A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

(2018) Edith L. Pfister et al.   HUMAN GENE THERAPY

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

(2018) Melvin M. Evers et al.   MOLECULAR THERAPY

Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo

(2018) Matthew R Hassler et al.   NUCLEIC ACIDS RESEARCH

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

(2018) Maja M. Janas et al.   Nature Communications

Improving siRNA Delivery In Vivo Through Lipid Conjugation

(2018) Maire F. Osborn et al.   Nucleic Acid Therapeutics

Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier

(2018) Bruno M.D.C. Godinho et al.   MOLECULAR THERAPY

Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin

(2018) Marie-Cécile Didiot et al.   Cell Reports

Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease

(2018) Amber L. Southwell et al.   Science Translational Medicine

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol

(2017) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

(2017) Jayaprakash K. Nair et al.   NUCLEIC ACIDS RESEARCH

Assessing single-stranded oligonucleotide drug-induced effects in vitro reveals key risk factors for thrombocytopenia

(2017) Sabine Sewing et al.   PLoS One

Visualization of self-delivering hydrophobically modified siRNA cellular internalization

(2016) Socheata Ly et al.   NUCLEIC ACIDS RESEARCH

Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis

(2016) Guohao Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Glial Fibrillary Acidic Protein and Ubiquitin C-Terminal Hydrolase-L1 as Outcome Predictors in Traumatic Brain Injury

(2016) Riikka S.K. Takala et al.   World Neurosurgery

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

(2016) Mehran Nikan et al.   Molecular Therapy-Nucleic Acids

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

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

Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic TrivalentN-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo

(2015) Kallanthottathil G. Rajeev et al.   CHEMBIOCHEM

Phosphorothioate backbone modifications of nucleotide-based drugs are potent platelet activators

(2015) Ulrike Flierl et al.   JOURNAL OF EXPERIMENTAL MEDICINE

N17 Modifies Mutant Huntingtin Nuclear Pathogenesis and Severity of Disease in HD BAC Transgenic Mice

(2015) Xiaofeng Gu et al.   NEURON

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

(2015) Julia F Alterman et al.   Molecular Therapy-Nucleic Acids

Tissue Plasminogen Activator Contributes to Alterations of Neuronal Migration and Activity-Dependent Responses in Fragile X Mice

(2014) V. S. Achuta et al.   JOURNAL OF NEUROSCIENCE

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

Apolipoprotein E and Alzheimer disease: risk, mechanisms and therapy

(2013) Chia-Chen Liu et al.   Nature Reviews Neurology

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

(2012) Richard Grondin et al.   BRAIN

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

(2012) Holly B. Kordasiewicz et al.   NEURON

Novel Oligonucleotides Containing Two 3′-Ends Complementary to Target mRNA Show Optimal Gene-Silencing Activity

(2011) Lakshmi Bhagat et al.   JOURNAL OF MEDICINAL CHEMISTRY

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

(2010) Johannes Winkler et al.   ChemMedChem

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

(2010) Chiara Zuccato et al.   PHYSIOLOGICAL REVIEWS

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY

Neuroanatomic Profile of Polyglutamine Immunoreactivity in Huntington Disease Brains

(2009) Emily S. Herndon et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Association of APOE with age at onset of sporadic amyotrophic lateral sclerosis

(2008) Henrik Zetterberg et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES