Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides

A Locked Nucleic Acid Oligonucleotide Targeting MicroRNA 122 Is Well-Tolerated in Cynomolgus Monkeys

(2018) Elisabeth S. Hildebrandt-Eriksen et al.   Nucleic Acid Therapeutics

Survival in a phase III, randomized, double-blind study of dacarbazine with or without oblimersen (Bcl-2 antisense) in patients with advanced melanoma and low-normal serum lactate dehydrogenase (LDH; AGENDA).

(2017) A. Y. Bedikian et al.   JOURNAL OF CLINICAL ONCOLOGY

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

(2012) Muthusamy Jayaraman et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Advanced morpholino oligomers: A novel approach to antiviral therapy

(2012) Travis K. Warren et al.   ANTIVIRAL RESEARCH

Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: a randomized, double-blind, placebo-controlled trial

(2012) Maartje E. Visser et al.   EUROPEAN HEART JOURNAL

Small heat shock proteins in cancer therapy and prognosis

(2012) Amina Zoubeidi et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Structure-dependent immunostimulatory effect of CpG oligodeoxynucleotides and their delivery system

(2012) Nobutaka Hanagata   International Journal of Nanomedicine

Dose-response effects of TPI ASM8 in asthmatics after allergen

(2011) G. M. Gauvreau et al.   ALLERGY

Phase 2 randomized study of p53 antisense oligonucleotide (cenersen) plus idarubicin with or without cytarabine in refractory and relapsed acute myeloid leukemia

(2011) Jorge Cortes et al.   CANCER

Randomized Phase II Trial of Custirsen (OGX-011) in Combination with Docetaxel or Mitoxantrone as Second-line Therapy in Patients with Metastatic Castrate-Resistant Prostate Cancer Progressing after First-line Docetaxel: CUOG Trial P-06c

(2011) F. Saad et al.   CLINICAL CANCER RESEARCH

Addition of AEG35156 XIAP Antisense Oligonucleotide in Reinduction Chemotherapy Does Not Improve Remission Rates in Patients With Primary Refractory Acute Myeloid Leukemia in a Randomized Phase II Study

(2011) Aaron D. Schimmer et al.   Clinical Lymphoma Myeloma & Leukemia

Intestinal delivery of non-viral gene therapeutics: physiological barriers and preclinical models

(2011) Martin J. O’Neill et al.   DRUG DISCOVERY TODAY

CpG DNA as a vaccine adjuvant

(2011) Christian Bode et al.   Expert Review of Vaccines

Antisense drug discovery and development

(2011) Tsuyoshi Yamamoto et al.   Future Medicinal Chemistry

Chemical and Mechanistic Toxicology Evaluation of Exon Skipping Phosphorodiamidate Morpholino Oligomers in mdx Mice

(2011) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Repeat-Dose Toxicology Evaluation in Cynomolgus Monkeys of AVI-4658, a Phosphorodiamidate Morpholino Oligomer (PMO) Drug for the Treatment of Duchenne Muscular Dystrophy

(2011) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

The long march of antisense

(2011) Dan Jones   NATURE REVIEWS DRUG DISCOVERY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

(2011) Erich Koller et al.   NUCLEIC ACIDS RESEARCH

Delivery of siRNA Therapeutics: Barriers and Carriers

(2010) Jie Wang et al.   AAPS Journal

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

(2010) Hong M. Moulton et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

A phase II study of the antisense oligonucleotide GTI-2040 plus docetaxel and prednisone as first-line treatment in castration-resistant prostate cancer

(2010) Srikala S. Sridhar et al.   CANCER CHEMOTHERAPY AND PHARMACOLOGY

Off-Target Effects Related to the Phosphorothioate Modification of Nucleic Acids

(2010) Johannes Winkler et al.   ChemMedChem

Genomic characterization of Wilms' tumor suppressor 1 targets in nephron progenitor cells during kidney development

(2010) S. Hartwig et al.   DEVELOPMENT

Morpholino artifacts provide pitfalls and reveal a novel role for pro-apoptotic genes in hindbrain boundary development

(2010) Sebastian S. Gerety et al.   DEVELOPMENTAL BIOLOGY

Akt inhibitors in clinical development for the treatment of cancer

(2010) Sumanta Kumar Pal et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

Mipomersen as a potential adjunctive therapy for hypercholesterolemia

(2010) Neeraj Patel et al.   EXPERT OPINION ON PHARMACOTHERAPY

Safety Pharmacology and Genotoxicity Evaluation of AVI-4658

(2010) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

CLINICAL TRIALS AND TRANSLATIONAL MEDICINE COMMENTARY: Drug Delivery Trends in Clinical Trials and Translational Medicine: Challenges and Opportunities in the Delivery of Nucleic Acid-Based Therapeutics

(2010) Long Xu et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Safety signal dampens reception for mipomersen antisense

(2010) Jim Kling   NATURE BIOTECHNOLOGY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Advanced antisense therapies for postexposure protection against lethal filovirus infections

(2010) Travis K Warren et al.   NATURE MEDICINE

Targeted therapy for high-grade glioma with the TGF- 2 inhibitor trabedersen: results of a randomized and controlled phase IIb study

(2010) U. Bogdahn et al.   NEURO-ONCOLOGY

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

Safety and efficacy of sodium caprate in promoting oral drug absorption: from in vitro to the clinic

(2009) Sam Maher et al.   ADVANCED DRUG DELIVERY REVIEWS

Chemical Modifications of Antisense Morpholino Oligomers Enhance Their Efficacy against Ebola Virus Infection

(2009) D. L. Swenson et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Cellular Uptake of Neutral Phosphorodiamidate Morpholino Oligomers

(2009) Patrick Iversen et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

Inhibition of Protein Tyrosine Phosphatase-1B with Antisense Oligonucleotides Improves Insulin Sensitivity and Increases Adiponectin Concentrations in Monkeys

(2009) Michael M. Swarbrick et al.   ENDOCRINOLOGY

Antisense oligonucleotide pharmacokinetics and metabolism

(2009) Richard S Geary   Expert Opinion on Drug Metabolism & Toxicology

Overcoming biological barriers to in vivo efficacy of antisense oligonucleotides

(2009) Paul J. White et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

Oral delivery of siRNA and antisense oligonucleotides

(2009) Saghir Akhtar   JOURNAL OF DRUG TARGETING

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

A proteomic study reveals unspecific apoptosis induction and reduction of glycolytic enzymes by the phosphorothioate antisense oligonucleotide oblimersen in human melanoma cells

(2009) Martina Stessl et al.   Journal of Proteomics

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Biological Barriers to Therapy with Antisense and siRNA Oligonucleotides

(2009) R. Juliano et al.   MOLECULAR PHARMACEUTICS

Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos

(2009) Jon Moulton et al.   MOLECULES

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents

(2009) C. A. Stein 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

A Primer for Morpholino Use in Zebrafish

(2009) Brent R. Bill et al.   Zebrafish

Targeting Bcl-2 protein in treatment of melanoma still requires further clarifications

(2008) M. Pisano et al.   ANNALS OF ONCOLOGY

Antisense Treatment for Myasthenia Gravis

(2008) Jon D. Sussman et al.   Annals of the New York Academy of Sciences

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

Vivo-Morpholinos: A non-peptide transporter delivers Morpholinos into a wide array of mouse tissues

(2008) Paul A. Morcos et al.   BIOTECHNIQUES

Nucleic Acids as Therapeutic Agents

(2008) Luis Alvarez-Salas   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Specific VDAC inhibitors: phosphorothioate oligonucleotides

(2008) C.A. Stein et al.   JOURNAL OF BIOENERGETICS AND BIOMEMBRANES

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

A RNA antagonist of hypoxia-inducible factor-1 , EZN-2968, inhibits tumor cell growth

(2008) L. M. Greenberger et al.   MOLECULAR CANCER THERAPEUTICS

Advantageous toxicity profile of inhaled antisense oligonucleotides following chronic dosing in non-human primates

(2008) Alain Guimond et al.   PULMONARY PHARMACOLOGY & THERAPEUTICS