Antisense drug discovery and development

Effect of Mipomersen, an Apolipoprotein B Synthesis Inhibitor, on Low-Density Lipoprotein Cholesterol in Patients With Familial Hypercholesterolemia

(2010) Fatima Akdim et al.   AMERICAN JOURNAL OF CARDIOLOGY

Development of potential anticancer agents that target the telomere sequence

(2010) Myunji Park et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Synthesis of Bicyclonucleosides Having a C−C Bridge†

(2010) Jacques Lebreton et al.   CHEMICAL REVIEWS

Nucleic Acids with a Six-Membered ‘Carbohydrate’ Mimic in the Backbone

(2010) Piet Herdewijn   CHEMISTRY & BIODIVERSITY

Oligonucleotide N3′→P5′ Phosphoramidates andThio-Phoshoramidates as Potential Therapeutic Agents

(2010) Sergei M. Gryaznov   CHEMISTRY & BIODIVERSITY

Apolipoprotein B synthesis inhibition: results from clinical trials

(2010) Maartje E Visser et al.   CURRENT OPINION IN LIPIDOLOGY

Molecular treatments in Duchenne muscular dystrophy

(2010) Michela Guglieri et al.   CURRENT OPINION IN PHARMACOLOGY

Bridged Nucleic Acids: Development, Synthesis and Properties

(2010) Satoshi Obika et al.   HETEROCYCLES

Antisense Oligonucleotides Containing Conformationally Constrained 2′,4′-(N-Methoxy)aminomethylene and 2′,4′-Aminooxymethylene and 2′-O,4′-C-Aminomethylene Bridged Nucleoside Analogues Show Improved Potency in Animal Models

(2010) Thazha P. Prakash et al.   JOURNAL OF MEDICINAL CHEMISTRY

Synthesis and Biophysical Evaluation of 2′,4′-Constrained 2′O-Methoxyethyl and 2′,4′-Constrained 2′O-Ethyl Nucleic Acid Analogues

(2010) Punit P. Seth et al.   JOURNAL OF ORGANIC CHEMISTRY

An Exocyclic Methylene Group Acts As a Bioisostere of the 2′-Oxygen Atom in LNA

(2010) Punit P. Seth et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Efficacy and Safety of Mipomersen, an Antisense Inhibitor of Apolipoprotein B, in Hypercholesterolemic Subjects Receiving Stable Statin Therapy

(2010) Fatima Akdim et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Optimization of Peptide Nucleic Acid Antisense Oligonucleotides for Local and Systemic Dystrophin Splice Correction in the mdx Mouse

(2010) HaiFang Yin et al.   MOLECULAR THERAPY

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates

(2010) Ellen Marie Straarup et al.   NUCLEIC ACIDS RESEARCH

Efficient inhibition of miR-155 function in vivo by peptide nucleic acids

(2010) Martin M. Fabani et al.   NUCLEIC ACIDS RESEARCH

A Locked Nucleic Acid Antisense Oligonucleotide (LNA) Silences PCSK9 and Enhances LDLR Expression In Vitro and In Vivo

(2010) Nidhi Gupta et al.   PLoS One

Progress in muscular dystrophy research with special emphasis on gene therapy

(2010) Hideo SUGITA et al.   PROCEEDINGS OF THE JAPAN ACADEMY SERIES B-PHYSICAL AND BIOLOGICAL SCIENCES

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

5-Year Survival in Patients With Relapsed or Refractory Chronic Lymphocytic Leukemia in a Randomized, Phase III Trial of Fludarabine Plus Cyclophosphamide With or Without Oblimersen

(2009) Susan O'Brien et al.   JOURNAL OF CLINICAL ONCOLOGY

Phase I/II Trial of AEG35156 X-Linked Inhibitor of Apoptosis Protein Antisense Oligonucleotide Combined With Idarubicin and Cytarabine in Patients With Relapsed or Primary Refractory Acute Myeloid Leukemia

(2009) Aaron D. Schimmer et al.   JOURNAL OF CLINICAL ONCOLOGY

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

(2009) Hans A. Heemskerk et al.   JOURNAL OF GENE MEDICINE

Synthesis of Functionalized Carbocyclic Locked Nucleic Acid Analogues by Ring-Closing Diene and Enyne Metathesis and Their Influence on Nucleic Acid Stability and Structure

(2009) Surender Kumar et al.   JOURNAL OF ORGANIC CHEMISTRY

Interplay of Structure, Hydration and Thermal Stability in Formacetal Modified Oligonucleotides: RNA May Tolerate Nonionic Modifications Better than DNA

(2009) Andrej Kolarovic et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Cationic PMMA Nanoparticles Bind and Deliver Antisense Oligoribonucleotides Allowing Restoration of Dystrophin Expression in the mdx Mouse

(2009) Paola Rimessi et al.   MOLECULAR THERAPY

Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

(2009) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos

(2009) Jon Moulton et al.   MOLECULES

Synthesis and characterization of 2′-modified-4′-thioRNA: a comprehensive comparison of nuclease stability

(2009) Mayumi Takahashi et al.   NUCLEIC ACIDS RESEARCH

Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection

(2009) R. E. Lanford et al.   SCIENCE

Short peptide nucleic acids (PNA) inhibit hepatitis C virus internal ribosome entry site (IRES) dependent translation in vitro

(2008) C ALOTTE et al.   ANTIVIRAL RESEARCH

Cross-species comparison of in vivo PK/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100

(2008) Rosie Z. Yu et al.   BIOCHEMICAL PHARMACOLOGY

Synthesis and properties of 3′-amino-2′,4′-BNA, a bridged nucleic acid with a N3′→P5′ phosphoramidate linkage

(2008) Satoshi Obika et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Chemically modified oligonucleotides with efficient RNase H response

(2008) Birte Vester et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Incorporation of positively charged ribonucleic guanidine linkages into oligodeoxyribonucleotides: Development of potent antisense agents

(2008) Myunji Park et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Antisense Oligonucleotide Directed to Human Apolipoprotein B-100 Reduces Lipoprotein(a) Levels and Oxidized Phospholipids on Human Apolipoprotein B-100 Particles in Lipoprotein(a) Transgenic Mice

(2008) Esther Merki et al.   CIRCULATION

Controlling morpholino experiments: don't stop making antisense

(2008) J. S. Eisen et al.   DEVELOPMENT

Antisense, RNAi, and gene silencing strategies for therapy: Mission possible or impossible?

(2008) E RAYBURN et al.   DRUG DISCOVERY TODAY

Recent Approaches in the Synthesis of Conformationally Restricted Nucleoside Analogues

(2008) Christophe Mathé et al.   EUROPEAN JOURNAL OF ORGANIC CHEMISTRY

Comparing In Vitro and In Vivo Activity of 2′-O-[2-(Methylamino)-2-oxoethyl]- and 2′-O-Methoxyethyl-Modified Antisense Oligonucleotides

(2008) Thazha P. Prakash et al.   JOURNAL OF MEDICINAL CHEMISTRY

Short Antisense Oligonucleotides with Novel 2′−4′ Conformationaly Restricted Nucleoside Analogues Show Improved Potency without Increased Toxicity in Animals

(2008) Punit P. Seth et al.   JOURNAL OF MEDICINAL CHEMISTRY

Solid-Phase Synthesis of Stereoregular Oligodeoxyribonucleoside Phosphorothioates Using Bicyclic Oxazaphospholidine Derivatives as Monomer Units

(2008) Natsuhisa Oka et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Design, Synthesis, and Properties of 2‘,4‘-BNANC:  A Bridged Nucleic Acid Analogue

(2008) S. M. Abdur Rahman et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY

LNA-mediated microRNA silencing in non-human primates

(2008) Joacim Elmén et al.   NATURE

Unexpected A-form formation of 4′-thioDNA in solution, revealed by NMR, and the implications as to the mechanism of nuclease resistance

(2008) Akimasa Matsugami et al.   NUCLEIC ACIDS RESEARCH

Improved cell-penetrating peptide–PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle

(2008) Gabriela D. Ivanova et al.   NUCLEIC ACIDS RESEARCH

Biodistribution of 68Ga-Labeled LNA–DNA Mixmer Antisense Oligonucleotides for Rat Chromogranin-A

(2008) Gabor Lendvai et al.   OLIGONUCLEOTIDES

Complexation of single strand telomere and telomerase RNA template polyanions by deoxyribonucleic guanidine (DNG) polycations: Plausible anticancer agents

(2007) Xiaohua Zhang et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Oral Delivery of Antisense Oligonucleotides in Man

(2007) Lloyd G. Tillman et al.   JOURNAL OF PHARMACEUTICAL SCIENCES