PFRED: A computational platform for siRNA and antisense oligonucleotides design

Efficacy and Safety of Mipomersen: A Systematic Review and Meta-Analysis of Randomized Clinical Trials

(2019) Federica Fogacci et al.   DRUGS

siRNA-Finder (si-Fi) Software for RNAi-Target Design and Off-Target Prediction

(2019) Stefanie Lück et al.   Frontiers in Plant Science

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Patisiran: First Global Approval

(2018) Sheridan M. Hoy   DRUGS

Spinraza—a rare disease success story

(2017) V Prakash   GENE THERAPY

HELM Software for Biopolymers

(2017) Jeff Milton et al.   Journal of Chemical Information and Modeling

Efficient Synthesis and Biological Evaluation of 5′-GalNAc Conjugated Antisense Oligonucleotides

(2015) Michael E. Østergaard et al.   BIOCONJUGATE CHEMISTRY

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides

(2014) Andrew D. Burdick et al.   NUCLEIC ACIDS RESEARCH

Antisense Oligonucleotide Therapies

(2014) Kendall S. Frazier   TOXICOLOGIC PATHOLOGY

HELM: A Hierarchical Notation Language for Complex Biomolecule Structure Representation

(2012) Tianhong Zhang et al.   Journal of Chemical Information and Modeling

Improved nucleic acid descriptors for siRNA efficacy prediction

(2012) Simone Sciabola et al.   NUCLEIC ACIDS RESEARCH

MysiRNA-Designer: A Workflow for Efficient siRNA Design

(2011) Mohamed Mysara et al.   PLoS One

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

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

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

Clinical status of duplex RNA

(2010) Jonathan K. Watts et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

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

(2010) Frederick J Raal et al.   LANCET

A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus

(2010) J. DeVincenzo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

siDirect 2.0: updated software for designing functional siRNA with reduced seed-dependent off-target effect

(2009) Yuki Naito et al.   BMC BIOINFORMATICS

siRNAs: their potential as therapeutic agents – Part I. Designing of siRNAs

(2009) Praveensingh B. Hajeri et al.   DRUG DISCOVERY TODAY

Ultrafast and memory-efficient alignment of short DNA sequences to the human genome

(2009) Ben Langmead et al.   GENOME BIOLOGY

Theoretical Analysis of Antisense Duplexes:  Determinants of the RNase H Susceptibility

(2008) Agnes Noy et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

OligoWalk: an online siRNA design tool utilizing hybridization thermodynamics

(2008) Z. J. Lu et al.   NUCLEIC ACIDS RESEARCH