Systemic delivery of mRNA and DNA to the lung using polymer-lipid nanoparticles

Poly(Beta-Amino Ester) Nanoparticles Enable Nonviral Delivery of CRISPR-Cas9 Plasmids for Gene Knockout and Gene Deletion

(2020) Yuan Rui et al.   Molecular Therapy-Nucleic Acids

Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium

(2019) Asha Kumari Patel et al.   ADVANCED MATERIALS

One Size Does Not Fit All: The Effect of Chain Length and Charge Density of Poly(ethylene imine) Based Copolymers on Delivery of pDNA, mRNA, and RepRNA Polyplexes

(2018) Anna K. Blakney et al.   BIOMACROMOLECULES

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

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

Rapid, Single-Cell Analysis and Discovery of Vectored mRNA Transfection In Vivo with a loxP-Flanked tdTomato Reporter Mouse

(2018) Kevin J. Kauffman et al.   Molecular Therapy-Nucleic Acids

Regulation of global translation during the cell cycle

(2018) Vilte Stonyte et al.   JOURNAL OF CELL SCIENCE

Optimization of a Degradable Polymer–Lipid Nanoparticle for Potent Systemic Delivery of mRNA to the Lung Endothelium and Immune Cells

(2018) James C. Kaczmarek et al.   NANO LETTERS

Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA

(2017) Siddharth Patel et al.   NANO LETTERS

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Advances in the delivery of RNA therapeutics: from concept to clinical reality

(2017) James C. Kaczmarek et al.   Genome Medicine

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

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

Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo

(2016) Kevin J. Kauffman et al.   BIOMATERIALS

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

(2016) F DeRosa et al.   GENE THERAPY

Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy

(2016) Lena M. Kranz et al.   NATURE

Codon Optimality Is a Major Determinant of mRNA Stability

(2015) Vladimir Presnyak et al.   CELL

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

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

(2015) Azita J Mahiny et al.   NATURE BIOTECHNOLOGY

mRNA-based therapeutics — developing a new class of drugs

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

Degradable Terpolymers with Alkyl Side Chains Demonstrate Enhanced Gene Delivery Potency and Nanoparticle Stability

(2013) Ahmed A. Eltoukhy et al.   ADVANCED MATERIALS

Gene therapy and DNA delivery systems

(2013) D. Ibraheem et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

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

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

(2013) Lior Zangi 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

Subtle Changes to Polymer Structure and Degradation Mechanism Enable Highly Effective Nanoparticles for siRNA and DNA Delivery to Human Brain Cancer (Adv. Healthcare Mater. 3/2013)

(2013) Stephany Y. Tzeng et al.   Advanced Healthcare Materials

In Vitro–In Vivo Translation of Lipid Nanoparticles for Hepatocellular siRNA Delivery

(2012) Kathryn A. Whitehead et al.   ACS Nano

Effect of molecular weight of amine end-modified poly(β-amino ester)s on gene delivery efficiency and toxicity

(2012) Ahmed A. Eltoukhy et al.   BIOMATERIALS

Rapid Discovery of Potent siRNA-Containing Lipid Nanoparticles Enabled by Controlled Microfluidic Formulation

(2012) Delai Chen et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Comparison of the Gene Transfer Efficiency of mRNA/GL67 and pDNA/GL67 Complexes in Respiratory Cells

(2012) Oliwia Andries et al.   MOLECULAR PHARMACEUTICS

Systemic Delivery of Modified mRNA Encoding Herpes Simplex Virus 1 Thymidine Kinase for Targeted Cancer Gene Therapy

(2012) Yuhua Wang et al.   MOLECULAR THERAPY

Effects of Base Polymer Hydrophobicity and End-Group Modification on Polymeric Gene Delivery

(2011) Joel C. Sunshine et al.   BIOMACROMOLECULES

Endosome maturation

(2011) Jatta Huotari et al.   EMBO JOURNAL

Global quantification of mammalian gene expression control

(2011) Björn Schwanhäusser et al.   NATURE

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

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

Endocytosis of nanomedicines

(2010) Gaurav Sahay 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

Quantitative Comparison of Constitutive Promoters in Human ES cells

(2010) Karin Norrman et al.   PLoS One

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

(2008) Katalin Karikó et al.   MOLECULAR THERAPY