pharmacology and Toxicology, University of Louisville Health Sciences Center, Louisville, KY 40292, USA.

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http://dx.doi.org/10.1016/j.abb.2008.11.004DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2672568PMC
February 2009

Reduction of JNK1 expression with antisense oligonucleotide improves adiposity in obese mice.

Am J Physiol Endocrinol Metab 2008 Aug 3;295(2):E436-45. Epub 2008 Jun 3.

Department of Antisense Drug Discovery, Isis Pharmaceuticals, 1896 Rutherford Rd., Carlsbad, CA 92008, USA.

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http://dx.doi.org/10.1152/ajpendo.00629.2007DOI Listing
August 2008

Antisense transcripts are targets for activating small RNAs.

Nat Struct Mol Biol 2008 Aug 6;15(8):842-8. Epub 2008 Jul 6.

Department of Pharmacology, University of Texas Southwestern Medical Center, Dallas, Texas 75390-9041, USA.

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http://dx.doi.org/10.1038/nsmb.1444DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2574822PMC
August 2008

Translational regulation of lipoprotein lipase in adipocytes: depletion of cellular protein kinase Calpha activates binding of the C subunit of protein kinase A to the 3'-untranslated region of the lipoprotein lipase mRNA.

Biochem J 2008 Jul;413(2):315-22

The Central Arkansas Veterans HealthCare System, Department of Medicine, Division of Endocrinology, University of Arkansas for Medical Sciences, Little Rock, AR 72205, USA.

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http://dx.doi.org/10.1042/BJ20071559DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2755514PMC
July 2008

Comparing in vitro and in vivo activity of 2'-O-[2-(methylamino)-2-oxoethyl]- and 2'-O-methoxyethyl-modified antisense oligonucleotides.

J Med Chem 2008 May 10;51(9):2766-76. Epub 2008 Apr 10.

Department of Medicinal Chemistry and Antisense Core Research, Isis Pharmaceuticals Inc., 1896 Rutherford Road, Carlsbad, California 92008, USA.

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http://dx.doi.org/10.1021/jm701537zDOI Listing
May 2008

Reduced adiposity and improved insulin sensitivity in obese mice with antisense suppression of 4E-BP2 expression.

Am J Physiol Endocrinol Metab 2008 Mar 15;294(3):E530-9. Epub 2008 Jan 15.

Department of Antisense Drug Discovery, Isis Pharmaceuticals Inc., Carlsbad, California 92008, USA.

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http://www.physiology.org/doi/10.1152/ajpendo.00350.2007
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http://dx.doi.org/10.1152/ajpendo.00350.2007DOI Listing
March 2008

Inhaled CD86 antisense oligonucleotide suppresses pulmonary inflammation and airway hyper-responsiveness in allergic mice.

J Pharmacol Exp Ther 2007 Jun 26;321(3):938-46. Epub 2007 Mar 26.

Antisense Drug Discovery, ISIS Pharmaceuticals, 1896 Rutherford Rd., Carlsbad, CA 92008, USA.

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http://dx.doi.org/10.1124/jpet.106.119214DOI Listing
June 2007

Inhibiting gene expression with locked nucleic acids (LNAs) that target chromosomal DNA.

Biochemistry 2007 Jun 31;46(25):7572-80. Epub 2007 May 31.

Department of Pharmacology, University of Texas Southwestern Medical Center at Dallas, 6001 Forest Park Road, Dallas, Texas 75390-9041, USA.

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http://dx.doi.org/10.1021/bi700227gDOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2527755PMC
June 2007

Antisense inhibition of proprotein convertase subtilisin/kexin type 9 reduces serum LDL in hyperlipidemic mice.

J Lipid Res 2007 Apr 22;48(4):763-7. Epub 2007 Jan 22.

Cardiovascular Group, Department of Antisense Drug Discovery, Isis Pharmaceuticals, Inc., Carlsbad, CA 92008, USA.

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http://www.jlr.org/lookup/doi/10.1194/jlr.C600025-JLR200
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http://dx.doi.org/10.1194/jlr.C600025-JLR200DOI Listing
April 2007

Selective down-regulation of glioma-associated oncogene 2 inhibits the proliferation of hepatocellular carcinoma cells.

Cancer Res 2007 Apr;67(8):3583-93

Isis Pharmaceuticals Inc., 1896 Rutherford Road, Carlsbad, CA 92008, USA.

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http://dx.doi.org/10.1158/0008-5472.CAN-06-3040DOI Listing
April 2007

Anti-inflammatory activity of inhaled IL-4 receptor-alpha antisense oligonucleotide in mice.

Am J Respir Cell Mol Biol 2007 Mar 21;36(3):276-85. Epub 2006 Sep 21.

Department of Clinical Development, Pharmaceuticals, 1896 Rutherford Road, Carlsbad, CA 92008, USA.

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http://dx.doi.org/10.1165/rcmb.2005-0456OCDOI Listing
March 2007

Inhibition of protein kinase Cepsilon prevents hepatic insulin resistance in nonalcoholic fatty liver disease.

J Clin Invest 2007 Mar 22;117(3):739-45. Epub 2007 Feb 22.

Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut 06536, USA.

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http://dx.doi.org/10.1172/JCI30400DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1797607PMC
March 2007

Therapeutic potential for microRNAs.

Adv Drug Deliv Rev 2007 Mar 16;59(2-3):101-14. Epub 2007 Mar 16.

Isis Pharmaceuticals, 1896 Rutherford Road, Carlsbad, California 92008, USA.

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http://dx.doi.org/10.1016/j.addr.2007.03.007DOI Listing
March 2007

Deficiency of adiponectin receptor 2 reduces diet-induced insulin resistance but promotes type 2 diabetes.

Endocrinology 2007 Feb 26;148(2):683-92. Epub 2006 Oct 26.

Type 2 Diabetes Drug Hunting Team, Lilly Research Laboratories, Eli Lilly and Company, Indianapolis, Indiana 46285, USA.

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https://academic.oup.com/endo/article-lookup/doi/10.1210/en.
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http://dx.doi.org/10.1210/en.2006-0708DOI Listing
February 2007

Mcl-1 is a relevant molecular target for antisense oligonucleotide strategies in gastric cancer cells.

Cancer Biol Ther 2006 Oct 26;5(10):1348-54. Epub 2006 Oct 26.

Department of Clinical Pharmacology, Section of Experimental Oncology/Molecular Pharmacology, Medical University Vienna, Vienna, Austria.

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http://dx.doi.org/10.4161/cbt.5.10.3224DOI Listing
October 2006

Antisense oligonucleotide therapy for neurodegenerative disease.

J Clin Invest 2006 Aug 27;116(8):2290-6. Epub 2006 Jul 27.

Ludwig Institute for Cancer Research, University of California, San Diego, La Jolla, California, USA.

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http://dx.doi.org/10.1172/JCI25424DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1518790PMC
August 2006

Critical role of stearoyl-CoA desaturase-1 (SCD1) in the onset of diet-induced hepatic insulin resistance.

J Clin Invest 2006 Jun;116(6):1686-95

Department of Medicine, Diabetes Research Center, Albert Einstein College of Medicine, Bronx, New York, USA.

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http://dx.doi.org/10.1172/JCI26991DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1464900PMC
June 2006

Raf-1 is the predominant Raf isoform that mediates growth factor-stimulated growth in ovarian cancer cells.

Carcinogenesis 2006 Apr 6;27(4):729-39. Epub 2005 Dec 6.

Cancer Research UK Centre, University of Edinburgh, Edinburgh EH4 2XR, UK.

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http://dx.doi.org/10.1093/carcin/bgi289DOI Listing
April 2006

Comparison of strategies targeting Raf-1 mRNA in ovarian cancer.

Int J Cancer 2006 Mar;118(6):1565-71

Cancer Research UK Centre, University of Edinburgh, Edinburgh, EH4 2XR, United Kingdom.

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http://dx.doi.org/10.1002/ijc.21520DOI Listing
March 2006

Mcl-1 overexpression in hepatocellular carcinoma: a potential target for antisense therapy.

J Hepatol 2006 Jan 25;44(1):151-7. Epub 2005 Oct 25.

Section of Experimental Oncology/Molecular Pharmacology, Department of Clinical Pharmacology, Medical University Vienna, Waehringer Guertel 18-20, 1090 Vienna, Austria.

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http://dx.doi.org/10.1016/j.jhep.2005.09.010DOI Listing
January 2006

Mcl-1 blocks radiation-induced apoptosis and inhibits clonogenic cell death.

Anticancer Res 2005 Jul-Aug;25(4):2697-703

Department of Radiotherapy and Radiobiology, University of Vienna, Waehringer Guertel 18-20, 1090 Vienna, Austria.

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September 2005