Dendrimer-Based Lipid Nanoparticles Deliver Therapeutic FAH mRNA to Normalize Liver Function and Extend Survival in a Mouse Model of Hepatorenal Tyrosinemia Type I

The Polyploid State Plays a Tumor-Suppressive Role in the Liver

(2018) Shuyuan Zhang et al.   DEVELOPMENTAL CELL

Knockdown of Anillin Actin Binding Protein Blocks Cytokinesis in Hepatocytes and Reduces Liver Tumor Development in Mice Without Affecting Regeneration

(2018) Shuyuan Zhang et al.   GASTROENTEROLOGY

Lipid Nanoparticle Formulations for Enhanced Co-delivery of siRNA and mRNA

(2018) Rebecca L. Ball et al.   NANO LETTERS

Diagnosis and treatment of tyrosinemia type I: a US and Canadian consensus group review and recommendations

(2017) Jeffrey M Chinsky et al.   GENETICS IN MEDICINE

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

(2017) Randy J. Chandler et al.   HUMAN GENE THERAPY

Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA

(2017) Siddharth Patel et al.   NANO LETTERS

Systemic delivery of factor IX messenger RNA for protein replacement therapy

(2017) Suvasini Ramaswamy et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Tools for translation: non-viral materials for therapeutic mRNA delivery

(2017) Khalid A. Hajj et al.   Nature Reviews Materials

The role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide delivery

(2016) Xinwei Cheng et al.   ADVANCED DRUG DELIVERY REVIEWS

Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery

(2016) Owen S. Fenton et al.   ADVANCED MATERIALS

A Single Methylene Group in Oligoalkylamine-Based Cationic Polymers and Lipids Promotes Enhanced mRNA Delivery

(2016) Anita Jarzębińska et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

(2016) Jason B. Miller et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs

(2016) James C. Kaczmarek et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

(2016) F DeRosa et al.   GENE THERAPY

Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo

(2016) Yizhou Dong et al.   NANO LETTERS

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Functional polyesters enable selective siRNA delivery to lung cancer over matched normal cells

(2016) Yunfeng Yan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Modular degradable dendrimers enable small RNAs to extend survival in an aggressive liver cancer model

(2016) Kejin Zhou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia

(2016) Francis P. Pankowicz et al.   Nature Communications

Effects of local structural transformation of lipid-like compounds on delivery of messenger RNA

(2016) Bin Li et al.   Scientific Reports

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

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

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

Rapid Synthesis of a Lipocationic Polyester Library via Ring-Opening Polymerization of Functional Valerolactones for Efficacious siRNA Delivery

(2015) Jing Hao et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo

(2015) Bin Li et al.   NANO LETTERS

Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

(2015) Kevin J. Kauffman et al.   NANO LETTERS

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

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

Precise let-7 expression levels balance organ regeneration against tumor suppression

(2015) Linwei Wu et al.   eLife

Modulated Protonation of Side Chain Aminoethylene Repeats in N-Substituted Polyaspartamides Promotes mRNA Transfection

(2014) Hirokuni Uchida et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

(2014) James E. Dahlman et al.   Nature Nanotechnology

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

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

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

Correction to “Lipid Nanoparticles Containing siRNA Synthesized by Microfluidic Mixing Exhibit an Electron-Dense Nanostructured Core”

(2012) Alex K. K. Leung et al.   Journal of Physical Chemistry C

Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection

(2012) Benjamin Petsch et al.   NATURE BIOTECHNOLOGY

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY