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

Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets

(2019) Anthony Conway et al.   MOLECULAR THERAPY

On the Formation and Morphology of Lipid Nanoparticles Containing Ionizable Cationic Lipids and siRNA

(2018) Jayesh A. Kulkarni et al.   ACS Nano

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

Nucleoside-modified mRNA immunization elicits influenza virus hemagglutinin stalk-specific antibodies

(2018) Norbert Pardi et al.   Nature Communications

Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination

(2017) Norbert Pardi et al.   NATURE

Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge

(2017) Norbert Pardi et al.   Nature Communications

Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routes

(2015) Norbert Pardi et al.   JOURNAL OF CONTROLLED RELEASE

Microfluidic Mixing: A General Method for Encapsulating Macromolecules in Lipid Nanoparticle Systems

(2015) Alex K. K. Leung et al.   JOURNAL OF PHYSICAL CHEMISTRY B

Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals

(2015) Andreas Thess et al.   MOLECULAR THERAPY

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

(2013) Barbara L Mui et al.   Molecular Therapy-Nucleic Acids

Liposomal drug delivery systems: From concept to clinical applications

(2012) Theresa M. Allen et al.   ADVANCED DRUG DELIVERY REVIEWS

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

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

Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA

(2012) Nathan M Belliveau et al.   Molecular Therapy-Nucleic Acids

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