Design of synthetic materials for intracellular delivery of RNAs: From siRNA-mediated gene silencing to CRISPR/Cas gene editing

Development of Cationic Quaternary Ammonium Sulfonamide Amino Lipids for Nucleic Acid Delivery

(2018) Jason B. Miller et al.   ACS Applied Materials & Interfaces

Macrophage-Specific in Vivo Gene Editing Using Cationic Lipid-Assisted Polymeric Nanoparticles

(2018) Ying-Li Luo et al.   ACS Nano

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

Incorporation of mRNA in Lamellar Lipid Matrices for Parenteral Administration

(2018) Antje Ziller et al.   MOLECULAR PHARMACEUTICS

A Direct Comparison of in Vitro and in Vivo Nucleic Acid Delivery Mediated by Hundreds of Nanoparticles Reveals a Weak Correlation

(2018) Kalina Paunovska et al.   NANO LETTERS

mRNA vaccines — a new era in vaccinology

(2018) Norbert Pardi et al.   NATURE REVIEWS DRUG DISCOVERY

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

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

Evolved Cas9 variants with broad PAM compatibility and high DNA specificity

(2018) Johnny H. Hu et al.   NATURE

Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems

(2017) S Guan et al.   GENE THERAPY

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

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

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Elimination of large tumors in mice by mRNA-encoded bispecific antibodies

(2017) Christiane R Stadler et al.   NATURE MEDICINE

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

(2017) David R Corey   NATURE NEUROSCIENCE

MicroRNA therapeutics: towards a new era for the management of cancer and other diseases

(2017) Rajesha Rupaimoole et al.   NATURE REVIEWS DRUG DISCOVERY

N1-methyl-pseudouridine in mRNA enhances translation through eIF2α-dependent and independent mechanisms by increasing ribosome density

(2017) Yuri V. Svitkin et al.   NUCLEIC ACIDS RESEARCH

Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals

(2017) Colin J. McKinlay et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

(2017) Yusuke Echigoya et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

RNAi-based treatment of chronically infected patients and chimpanzees reveals that integrated hepatitis B virus DNA is a source of HBsAg

(2017) Christine I. Wooddell et al.   Science Translational Medicine

Delivering the right message: Challenges and opportunities in lipid nanoparticles-mediated modified mRNA therapeutics—An innate immune system standpoint

(2017) Yasmin Granot et al.   SEMINARS IN IMMUNOLOGY

Eteplirsen in the treatment of Duchenne muscular dystrophy

(2017) Kenji Rowel Lim et al.   Drug Design Development and Therapy

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2017) Ding An et al.   Cell Reports

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

(2017) Ngoc Lu-Nguyen et al.   Molecular Therapy-Nucleic Acids

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

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

Improved base excision repair inhibition and bacteriophage Mu Gam protein yields C:G-to-T:A base editors with higher efficiency and product purity

(2017) Alexis C. Komor et al.   Science Advances

Current preclinical small interfering RNA (siRNA)-based conjugate systems for RNA therapeutics

(2016) Soo Hyeon Lee et al.   ADVANCED DRUG DELIVERY REVIEWS

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

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

Recent progress in development of siRNA delivery vehicles for cancer therapy

(2016) Hyun Jin Kim 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

RNA Interference (RNAi)-Based Therapeutics: Delivering on the Promise?

(2016) Maggie L. Bobbin et al.   Annual Review of Pharmacology and Toxicology

Effects of Chemically Modified Messenger RNA on Protein Expression

(2016) Bin Li et al.   BIOCONJUGATE CHEMISTRY

Chemically modified RNA induces osteogenesis of stem cells and human tissue explants as well as accelerates bone healing in rats

(2016) Elizabeth R. Balmayor et al.   BIOMATERIALS

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

Non-viral delivery of genome-editing nucleases for gene therapy

(2016) M Wang et al.   GENE THERAPY

Lipid nanoparticles for targeted siRNA delivery – going from bench to bedside

(2016) Victor Kotelianski et al.   International Journal of Nanomedicine

Enhanced intranasal delivery of mRNA vaccine by overcoming the nasal epithelial barrier via intra- and paracellular pathways

(2016) Man Li et al.   JOURNAL OF CONTROLLED RELEASE

Materials for non-viral intracellular delivery of messenger RNA therapeutics

(2016) Kevin J. Kauffman et al.   JOURNAL OF CONTROLLED RELEASE

Direct Pharmacological Inhibition of  -Catenin by RNA Interference in Tumors of Diverse Origin

(2016) S. Ganesh et al.   MOLECULAR CANCER THERAPEUTICS

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

(2016) Yizhou Dong et al.   NANO LETTERS

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

(2016) Lena M. Kranz et al.   NATURE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

(2016) Christopher D Richardson et al.   NATURE BIOTECHNOLOGY

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

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Non-coding RNAs as drug targets

(2016) Masayuki Matsui et al.   NATURE REVIEWS DRUG DISCOVERY

mRNA capping: biological functions and applications

(2016) Anand Ramanathan et al.   NUCLEIC ACIDS RESEARCH

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

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

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

Progress towards the Synthesis of Amino Polyesters via Ring-Opening Polymerization (ROP) of Functional Lactones

(2016) Daniel Siegwart et al.   SYNLETT

Self-adjuvanted mRNA vaccines induce local innate immune responses that lead to a potent and boostable adaptive immunity

(2016) Aleksandra Kowalczyk et al.   VACCINE

Utilising inorganic nanocarriers for gene delivery

(2016) Xian Jun Loh et al.   Biomaterials Science

Lipid Nanoparticle-mediated siRNA Transfer Against PCTAIRE1/PCTK1/Cdk16 Inhibits In Vivo Cancer Growth

(2016) Teruki Yanagi et al.   Molecular Therapy-Nucleic Acids

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

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

Production and clinical development of nanoparticles for gene delivery

(2016) Jie Chen et al.   Molecular Therapy-Methods & Clinical Development

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

(2016) Bin Li et al.   Scientific Reports

Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing

(2015) Wujin Sun et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Genome-wide CRISPR Screen in a Mouse Model of Tumor Growth and Metastasis

(2015) Sidi Chen et al.   CELL

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Technologies for controlled, local delivery of siRNA

(2015) Samantha M. Sarett et al.   JOURNAL OF CONTROLLED RELEASE

Theranostic applications of carbon nanomaterials in cancer: Focus on imaging and cargo delivery

(2015) Daiqin Chen et al.   JOURNAL OF CONTROLLED RELEASE

Going beyond the liver: Progress and challenges of targeted delivery of siRNA therapeutics

(2015) Cornelia Lorenzer 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

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

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

(2015) Andreas Thess et al.   MOLECULAR THERAPY

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

Clinical experiences with systemically administered siRNA-based therapeutics in cancer

(2015) Jonathan E. Zuckerman et al.   NATURE REVIEWS DRUG DISCOVERY

One-pot synthesis of functional poly(amino ester sulfide)s and utility in delivering pDNA and siRNA

(2015) Yunfeng Yan et al.   POLYMER

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

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

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

CRISPR-Cas9 delivery to hard-to-transfect cells via membrane deformation

(2015) X. Han et al.   Science Advances

Self-adjuvanted mRNA vaccination in advanced prostate cancer patients: a first-in-man phase I/IIa study

(2015) Hubert Kübler et al.   Journal for ImmunoTherapy of Cancer

Bioreducible Cationic Polymer-Based Nanoparticles for Efficient and Environmentally Triggered Cytoplasmic siRNA Delivery to Primary Human Brain Cancer Cells

(2014) Kristen L. Kozielski et al.   ACS Nano

Molecular Understanding and Design of Zwitterionic Materials

(2014) Qing Shao et al.   ADVANCED MATERIALS

Novel Endosomolytic Poly(amido amine) Polymer Conjugates for Systemic Delivery of siRNA to Hepatocytes in Rodents and Nonhuman Primates

(2014) Rubina Giare Parmar et al.   BIOCONJUGATE CHEMISTRY

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA

(2014) Hiroshi Nishimasu et al.   CELL

Sulfated quaternary amine lipids: a new class of inverse charge zwitterlipids

(2014) Vincent J. Venditto et al.   CHEMICAL COMMUNICATIONS

First-in-Human Phase I Study of the Liposomal RNA Interference Therapeutic Atu027 in Patients With Advanced Solid Tumors

(2014) Beate Schultheis et al.   JOURNAL OF CLINICAL ONCOLOGY

Designer lipids for drug delivery: From heads to tails

(2014) Aditya G. Kohli et al.   JOURNAL OF CONTROLLED RELEASE

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

Systemic Delivery of a miR34a Mimic as a Potential Therapeutic for Liver Cancer

(2014) C. L. Daige et al.   MOLECULAR CANCER THERAPEUTICS

Delivery of Therapeutic siRNA to the Lung Endothelium via Novel Lipoplex Formulation DACC

(2014) V Fehring et al.   MOLECULAR THERAPY

Drug development: Try and try again

(2014) Megan Scudellari   NATURE

Rapid modelling of cooperating genetic events in cancer through somatic genome editing

(2014) Francisco J. Sánchez-Rivera et al.   NATURE

CRISPR-mediated direct mutation of cancer genes in the mouse liver

(2014) Wen Xue et al.   NATURE

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg et al.   NATURE

Efficient delivery of RNAi prodrugs containing reversible charge-neutralizing phosphotriester backbone modifications

(2014) Bryan R Meade et al.   NATURE BIOTECHNOLOGY

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

CRISPR-Cas systems for editing, regulating and targeting genomes

(2014) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

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

Drugging the undruggable RAS: Mission Possible?

(2014) Adrienne D. Cox et al.   NATURE REVIEWS DRUG DISCOVERY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

Cystic fibrosis genetics: from molecular understanding to clinical application

(2014) Garry R. Cutting   NATURE REVIEWS GENETICS

Synthesis, properties, and biological activity of boranophosphate analogs of the mRNA cap: versatile tools for manipulation of therapeutically relevant cap-dependent processes

(2014) Joanna Kowalska et al.   NUCLEIC ACIDS RESEARCH

Correlating animal and human phase Ia/Ib clinical data with CALAA-01, a targeted, polymer-based nanoparticle containing siRNA

(2014) J. E. Zuckerman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

(2014) Y. Dong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

A critical analysis of codon optimization in human therapeutics

(2014) Vincent P. Mauro et al.   TRENDS IN MOLECULAR MEDICINE

Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity

(2014) Kathryn A. Whitehead et al.   Nature Communications

Lipid-Like Nanomaterials for Simultaneous Gene Expression and Silencing In Vivo

(2014) Yizhou Dong et al.   Advanced Healthcare Materials

The Role of Surface Functionality in Determining Nanoparticle Cytotoxicity

(2013) Sung Tae Kim et al.   ACCOUNTS OF CHEMICAL RESEARCH

The Headgroup Evolution of Cationic Lipids for Gene Delivery

(2013) Defu Zhi et al.   BIOCONJUGATE CHEMISTRY

Endosomolytic Bioreducible Poly(amido amine disulfide) Polymer Conjugates for the in Vivo Systemic Delivery of siRNA Therapeutics

(2013) Rubina Giare Parmar et al.   BIOCONJUGATE CHEMISTRY

A bioreducible linear poly(β-amino ester) for siRNA delivery

(2013) Kristen L. Kozielski et al.   CHEMICAL COMMUNICATIONS

Nanoparticles encapsulating hepatitis B virus cytosine-phosphate-guanosine induce therapeutic immunity against HBV infection

(2013) Shujuan Lv et al.   HEPATOLOGY

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

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Knockdown of β-catenin with Dicer-Substrate siRNAs Reduces Liver Tumor Burden In vivo

(2013) Henryk Dudek 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

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

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

MicroRNAs and other non-coding RNAs as targets for anticancer drug development

(2013) Hui Ling et al.   NATURE REVIEWS DRUG DISCOVERY

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

Role of Phospholipids in Endocytosis, Phagocytosis, and Macropinocytosis

(2013) Michal Bohdanowicz et al.   PHYSIOLOGICAL REVIEWS

Scalable synthesis and derivation of functional polyesters bearing ene and epoxide side chains

(2013) Yunfeng Yan et al.   Polymer Chemistry

Mutant KRAS is a druggable target for pancreatic cancer

(2013) E. Zorde Khvalevsky et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Multiparametric approach for the evaluation of lipid nanoparticles for siRNA delivery

(2013) C. A. Alabi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Enhancing tumor cell response to chemotherapy through nanoparticle-mediated codelivery of siRNA and cisplatin prodrug

(2013) X. Xu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Single-Step Assembly of Cationic Lipid–Polymer Hybrid Nanoparticles for Systemic Delivery of siRNA

(2012) Xian-Zhu Yang et al.   ACS Nano

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

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

2′-Fluoro RNA Shows Increased Watson-Crick H-Bonding Strength and Stacking Relative to RNA: Evidence from NMR and Thermodynamic Data

(2012) Amritraj Patra et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

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

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

Synthesis and characterization of novel zwitterionic lipids with pH-responsive biophysical properties

(2012) Colin L. Walsh et al.   CHEMICAL COMMUNICATIONS

How cationic lipids transfer nucleic acids into cells and across cellular membranes: Recent advances

(2012) Zia ur Rehman et al.   JOURNAL OF CONTROLLED RELEASE

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

RGD-Based Strategies To Target Alpha(v) Beta(3) Integrin in Cancer Therapy and Diagnosis

(2012) Fabienne Danhier et al.   MOLECULAR PHARMACEUTICS

Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin

(2012) Katalin Karikó et al.   MOLECULAR THERAPY

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

(2012) Benjamin Petsch et al.   NATURE BIOTECHNOLOGY

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

(2012) Ryszard Kole et al.   NATURE REVIEWS DRUG DISCOVERY

Nonviral delivery of self-amplifying RNA vaccines

(2012) A. J. Geall et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Defined Folate-PEG-siRNA Conjugates for Receptor-specific Gene Silencing

(2012) Christian Dohmen et al.   Molecular Therapy-Nucleic Acids

The Business of RNAi Therapeutics in 2012

(2012) Dirk Haussecker   Molecular Therapy-Nucleic Acids

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

miR-34 – a microRNA replacement therapy is headed to the clinic

(2012) Andreas G. Bader   Frontiers in Genetics

Differentially Charged Hollow Core/Shell Lipid-Polymer-Lipid Hybrid Nanoparticles for Small Interfering RNA Delivery

(2011) Jinjun Shi et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Multivalent Cyclic RGD Conjugates for Targeted Delivery of Small Interfering RNA

(2011) Md. Rowshon Alam et al.   BIOCONJUGATE CHEMISTRY

Systemic delivery of siRNA with cationic lipid assisted PEG-PLA nanoparticles for cancer therapy

(2011) Xian-Zhu Yang et al.   JOURNAL OF CONTROLLED RELEASE

In Vitroandin VivomRNA Delivery Using Lipid-Enveloped pH-Responsive Polymer Nanoparticles

(2011) Xingfang Su et al.   MOLECULAR PHARMACEUTICS

An Amino Acid-based Amphoteric Liposomal Delivery System for Systemic Administration of siRNA

(2011) Roger C Adami et al.   MOLECULAR THERAPY

MicroRNA history: Discovery, recent applications, and next frontiers

(2011) Maria I. Almeida et al.   MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS

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

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

Transfection Efficiency of Cationic Lipids with Different Hydrophobic Domains in Gene Delivery

(2010) DeFu Zhi et al.   BIOCONJUGATE CHEMISTRY

Atu027 Prevents Pulmonary Metastasis in Experimental and Spontaneous Mouse Metastasis Models

(2010) A. Santel et al.   CLINICAL CANCER RESEARCH

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

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

(2010) Mark E. Davis et al.   NATURE

Rapid translocation of nanoparticles from the lung airspaces to the body

(2010) Hak Soo Choi et al.   NATURE BIOTECHNOLOGY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs

(2010) Narendra Vaish et al.   NUCLEIC ACIDS RESEARCH

Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation

(2010) Bart R. Anderson et al.   NUCLEIC ACIDS RESEARCH

Lipid-like materials for low-dose, in vivo gene silencing

(2010) K. T. Love et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Novel Brush Polymers with Phosphorylcholine Bristle Ends: Synthesis, Structure, Properties, and Biocompatibility

(2009) Gahee Kim et al.   ADVANCED FUNCTIONAL MATERIALS

Knocking down barriers: advances in siRNA delivery

(2009) Kathryn A. Whitehead et al.   NATURE REVIEWS DRUG DISCOVERY

A Combinatorial Polymer Library Approach Yields Insight into Nonviral Gene Delivery

(2008) Jordan J. Green et al.   ACCOUNTS OF CHEMICAL RESEARCH

Efficient Gene Delivery Vectors by Tuning the Surface Charge Density of Amino Acid-Functionalized Gold Nanoparticles

(2008) Partha S. Ghosh et al.   ACS Nano

Atu027, a Liposomal Small Interfering RNA Formulation Targeting Protein Kinase N3, Inhibits Cancer Progression

(2008) M. Aleku et al.   CANCER RESEARCH

The Business of RNAi Therapeutics

(2008) Dirk Haussecker   HUMAN GENE THERAPY

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

(2008) Katalin Karikó et al.   MOLECULAR THERAPY

Resolution of liver cirrhosis using vitamin A–coupled liposomes to deliver siRNA against a collagen-specific chaperone

(2008) Yasushi Sato et al.   NATURE BIOTECHNOLOGY

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

(2008) Akin Akinc et al.   NATURE BIOTECHNOLOGY

Membrane lipids: where they are and how they behave

(2008) Gerrit van Meer et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Sorafenib in Liver Cancer — Just the Beginning

(2008) Lewis R. Roberts   NEW ENGLAND JOURNAL OF MEDICINE

Chemical Modification of siRNAs for In Vivo Use

(2008) Mark A. Behlke   OLIGONUCLEOTIDES

Synthesis and characterization of mRNA cap analogs containing phosphorothioate substitutions that bind tightly to eIF4E and are resistant to the decapping pyrophosphatase DcpS

(2008) J. Kowalska et al.   RNA

Functionalized Carbon Nanotubes in Drug Design and Discovery

(2007) Maurizio Prato et al.   ACCOUNTS OF CHEMICAL RESEARCH

Inorganic Nanoparticles as Carriers of Nucleic Acids into Cells

(2007) Viktoriya Sokolova et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION