Drug delivery systems for RNA therapeutics

Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles

(2022) Marine Z. C. Hatit et al.   Nature Nanotechnology

Safety, Pharmacokinetic and Pharmacodynamic Evaluation of a 2'-MOE Antisense Oligonucleotide-GalNAc3 Conjugate that Targets the Human Transmembrane Protease Serine 6 (TMPRSS6)

(2021) Thomas A Zanardi et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

A noninflammatory mRNA vaccine for treatment of experimental autoimmune encephalomyelitis

(2021) Christina Krienke et al.   SCIENCE

Ionizable lipid nanoparticles for in utero mRNA delivery

(2021) Rachel S. Riley et al.   Science Advances

How to redesign COVID vaccines so they protect against variants

(2021) Ewen Callaway et al.   NATURE

Treatment of influenza and SARS-CoV-2 infections via mRNA-encoded Cas13a in rodents

(2021) Emmeline L. Blanchard et al.   NATURE BIOTECHNOLOGY

Current understanding of biological identity at the nanoscale and future prospects

(2021) Kenneth A. Dawson et al.   Nature Nanotechnology

BNT162b2 mRNA Covid-19 Vaccine in a Nationwide Mass Vaccination Setting

(2021) Noa Dagan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Serum Neutralizing Activity Elicited by mRNA-1273 Vaccine — Preliminary Report

(2021) Kai Wu et al.   NEW ENGLAND JOURNAL OF MEDICINE

Dual modes of CRISPR-associated transposon homing

(2021) Makoto Saito et al.   CELL

Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing

(2021) James K. Nuñez et al.   CELL

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

(2021) Tanja Rothgangl et al.   NATURE BIOTECHNOLOGY

Initial report of decreased SARS-CoV-2 viral load after inoculation with the BNT162b2 vaccine

(2021) Matan Levine-Tiefenbrun et al.   NATURE MEDICINE

Therapeutically reprogrammed nutrient signalling enhances nanoparticulate albumin bound drug uptake and efficacy in KRAS-mutant cancer

(2021) Ran Li et al.   Nature Nanotechnology

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

(2021) Sander F. Garrelfs et al.   NEW ENGLAND JOURNAL OF MEDICINE

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

(2021) Donald B. Kohn et al.   NEW ENGLAND JOURNAL OF MEDICINE

Vaccine Breakthrough Infections with SARS-CoV-2 Variants

(2021) Ezgi Hacisuleyman et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of Serum Proteins on the Uptake and RNA Interference Activity of N-Acetylgalactosamine-Conjugated Small Interfering RNAs

(2021) Saket Agarwal et al.   Nucleic Acid Therapeutics

The evolution of commercial drug delivery technologies

(2021) Ava M. Vargason et al.   Nature Biomedical Engineering

From Antisense RNA to RNA Modification: Therapeutic Potential of RNA-Based Technologies

(2021) Hironori Adachi et al.   Biomedicines

Lipids and Lipid Derivatives for RNA Delivery

(2021) Yuebao Zhang et al.   CHEMICAL REVIEWS

Engineered miniature CRISPR-Cas system for mammalian genome regulation and editing

(2021) Xiaoshu Xu et al.   MOLECULAR CELL

Programmable RNA targeting with the single-protein CRISPR effector Cas7-11

(2021) Ahsen Özcan et al.   NATURE

Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus

(2021) Do Yon Kim et al.   NATURE BIOTECHNOLOGY

Compact RNA editors with small Cas13 proteins

(2021) Soumya Kannan et al.   NATURE BIOTECHNOLOGY

Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS

(2021) Tetsuya Nagata et al.   NATURE BIOTECHNOLOGY

Conformation-sensitive targeting of lipid nanoparticles for RNA therapeutics

(2021) Niels Dammes et al.   Nature Nanotechnology

Extracellular vesicles as a next-generation drug delivery platform

(2021) Inge Katrin Herrmann et al.   Nature Nanotechnology

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mammalian retrovirus-like protein PEG10 packages its own mRNA and can be pseudotyped for mRNA delivery

(2021) Michael Segel et al.   SCIENCE

Highly Potent GalNAc-Conjugated Tiny LNA Anti-miRNA-122 Antisense Oligonucleotides

(2021) Tsuyoshi Yamamoto et al.   Pharmaceutics

The Effect of Size and Charge of Lipid Nanoparticles Prepared by Microfluidic Mixing on Their Lymph Node Transitivity and Distribution

(2020) Takashi Nakamura et al.   MOLECULAR PHARMACEUTICS

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

(2020) Diane E. Frank et al.   NEUROLOGY

An RNA vaccine drives expansion and efficacy of claudin-CAR-T cells against solid tumors

(2020) Katharina Reinhard et al.   SCIENCE

Novel Engineered Programmable Systems for ADAR-Mediated RNA Editing

(2020) Guillermo Aquino-Jarquin   Molecular Therapy-Nucleic Acids

Optimization of an mRNA vaccine assisted with cyclodextrin-polyethyleneimine conjugates

(2020) Lu Tan et al.   Drug Delivery and Translational Research

Engineering Caveolae-Targeted Lipid Nanoparticles To Deliver mRNA to the Lungs

(2020) Qing Li et al.   ACS Chemical Biology

Phase 1 study of MRX34, a liposomal miR-34a mimic, in patients with advanced solid tumours

(2020) David S. Hong et al.   BRITISH JOURNAL OF CANCER

Development of CRISPR as an Antiviral Strategy to Combat SARS-CoV-2 and Influenza

(2020) Timothy R. Abbott et al.   CELL

Emerging siRNA Design Principles and Consequences for Biotransformation and Disposition in Drug Development

(2020) Sara C. Humphreys et al.   JOURNAL OF MEDICINAL CHEMISTRY

Deconvoluting Lipid Nanoparticle Structure for Messenger RNA Delivery

(2020) Yulia Eygeris et al.   NANO LETTERS

Challenges to optimizing RNA nanostructures for large scale production and controlled therapeutic properties

(2020) Morgan Chandler et al.   Nanomedicine

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

(2020) Christian Leborgne et al.   NATURE MEDICINE

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

(2020) Qiang Cheng et al.   Nature Nanotechnology

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

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

Recent Advances in Understanding the Protein Corona of Nanoparticles and in the Formulation of “Stealthy” Nanomaterials

(2020) Riccardo Rampado et al.   Frontiers in Bioengineering and Biotechnology

mRNA Vaccines Encoding the HA Protein of Influenza A H1N1 Virus Delivered by Cationic Lipid Nanoparticles Induce Protective Immune Responses in Mice

(2020) Xinyu Zhuang et al.   Vaccines

Surface-Functionalized PEGylated Nanoparticles Deliver Messenger RNA to Pulmonary Immune Cells

(2020) Xiyu Ke et al.   ACS Applied Materials & Interfaces

Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

(2020) Bahru A. Habtemariam et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

A Single Immunization with Nucleoside-Modified mRNA Vaccines Elicits Strong Cellular and Humoral Immune Responses against SARS-CoV-2 in Mice

(2020) Dorottya Laczkó et al.   IMMUNITY

A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing

(2020) Beverly Y. Mok et al.   NATURE

An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma

(2020) Ugur Sahin et al.   NATURE

Advances in oligonucleotide drug delivery

(2020) Thomas C. Roberts et al.   NATURE REVIEWS DRUG DISCOVERY

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

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

Increased PIP3 activity blocks nanoparticle mRNA delivery

(2020) Kalina Paunovska et al.   Science Advances

Development of a potent Zika virus vaccine using self-amplifying messenger RNA

(2020) Kate Luisi et al.   Science Advances

Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

(2020) Karishma Dhuri et al.   Journal of Clinical Medicine

Long-term storage of lipid-like nanoparticles for mRNA delivery

(2020) Pengxuan Zhao et al.   Bioactive Materials

Treating Cystic Fibrosis with mRNA and CRISPR

(2020) Alejandro Da Silva Sanchez et al.   HUMAN GENE THERAPY

Base editing: advances and therapeutic opportunities

(2020) Elizabeth M. Porto et al.   NATURE REVIEWS DRUG DISCOVERY

Cell Type Impacts Accessibility of mRNA to Silencing by RNA Interference

(2020) Chantal M. Ferguson et al.   Molecular Therapy-Nucleic Acids

Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing

(2020) Xinfu Zhang et al.   Science Advances

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

(2020) Giang N. Nguyen et al.   NATURE BIOTECHNOLOGY

Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine

(2020) Lindsey R. Baden et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

(2020) Fernando P. Polack et al.   NEW ENGLAND JOURNAL OF MEDICINE

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

(2020) Erica B. Esrick et al.   NEW ENGLAND JOURNAL OF MEDICINE

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Precise RNA editing by recruiting endogenous ADARs with antisense oligonucleotides

(2019) Tobias Merkle et al.   NATURE BIOTECHNOLOGY

Genome Editing with mRNA Encoding ZFN, TALEN, and Cas9

(2019) Hong-Xia Zhang et al.   MOLECULAR THERAPY

Nanoparticles Containing Oxidized Cholesterol Deliver mRNA to the Liver Microenvironment at Clinically Relevant Doses

(2019) Kalina Paunovska et al.   ADVANCED MATERIALS

Lipid-Based DNA Therapeutics – Hallmarks of Non-Viral Gene Delivery

(2019) Jonas Buck et al.   ACS Nano

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

(2019) Wen Shen et al.   NATURE BIOTECHNOLOGY

mRNA vaccines against H10N8 and H7N9 influenza viruses of pandemic potential are immunogenic and well tolerated in healthy adults in phase 1 randomized clinical trials

(2019) Robert A. Feldman et al.   VACCINE

Polymeric Nanoparticles in Gene Therapy: New Avenues of Design and Optimization for Delivery Applications

(2019) Raj Rai et al.   Polymers

The Potential for microRNA Therapeutics and Clinical Research

(2019) Johora Hanna et al.   Frontiers in Genetics

Active Targeting Strategies Using Biological Ligands for Nanoparticle Drug Delivery Systems

(2019) Jihye Yoo et al.   Cancers

Visualization of early events in mRNA vaccine delivery in non-human primates via PET–CT and near-infrared imaging

(2019) Kevin E. Lindsay et al.   Nature Biomedical Engineering

Cancer-selective nanoparticles for combinatorial siRNA delivery to primary human GBM in vitro and in vivo

(2019) Kristen L. Kozielski et al.   BIOMATERIALS

Pharmacokinetics and Clinical Pharmacology Considerations of GalNAc3-Conjugated Antisense Oligonucleotides

(2019) Yanfeng Wang et al.   Expert Opinion on Drug Metabolism & Toxicology

Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure‐Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin‐Mediated (hATTR) Amyloidosis

(2019) Xiaoping Zhang et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs

(2019) Liang Qu et al.   NATURE BIOTECHNOLOGY

Gene Therapy

(2019) Katherine A. High et al.   NEW ENGLAND JOURNAL OF MEDICINE

A cytosine deaminase for programmable single-base RNA editing

(2019) Omar O. Abudayyeh et al.   SCIENCE

Constrained Nanoparticles Deliver siRNA and sgRNA to T Cells In Vivo without Targeting Ligands

(2019) Melissa P. Lokugamage et al.   ADVANCED MATERIALS

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler–Najjar Syndrome

(2019) Sem J. Aronson et al.   HUMAN GENE THERAPY

Mild Innate Immune Activation Overrides Efficient Nanoparticle‐Mediated RNA Delivery

(2019) Melissa P. Lokugamage et al.   ADVANCED MATERIALS

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

Nonviral polymeric nanoparticles for gene therapy in pediatric CNS malignancies

(2019) John Choi et al.   Nanomedicine-Nanotechnology Biology and Medicine

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

(2019) Akin Akinc et al.   Nature Nanotechnology

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

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

Analyzing 2000 in Vivo Drug Delivery Data Points Reveals Cholesterol Structure Impacts Nanoparticle Delivery

(2018) Kalina Paunovska et al.   ACS Nano

Nucleoside-modified mRNA vaccines induce potent T follicular helper and germinal center B cell responses

(2018) Norbert Pardi et al.   JOURNAL OF EXPERIMENTAL MEDICINE

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

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

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

A modular platform for targeted RNAi therapeutics

(2018) Ranit Kedmi et al.   Nature Nanotechnology

mRNA vaccines — a new era in vaccinology

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

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

(2018) Jayesh A. Kulkarni et al.   Nucleic Acid Therapeutics

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

Endothelial siRNA delivery in nonhuman primates using ionizable low–molecular weight polymeric nanoparticles

(2018) Omar F. Khan et al.   Science Advances

Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

(2018) Bumwhee Lee et al.   Nature Biomedical Engineering

Modifying a Commonly Expressed Endocytic Receptor Retargets Nanoparticles in Vivo

(2018) Cory D. Sago et al.   NANO LETTERS

The Nonclinical Safety Profile of GalNAc-conjugated RNAi Therapeutics in Subacute Studies

(2018) Maja M. Janas et al.   TOXICOLOGIC PATHOLOGY

MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct

(2018) Ruchi Jain et al.   Nucleic Acid Therapeutics

Phase I clinical trial of an intranodally administered mRNA based therapeutic vaccine against HIV-1 infection

(2018) Lorna Leal et al.   AIDS

Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution

(2018) Cory D. Sago et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Improving mRNA-Based Therapeutic Gene Delivery by Expression-Augmenting 3′ UTRs Identified by Cellular Library Screening

(2018) Alexandra G. Orlandini von Niessen et al.   MOLECULAR THERAPY

Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways

(2018) Maire F Osborn et al.   NUCLEIC ACIDS RESEARCH

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo

(2018) Annabelle Biscans et al.   NUCLEIC ACIDS RESEARCH

High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing

(2018) Cory D. Sago et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

(2018) Nuphar Veiga et al.   Nature Communications

A Comprehensive Map of FDA-Approved Pharmaceutical Products

(2018) Hao Zhong et al.   Pharmaceutics

An RNA Aptamer Targeting the Receptor Tyrosine Kinase PDGFRα Induces Anti-tumor Effects through STAT3 and p53 in Glioblastoma

(2018) Sorah Yoon et al.   Molecular Therapy-Nucleic Acids

Use and Importance of Nonhuman Primates in Metabolic Disease Research: Current State of the Field

(2017) Peter J Havel et al.   ILAR JOURNAL

Safety and immunogenicity of a mRNA rabies vaccine in healthy adults: an open-label, non-randomised, prospective, first-in-human phase 1 clinical trial

(2017) Martin Alberer et al.   LANCET

Safety, tolerability, and antiviral effect of RG-101 in patients with chronic hepatitis C: a phase 1B, double-blind, randomised controlled trial

(2017) Meike H van der Ree et al.   LANCET

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Polymeric nanoparticles: A study on the preparation variables and characterization methods

(2017) Carina I.C. Crucho et al.   Materials Science & Engineering C-Materials for Biological Applications

Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA

(2017) Siddharth Patel et al.   NANO LETTERS

Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer

(2017) Ugur Sahin et al.   NATURE

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

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease

(2017) Marc S. Sabatine et al.   NEW ENGLAND JOURNAL OF MEDICINE

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

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

Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics

(2017) James E. Dahlman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

RNA editing with CRISPR-Cas13

(2017) David B. T. Cox et al.   SCIENCE

Safety Evaluation of Lipid Nanoparticle–Formulated Modified mRNA in the Sprague-Dawley Rat and Cynomolgus Monkey

(2017) Maja Sedic et al.   VETERINARY PATHOLOGY

Eteplirsen in the treatment of Duchenne muscular dystrophy

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

Molecular Mechanisms of Antisense Oligonucleotides

(2017) Stanley T. Crooke   Nucleic Acid Therapeutics

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

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

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

PEGylation as a strategy for improving nanoparticle-based drug and gene delivery

(2016) Jung Soo Suk et al.   ADVANCED DRUG DELIVERY REVIEWS

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

Influenza H3N2 infection of the collaborative cross founder strains reveals highly divergent host responses and identifies a unique phenotype in CAST/EiJ mice

(2016) Sarah R. Leist et al.   BMC GENOMICS

Degradable Controlled-Release Polymers and Polymeric Nanoparticles: Mechanisms of Controlling Drug Release

(2016) Nazila Kamaly et al.   CHEMICAL REVIEWS

Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles

(2016) Tsukasa Sugo et al.   JOURNAL OF CONTROLLED RELEASE

Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA

(2016) Sam Chen et al.   JOURNAL OF CONTROLLED RELEASE

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

(2016) Lena M. Kranz et al.   NATURE

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

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

(2016) Pratiksha I Thakore et al.   NATURE METHODS

Protein adsorption is required for stealth effect of poly(ethylene glycol)- and poly(phosphoester)-coated nanocarriers

(2016) Susanne Schöttler et al.   Nature Nanotechnology

Aptamers as targeted therapeutics: current potential and challenges

(2016) Jiehua Zhou et al.   NATURE REVIEWS DRUG DISCOVERY

Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver

(2016) Colton M. Miller et al.   NUCLEIC ACIDS RESEARCH

Dendrimer-RNA nanoparticles generate protective immunity against lethal Ebola, H1N1 influenza, andToxoplasma gondiichallenges with a single dose

(2016) Jasdave S. Chahal et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector

(2016) Omar O. Abudayyeh et al.   SCIENCE

Global Patterns of Zoonotic Disease in Mammals

(2016) Barbara A. Han et al.   TRENDS IN PARASITOLOGY

Theranostic barcoded nanoparticles for personalized cancer medicine

(2016) Zvi Yaari et al.   Nature Communications

Combination Therapy for Ulcerative Colitis: Orally Targeted Nanoparticles Prevent Mucosal Damage and Relieve Inflammation

(2016) Bo Xiao et al.   Theranostics

Optimized polyethylenimine (PEI)-based nanoparticles for siRNA delivery, analyzed in vitro and in an ex vivo tumor tissue slice culture model

(2016) Alexander Ewe et al.   Drug Delivery and Translational Research

Antisense oligonucleotides in therapy for neurodegenerative disorders

(2015) Melvin M. Evers et al.   ADVANCED DRUG DELIVERY REVIEWS

Miravirsen dosing in chronic hepatitis C patients results in decreased microRNA-122 levels without affecting other microRNAs in plasma

(2015) M. H. van der Ree et al.   ALIMENTARY PHARMACOLOGY & THERAPEUTICS

Normal Organ Weights in Women

(2015) D. Kimberley Molina et al.   AMERICAN JOURNAL OF FORENSIC MEDICINE AND PATHOLOGY

Normal Organ Weights in Women

(2015) D. Kimberley Molina et al.   AMERICAN JOURNAL OF FORENSIC MEDICINE AND PATHOLOGY

Mipomersen, an Antisense Oligonucleotide to Apolipoprotein B-100, Reduces Lipoprotein(a) in Various Populations With HypercholesterolemiaSignificance

(2015) Raul D. Santos et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

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

(2015) Bernd Zetsche et al.   CELL

Systems genetic analysis of hippocampal neuroanatomy and spatial learning in mice

(2015) A. Delprato et al.   GENES BRAIN AND BEHAVIOR

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

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

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

Combinatorial library strategies for synthesis of cationic lipid-like nanoparticles and their potential medical applications

(2015) Sarah Altınoglu et al.   Nanomedicine

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

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

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

A holistic approach to targeting disease with polymeric nanoparticles

(2015) Christopher J. Cheng et al.   NATURE REVIEWS DRUG DISCOVERY

Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy

(2015) Panagiotis Mastorakos et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Ionizable Amphiphilic Dendrimer-Based Nanomaterials with Alkyl-Chain-Substituted Amines for Tunable siRNA Delivery to the Liver Endothelium In Vivo

(2014) Omar F. Khan et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

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

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

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

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Sequence motifs associated with hepatotoxicity of locked nucleic acid—modified antisense oligonucleotides

(2014) Andrew D. Burdick et al.   NUCLEIC ACIDS RESEARCH

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

(2014) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

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

A Systems Biology Approach Utilizing a Mouse Diversity Panel Identifies Genetic Differences Influencing Isoniazid-Induced Microvesicular Steatosis

(2014) Rachel J. Church et al.   TOXICOLOGICAL SCIENCES

Dendrimer Advances for the Central Nervous System Delivery of Therapeutics

(2013) Leyuan Xu et al.   ACS Chemical Neuroscience

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

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

Molecular Mechanisms of RNA Interference

(2013) Ross C. Wilson et al.   Annual Review of Biophysics

CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes

(2013) Luke A. Gilbert et al.   CELL

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

Transferrin-functionalized nanoparticles lose their targeting capabilities when a biomolecule corona adsorbs on the surface

(2013) Anna Salvati et al.   Nature Nanotechnology

Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals

(2013) Gene Hung et al.   Nucleic Acid Therapeutics

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

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

A murine lung cancer co-clinical trial identifies genetic modifiers of therapeutic response

(2012) Zhao Chen et al.   NATURE

Multifunctional Nanoparticles: Cost Versus Benefit of Adding Targeting and Imaging Capabilities

(2012) Z. Cheng et al.   SCIENCE

Preclinical Development and Clinical Translation of a PSMA-Targeted Docetaxel Nanoparticle with a Differentiated Pharmacological Profile

(2012) J. Hrkach et al.   Science Translational Medicine

A Mouse Diversity Panel Approach Reveals the Potential for Clinical Kidney Injury Due to DB289 Not Predicted by Classical Rodent Models

(2012) Alison H. Harrill et al.   TOXICOLOGICAL SCIENCES

Normal Organ Weights in Men

(2011) D. Kimberley Molina et al.   AMERICAN JOURNAL OF FORENSIC MEDICINE AND PATHOLOGY

Normal Organ Weights in Men

(2011) D. Kimberley Molina et al.   AMERICAN JOURNAL OF FORENSIC MEDICINE AND PATHOLOGY

The association of autophagy with polyethylenimine-induced cytotoxity in nephritic and hepatic cell lines

(2011) Xiaoling Gao et al.   BIOMATERIALS

Plasma lipid profiling across species for the identification of optimal animal models of human dyslipidemia

(2011) Wu Yin et al.   JOURNAL OF LIPID RESEARCH

Silencing disease genes in the laboratory and the clinic

(2011) Jonathan K Watts et al.   JOURNAL OF PATHOLOGY

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

(2011) Xingfang Su et al.   MOLECULAR PHARMACEUTICS

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

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

Effect of Genome Size on AAV Vector Packaging

(2009) Zhijian Wu et al.   MOLECULAR THERAPY

Understanding biophysicochemical interactions at the nano–bio interface

(2009) Andre E. Nel et al.   NATURE MATERIALS

A Combinatorial Polymer Library Approach Yields Insight into Nonviral Gene Delivery

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

Prolonged gene silencing in hepatoma cells and primary hepatocytes after small interfering RNA delivery with biodegradable poly(β‐amino esters)

(2008) Roosmarijn E. Vandenbroucke et al.   JOURNAL OF GENE MEDICINE