New Articles

Inhibition of Vascular Permeability by Antisense-Mediated Inhibition of Plasma Kallikrein and Coagulation Factor 12

Bhattacharjee G, Revenko AS, Crosby JR, May C, Gao D, Zhao C, Monia BP, Macleod AR

Nucleic Acid Ther. 2013 Apr 12. [Epub ahead of print]

Reducing TMPRSS6 Ameliorates Hemochromatosis and β-thalassemia in Mice

Guo S, Casu C, Gardenghi S, Booten S, Aghajan M, Peralta R, Watt A, Freier S, Monia BP, Rivella S

J Clin Invest. 2013 Apr 1;123(4):1531-41

Antisense Oligonucleotide Inhibition of Apolipoprotein C-III Reduces Plasma Triglycerides in Rodents, Nonhuman Primates, and Humans

Graham MJ, Lee RG, Bell TA 3rd, Fu W, Mullick AE, Alexander VJ, Singleton W, Viney N, Geary R, Su JQ, Baker BF, Burkey J, Crooke ST, Crooke RM

Circ Res. 2013 Mar 29. [Epub ahead of print]

An Antisense Oligonucleotide Against SOD1 Delivered Intrathecally for Patients With SOD1 Familial Amyotrophic Lateral Sclerosis: a Phase 1, Randomised, First-in-Man Study

Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME

Lancet Neurol. 2013 Mar 28. pii: S1474-4422(13)70061-9

Comparison of the Pharmacological Profiles of Murine Antisense Oligonucleotides Targeting Apolipoprotein B and Microsomal Triglyceride Transfer Protein

Lee RG, Fu W, Graham MJ, Mullick AE, Sipe D, Gattis D, Bell TA, Booten S, Crooke RM

J Lipid Res. 2013 Mar;54(3):602-14

Rescue of Hearing and Vestibular Function by Antisense Oligonucleotides in a Mouse Model of Human Deafness

Lentz JJ, Jodelka FM, Hinrich AJ, McCaffrey KE, Farris HE, Spalitta MJ, Bazan NG, Duelli DM, Rigo F, Hastings ML

Nat Med. 2013 Mar;19(3):345-50

Transfection of siRNAs Can Alter miRNA Levels and Trigger Non-Specific Protein Degradation in Mammalian Cells

Liang XH, Hart CE, Crooke ST

Biochim Biophys Acta. 2013 Feb 8;1829(5):455-468

Clinical Pharmacokinetics of Second Generation Antisense Oligonucleotides

Expert Opin Drug Metab Toxicol. 2013 Feb;9(2):169-82

Yu RZ, Grundy JS, Geary RS

Transcriptional Silencing by Single-Stranded RNAs Targeting a Noncoding RNA That Overlaps a Gene Promoter

Matsui M, Prakash TP, Corey DR

ACS Chem Biol. 2013 Jan 18;8(1):122-6

Trimethylamine-N-Oxide, a Metabolite Associated with Atherosclerosis, Exhibits Complex Genetic and Dietary Regulation

Bennett BJ, Vallim TQ, Wang Z, Shih DM, Meng Y, Gregory J, Allayee H, Lee R, Graham M, Crooke R, Edwards PA, Hazen SL, Lusis AJ

Cell Metab. 2013 Jan 8;17(1):49-60

Synthesis, Duplex Stabilization and Structural Properties of a Fluorinated Carbocyclic LNA Analogue

Seth PP, Pallan PS, Swayze EE, Egli M

Chembiochem. 2013 Jan 2;14(1):58-62

The Role of the Carbohydrate Response Element-Binding Protein in Male Fructose-Fed Rats

Erion DM, Popov V, Hsiao JJ, Vatner D, Mitchell K, Yonemitsu S, Nagai Y, Kahn M, Gillum MP, Dong J, Murray SF, Manchem VP, Bhanot S, Cline GW, Shulman GI, Samuel VT

Endocrinology. 2013 Jan;154(1):36-44

The Transcription Factor GLI1 Mediates TGFβ1 Driven EMT in Hepatocellular Carcinoma via a SNAI1-Dependent Mechanism

Zheng X, Rumie Vittar NB, Gai X, Fernandez-Barrena MG, Moser CD, Hu C, Almada LL, McCleary-Wheeler AL, Elsawa SF, Vrabel AM, Shire AM, Comba A, Thorgeirsson SS, Kim Y, Liu Q, Fernandez-Zapico ME, Roberts LR

PLoS One. 2012;7(11):e49581

Renal Uptake and Tolerability of a 2'-o-Methoxyethyl Modified Antisense Oligonucleotide (ISIS 113715) in Monkey

Henry SP, Johnson M, Zanardi TA, Fey R, Auyeung D, Lappin PB, Levin AA

Toxicology. 2012 Nov 15;301(1-3):13-20

Effects of Perilipin 2 Antisense Oligonucleotide Treatment on Hepatic Lipid Metabolism and Gene Expression

Imai Y, Boyle S, Varela GM, Caron E, Yin X, Dhir R, Dhir R, Graham MJ, Ahima RS

Physiol Genomics. 2012 Nov 15;44(22):1125-31

A Selective Inhibitor of Human C-reactive Protein Translation Is Efficacious In Vitro and in C-reactive Protein Transgenic Mice and Humans

Jones NR, Pegues MA, McCrory MA, Singleton W, Bethune C, Baker BF, Norris DA, Crooke RM, Graham MJ, Szalai AJ

Molecular Therapy Nucleic Acids. 2012(1):e52

Apolipoprotein B Synthesis Inhibition with Mipomersen in Heterozygous Familial Hypercholesterolemia: Results of a Randomized, Double-Blind, Placebo Controlled Trial to Assess Efficacy and Safety as Add-on Therapy in Patients with Coronary Artery Disease

Stein EA, Dufour R, Gagne C, Gaudet D, East C, Donovan JM, Chin W, Tribble DL, McGowan M

Circulation. 2012 Nov 6;126(19):2283-92

Structure-Based Design of a Highly Constrained Nucleic Acid Analogue: Improved Duplex Stabilization by Restricting Sugar Pucker and Torsion Angle γ

Hanessian S, Schroeder BR, Giacometti RD, Merner BL, Ostergaard M, Swayze EE, Seth PP

Angew Chem Int Ed Engl. 2012 Nov 5;51(45):11242-5

Divergent Roles of ALS-Linked Proteins FUS/TLS and TDP-43 Intersect in Processing Long Pre-mRNAs

Lagier-Tourenne C, Polymenidou M, Hutt KR, Vu AQ, Baughn M, Huelga SC, Clutario KM, Ling SC, Liang TY, Mazur C, Wancewicz E, Kim AS, Watt A, Freier S, Hicks GG, Donohue JP, Shiue L, Bennett CF, Ravits J, Cleveland DW, Yeo GW

Nat Neurosci. 2012 Nov;15(11):1488-97

Metabolic Consequences of High-Fat Diet Are Attenuated by Suppression of HIF-1α

Shin M, Drager LF, Yao Q, Bevans-Fonti S, Yoo D, Jun JC, Aja S, Bhanot S, Polotsky VY

PLoS One. 2012;7(10):e46562

Antisense-Based Therapy for the Treatment of Spinal Muscular Atrophy

Rigo F, Hua Y, Krainer AR, Bennett CF

J Cell Biol. 2012 Oct 1;199(1):21-5

Manipulation of PK-M Mutually Exclusive Alternative Splicing by Antisense Oligonucleotides

Wang Z, Jeon HY, Rigo F, Bennett CF, Krainer AR

Open Biol. 2012 Oct;2(10):120133

Clinical Pharmacological Properties of Mipomersen (Kynamro), a Second Generation Antisense Inhibitor of Apolipoprotein B

Crooke ST, Geary RS

Br J Clin Pharmacol. 2012 Sep 26. doi: 10.1111/j.1365-2125.2012.04469

Structure Activity Relationships of α-L-LNA Modified Phosphorothioate Gapmer Antisense Oligonucleotides in Animals

Seth PP, Jazayeri A, Yu J, Allerson CR, Bhat B, Swayze EE

Mol Ther Nucleic Acids. 2012 Sep 18;1:e47

Single-Stranded siRNAs Activate RNAi in Animals

Lima WF, Prakash TP, Murray HM, Kinberger GA, Li W, Chappell AE, Li CS, Murray SF, Gaus H, Seth PP, Swayze EE, Crooke ST

Cell. 2012 Aug 31;150(5):883-94

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

Yu D, Pendergraff H, Liu J, Kordasiewicz HB, Cleveland DW, Swayze EE, Lima WF, Crooke ST, Prakash TP, Corey DR

Cell. 2012 Aug 31;150(5):895-908

Structure and Nuclease Resistance of 2',4'-Constrained 2'-O-Methoxyethyl (cMOE) and 2'-O-Ethyl (cEt) Modified DNAs

Pallan PS, Allerson CR, Berdeja A, Seth PP, Swayze EE, Prakash TP, Egli M

Chem Commun (Camb). 2012 Aug 25;48(66):8195-7

Pharmacodynamics and Subchronic Toxicity in Mice and Monkeys of ISIS 388626, a Second Generation Antisense Oligonucleotide that Targets the Human Sodium Glucose Cotransporter 2 (SGLT2)

Zanardi TA, Han SC, Jeong EJ, Rime S, Yu RZ, Chakravarty K, Henry SP

J Pharmacol Exp Ther. 2012 Nov;343(2):489-96

TSUNAMI: an Antisense Method to Phenocopy Splicing-Associated Diseases in Animals

Sahashi K, Hua Y, Ling KK, Hung G, Rigo F, Horev G, Katsuno M, Sobue G, Ko CP, Bennett CF, Krainer AR

Genes Dev. 2012 Aug 15;26(16):1874-84

Targeting Nuclear RNA for in Vivo Correction of Myotonic Dystrophy

Wheeler TM, Leger AJ, Pandey SK, MacLeod AR, Nakamori M, Cheng SH, Wentworth BM, Bennett CF, Thornton CA

Nature. 2012 Aug 2;488(7409):111-5

The lncRNA Malat1 Is Dispensable for Mouse Development but Its Transcription Plays a cis-Regulatory Role in the Adult

Zhang B, Arun G, Mao YS, Lazar Z, Hung G, Bhattacharjee G, Xiao X, Booth CJ, Wu J, Zhang C, Spector DL

Cell Rep. 2012 Jul 26;2(1):111-23

TricycloDNA-Modified Oligo-2'-Deoxyribonucleotides Reduce Scavenger Receptor B1 mRNA in Hepatic and Extra-Hepatic Tissues--a Comparative Study of Oligonucleotide Length, Design and Chemistry

Murray S, Ittig D, Koller E, Berdeja A, Chappell A, Prakash TP, Norrbom M, Swayze EE, Leumann CJ, Seth PP

Nucleic Acids Res. 2012 Jul;40(13):6135-43

Unique O-Methoxyethyl Ribose-DNA Chimeric Oligonucleotide Induces an Atypical Melanoma Differentiation-Associated Gene 5-Dependent Induction of Type I Interferon Response

Burel SA, Machemer T, Ragone FL, Kato H, Cauntay P, Greenlee S, Salim A, Gaarde WA, Hung G, Peralta R, Freier SM, Henry SP

J Pharmacol Exp Ther. 2012 Jul;342(1):150-62

siRNAs Targeted to Certain Polyadenylation Sites Promote Specific, RISC-Independent Degradation of Messenger RNAs

Vickers TA, Crooke ST

Nucleic Acids Res. 2012 Jul;40(13):6223-34

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Kordasiewicz HB, Stanek LM, Wancewicz EV, Mazur C, McAlonis MM, Pytel KA, Artates JW, Weiss A, Cheng SH, Shihabuddin LS, Hung G, Bennett CF, Cleveland DW

Neuron. 2012 Jun 21;74(6):1031-44

Clinical Development of an Antisense Therapy for the Treatment of Transthyretin-Associated Polyneuropathy

Ackermann EJ, Guo S, Booten S, Alvarado L, Benson M, Hughes S, Monia BP

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

The Use of Stable Isotope-Labeled Glycerol and Oleic Acid to Differentiate the Hepatic Functions of DGAT1 and -2

Qi J, Lang W, Geisler JG, Wang P, Petrounia I, Mai S, Smith C, Askari H, Struble GT, Williams R, Bhanot S, Monia BP, Bayoumy S, Grant E, Caldwell GW, Todd MJ, Liang Y, Gaul MD, Demarest KT, Connelly MA

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

Synthesis and Antisense Properties of Fluoro Cyclohexenyl Nucleic Acid (F-CeNA), a Nuclease Stable Mimic of 2'-Fluoro RNA

Seth PP, Yu J, Jazayeri A, Pallan PS, Allerson CR, Ostergaard ME, Liu F, Herdewijn P, Egli M, Swayze EE

J Org Chem. 2012 Jun 1;77(11):5074-85

Hepatic Hdac3 Promotes Gluconeogenesis by Repressing Lipid Synthesis and Sequestration

Sun Z, Miller RA, Patel RT, Chen J, Dhir R, Wang H, Zhang D, Graham MJ, Unterman TG, Shulman GI, Sztalryd C, Bennett MJ, Ahima RS, Birnbaum MJ, Lazar MA

Nat Med. 2012 Jun;18(6):934-42

Antisense Reduction of 11β-Hydroxysteroid Dehydrogenase Type 1 Enhances Energy Expenditure and Insulin Sensitivity iIdependent of Food Intake in C57BL/6J Mice on a Western-Type Diet

Li G, Hernandez-Ono A, Crooke RM, Graham MJ, Ginsberg HN

Metabolism. 2012 Jun;61(6):823-35

Inhibition of Hepatic Sulf2 in vivo: A Novel Strategy to Correct Diabetic Dyslipidemia

Hassing HC, Mooij H, Guo S, Monia BP, Chen K, Kulik W, Dallinga-Thie GM, Nieuwdorp M, Stroes ES, Williams KJ

Hepatology. 2012 Jun;55(6):1746-53

Quality Aspects of Oligonucleotide Drug Development: Specifications for Active Pharmaceutical Ingredients

Capaldi D, Ackley K, Brooks D, Carmody J, Draper K, Kambhampati R, Kretschmer M, Levin D, McArdle J, Noll B, Raghavachari R, Roymoulik I, Sharma BP, Thuermer R, Wincott F

Drug Information Journal, 46(5) 611-626

Mipomersen, an Apolipoprotein B Synthesis Inhibitor, Lowers Low-Density Lipoprotein Cholesterol in High-Risk Statin-Intolerant Patients: a Randomized, Double-Blind, Placebo-Controlled Trial

Visser ME, Wagener G, Baker BF, Geary RS, Donovan JM, Beuers UH, Nederveen AJ, Verheij J, Trip MD, Basart DC, Kastelein JJ, Stroes ES

Eur Heart J. 2012 May;33(9):1142-9