Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

(2020) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

RT-qPCR Methods to Support Pharmacokinetics and Drug Mechanism of Action to Advance Development of RNAi Therapeutics

(2020) Elena Castellanos-Rizaldos et al.   Nucleic Acid Therapeutics

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

(2019) Akin Akinc et al.   Nature Nanotechnology

Facile Synthesis, Geometry, and 2′-Substituent-Dependent in Vivo Activity of 5′-(E)- and 5′-(Z)-Vinylphosphonate-Modified siRNA Conjugates

(2018) Rubina Giare Parmar et al.   JOURNAL OF MEDICINAL CHEMISTRY

Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

(2018) Donald J. Foster et al.   MOLECULAR THERAPY

Intracellular Trafficking and Endosomal Release of Oligonucleotides: What We Know and What We Don’t

(2018) R.L. Juliano   Nucleic Acid Therapeutics

GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

(2018) Aaron D. Springer et al.   Nucleic Acid Therapeutics

RNA Therapeutics (Almost) Comes of Age: Targeting, Delivery and Endosomal Escape

(2018) Steven F. Dowdy et al.   Nucleic Acid Therapeutics

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   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

Intra-endosomal trafficking mediated by lysobisphosphatidic acid contributes to intracellular release of phosphorothioate-modified antisense oligonucleotides

(2017) Shiyu Wang et al.   NUCLEIC ACIDS RESEARCH

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

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

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2

(2016) Elad Elkayam et al.   NUCLEIC ACIDS RESEARCH

Visualization of self-delivering hydrophobically modified siRNA cellular internalization

(2016) Socheata Ly et al.   NUCLEIC ACIDS RESEARCH

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   NUCLEIC ACIDS RESEARCH

siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

(2015) Shigeo Matsuda et al.   ACS Chemical Biology

A role for peptides in overcoming endosomal entrapment in siRNA delivery — A focus on melittin

(2015) Kirk K. Hou et al.   BIOTECHNOLOGY ADVANCES

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

Biochemical isolation of Argonaute protein complexes by Ago-APP

(2015) Judith Hauptmann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Identification of the endosomal sorting complex required for transport-I (ESCRT-I) as an important modulator of anti-miR uptake by cancer cells

(2014) Timothy R. Wagenaar et al.   NUCLEIC ACIDS RESEARCH

The rough endoplasmatic reticulum is a central nucleation site of siRNA-mediated RNA silencing

(2013) Lukas Stalder et al.   EMBO JOURNAL

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling

(2013) Gaurav Sahay et al.   NATURE BIOTECHNOLOGY

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

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

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

Long-lived microRNA-Argonaute complexes in quiescent cells can be activated to regulate mitogenic responses

(2012) S. H. Olejniczak et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A rapid two-step method for isolation of functional primary mouse hepatocytes: cell characterization and asialoglycoprotein receptor based assay development

(2011) Mariano Severgnini et al.   CYTOTECHNOLOGY

RNA-Induced Silencing Complex-Bound Small Interfering RNA Is a Determinant of RNA Interference-Mediated Gene Silencing in Mice

(2011) J. Wei et al.   MOLECULAR PHARMACOLOGY

Analysis of microRNA turnover in mammalian cells following Dicer1 ablation

(2011) Michael P. Gantier et al.   NUCLEIC ACIDS RESEARCH

Argonaute proteins regulate microRNA stability: Increased microRNA abundance by Argonaute proteins is due to microRNA stabilization

(2011) Julia Winter et al.   RNA Biology

Endosomal escape pathways for delivery of biologicals

(2010) Amir K. Varkouhi et al.   JOURNAL OF CONTROLLED RELEASE

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Quantitative evaluation of siRNA delivery in vivo

(2010) Y. Pei et al.   RNA

RISC hitches onto endosome trafficking

(2009) Haruhiko Siomi et al.   NATURE CELL BIOLOGY

Silencing by small RNAs is linked to endosomal trafficking

(2009) Young Sik Lee et al.   NATURE CELL BIOLOGY

Multivesicular bodies associate with components of miRNA effector complexes and modulate miRNA activity

(2009) Derrick J. Gibbings et al.   NATURE CELL BIOLOGY

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

(2009) Mayumi Takahashi et al.   NUCLEIC ACIDS RESEARCH