Recent advances in lipid nanoparticles for delivery of nucleic acid, mRNA, and gene editing-based therapeutics

Ionizable lipid nanoparticles for in utero mRNA delivery

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

In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

(2021) Lukas Villiger et al.   Nature Biomedical Engineering

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

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

Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

(2021) Min Qiu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Engineered ionizable lipid nanoparticles for targeted delivery of RNA therapeutics into different types of cells in the liver

(2021) M. Kim et al.   Science Advances

Apolipoprotein E Binding Drives Structural and Compositional Rearrangement of mRNA-Containing Lipid Nanoparticles

(2021) Federica Sebastiani et al.   ACS Nano

mRNA Delivery of a Bispecific Single‐Domain Antibody to Polarize Tumor‐Associated Macrophages and Synergize Immunotherapy against Liver Malignancies

(2021) Ying Wang et al.   ADVANCED MATERIALS

Novel approaches for vaccine development

(2021) Makda S. Gebre et al.   CELL

mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability

(2021) Linde Schoenmaker et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

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

All‐In‐One Dendrimer‐Based Lipid Nanoparticles Enable Precise HDR‐Mediated Gene Editing In Vivo

(2021) Lukas Farbiak et al.   ADVANCED MATERIALS

Conformation-sensitive targeting of lipid nanoparticles for RNA therapeutics

(2021) Niels Dammes et al.   Nature Nanotechnology

The Importance of Apparent pKa in the Development of Nanoparticles Encapsulating siRNA and mRNA

(2021) Pratikkumar Patel et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Different kinetics for the hepatic uptake of lipid nanoparticles between the apolipoprotein E/low density lipoprotein receptor and the N-acetyl-d-galactosamine/asialoglycoprotein receptor pathway

(2020) Yusuke Sato et al.   JOURNAL OF CONTROLLED RELEASE

ImmunoPET: Concept, Design, and Applications

(2020) Weijun Wei et al.   CHEMICAL REVIEWS

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

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

(2020) Qiang Cheng et al.   Nature Nanotechnology

Development of lipid-like materials for RNA delivery based on intracellular environment-responsive membrane destabilization and spontaneous collapse

(2020) Hiroki Tanaka et al.   ADVANCED DRUG DELIVERY REVIEWS

Lipid nanoparticles for nucleic acid delivery: Current perspectives

(2020) Eleni Samaridou et al.   ADVANCED DRUG DELIVERY REVIEWS

Lipid nanoparticle technology for therapeutic gene regulation in the liver

(2020) Dominik Witzigmann et al.   ADVANCED DRUG DELIVERY REVIEWS

The Biomolecular Corona of Lipid Nanoparticles for Gene Therapy

(2020) Valentina Francia et al.   BIOCONJUGATE CHEMISTRY

A Thermostable mRNA Vaccine against COVID-19

(2020) Na-Na Zhang et al.   CELL

The delivery challenge: fulfilling the promise of therapeutic genome editing

(2020) Joost van Haasteren et al.   NATURE BIOTECHNOLOGY

COVID-19 vaccine development and a potential nanomaterial path forward

(2020) Matthew D. Shin et al.   Nature Nanotechnology

Review: PET imaging with macro- and middle-sized molecular probes

(2020) Hidefumi Mukai et al.   NUCLEAR MEDICINE AND BIOLOGY

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Increased PIP3 activity blocks nanoparticle mRNA delivery

(2020) Kalina Paunovska et al.   Science Advances

Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection

(2020) Feihe Ma et al.   Science Advances

Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia

(2020) Lei Jiang et al.   Nature Communications

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

Durable anticancer immunity from intratumoral administration of IL-23, IL-36γ, and OX40L mRNAs

(2019) Susannah L. Hewitt et al.   Science Translational Medicine

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

(2019) Piotr S. Kowalski et al.   MOLECULAR THERAPY

MRNA Delivery For Therapeutic Anti-Her2 Antibody Expression In Vivo

(2019) Yulia Rybakova et al.   MOLECULAR THERAPY

Macrocyclic peptide-based inhibition and imaging of hepatocyte growth factor

(2019) Katsuya Sakai et al.   Nature Chemical Biology

Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines

(2019) Kimberly J. Hassett et al.   Molecular Therapy-Nucleic Acids

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

Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

(2019) Ji Liu et al.   ADVANCED MATERIALS

Mannosylation of LNP Results in Improved Potency for Self-Amplifying RNA (SAM) Vaccines

(2019) Roshan Goswami et al.   ACS Infectious Diseases

A lipid-encapsulated mRNA encoding a potently neutralizing human monoclonal antibody protects against chikungunya infection

(2019) Nurgun Kose et al.   Science Immunology

Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics

(2019) Jayesh A. Kulkarni et al.   ACCOUNTS OF CHEMICAL RESEARCH

Rational designs of in vivo CRISPR-Cas delivery systems

(2019) Cong-Fei Xu et al.   ADVANCED DRUG DELIVERY REVIEWS

Mild Innate Immune Activation Overrides Efficient Nanoparticle‐Mediated RNA Delivery

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

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening

(2019) Pedro P.G. Guimaraes et al.   JOURNAL OF CONTROLLED RELEASE

Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency

(2019) David N. Nguyen et al.   NATURE BIOTECHNOLOGY

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

(2019) Akin Akinc et al.   Nature Nanotechnology

Advances in microfluidics for lipid nanoparticles and extracellular vesicles and applications in drug delivery systems

(2018) Masatoshi Maeki et al.   ADVANCED DRUG DELIVERY REVIEWS

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

Structure of Lipid Nanoparticles Containing siRNA or mRNA by Dynamic Nuclear Polarization-Enhanced NMR Spectroscopy

(2018) Jasmine Viger-Gravel et al.   JOURNAL OF PHYSICAL CHEMISTRY B

A modular platform for targeted RNAi therapeutics

(2018) Ranit Kedmi et al.   Nature Nanotechnology

Successful reprogramming of cellular protein production through mRNA delivered by functionalized lipid nanoparticles

(2018) Marianna Yanez Arteta et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Single-cell bioluminescence imaging of deep tissue in freely moving animals

(2018) Satoshi Iwano et al.   SCIENCE

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

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

Modifying a Commonly Expressed Endocytic Receptor Retargets Nanoparticles in Vivo

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

CRISPR-Cas guides the future of genetic engineering

(2018) Gavin J. Knott et al.   SCIENCE

Pharmacokinetic evaluation of liposomal nanoparticle-encapsulated nucleic acid drug: A combined study of dynamic PET imaging and LC/MS/MS analysis

(2018) Hidefumi Mukai et al.   JOURNAL OF CONTROLLED RELEASE

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

(2018) Lukas Villiger et al.   NATURE MEDICINE

Base editing: precision chemistry on the genome and transcriptome of living cells

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

(2018) Nuphar Veiga et al.   Nature Communications

Delivery strategies of the CRISPR-Cas9 gene-editing system for therapeutic applications

(2017) Chang Liu et al.   JOURNAL OF CONTROLLED RELEASE

Lipid Nanoparticle Systems for Enabling Gene Therapies

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

RNA targeting with CRISPR–Cas13

(2017) Omar O. Abudayyeh et al.   NATURE

Delivery technologies for genome editing

(2017) Hao Yin et al.   NATURE REVIEWS DRUG DISCOVERY

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

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

Nucleic acid detection with CRISPR-Cas13a/C2c2

(2017) Jonathan S. Gootenberg et al.   SCIENCE

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

(2017) Norbert Pardi et al.   Nature Communications

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

(2016) Alexis C. Komor 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

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

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

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

(2016) Omar O. Abudayyeh et al.   SCIENCE

A luciferin analogue generating near-infrared bioluminescence achieves highly sensitive deep-tissue imaging

(2016) Takahiro Kuchimaru et al.   Nature Communications

Fc-Binding Ligands of Immunoglobulin G: An Overview of High Affinity Proteins and Peptides

(2016) Weonu Choe et al.   Materials

Systemic Gene Silencing in Primary T Lymphocytes Using Targeted Lipid Nanoparticles

(2015) Srinivas Ramishetti et al.   ACS Nano

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

Selection-Based Discovery of Druglike Macrocyclic Peptides

(2014) Toby Passioura et al.   Annual Review of Biochemistry

Glycosylation-mediated targeting of carriers

(2014) Shigeru Kawakami et al.   JOURNAL OF CONTROLLED RELEASE

Quantitative evaluation of the improvement in the pharmacokinetics of a nucleic acid drug delivery system by dynamic PET imaging with 18F-incorporated oligodeoxynucleotides

(2014) Hidefumi Mukai et al.   JOURNAL OF CONTROLLED RELEASE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Genetic Approaches to Neural Circuits in the Mouse

(2013) Z. Josh Huang et al.   Annual Review of Neuroscience

The Polyethyleneglycol Dilemma: Advantage and Disadvantage of PEGylation of Liposomes for Systemic Genes and Nucleic Acids Delivery to Tumors

(2013) Hiroto Hatakeyama et al.   BIOLOGICAL & PHARMACEUTICAL BULLETIN

mRNA display: from basic principles to macrocycle drug discovery

(2013) Kristopher Josephson et al.   DRUG DISCOVERY TODAY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

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

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

A Neutral Envelope-Type Nanoparticle Containing pH-Responsive and SS-Cleavable Lipid-Like Material as a Carrier for Plasmid DNA

(2013) Hidetaka Akita et al.   Advanced Healthcare Materials

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

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

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

A review of glycosylated carriers for drug delivery

(2012) Keerti Jain et al.   BIOMATERIALS

Lineage Tracing

(2012) Kai Kretzschmar et al.   CELL

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

Bottom-Up Design and Synthesis of Limit Size Lipid Nanoparticle Systems with Aqueous and Triglyceride Cores Using Millisecond Microfluidic Mixing

(2012) Igor V. Zhigaltsev et al.   LANGMUIR

Nuclear ribosomal internal transcribed spacer (ITS) region as a universal DNA barcode marker for Fungi

(2012) C. L. Schoch et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

High-Throughput Droplet Digital PCR System for Absolute Quantitation of DNA Copy Number

(2011) Benjamin J. Hindson et al.   ANALYTICAL CHEMISTRY

PET Imaging of the Gastrointestinal Absorption of Orally Administered Drugs in Conscious and Anesthetized Rats

(2011) S. Yamashita et al.   JOURNAL OF NUCLEAR MEDICINE

Developmental Changes in P-Glycoprotein Function in the Blood-Brain Barrier of Nonhuman Primates: PET Study with R-11C-Verapamil and 11C-Oseltamivir

(2011) T. Takashima et al.   JOURNAL OF NUCLEAR MEDICINE

Therapeutic siRNA silencing in inflammatory monocytes in mice

(2011) Florian Leuschner et al.   NATURE BIOTECHNOLOGY

Impact of microdosing clinical study — Why necessary and how useful?

(2010) Yuichi Sugiyama et al.   ADVANCED DRUG DELIVERY REVIEWS

A multifunctional envelope type nano device (MEND) for gene delivery to tumours based on the EPR effect: A strategy for overcoming the PEG dilemma

(2010) Hiroto Hatakeyama et al.   ADVANCED DRUG DELIVERY REVIEWS

Stoichiometry-focused 18F-labeling of alkyne-substituted oligodeoxynucleotides using azido([18F]fluoromethyl)benzenes by Cu-catalyzed Huisgen reaction

(2010) Takeshi Kuboyama et al.   BIOORGANIC & MEDICINAL CHEMISTRY

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

A robust and high-throughput Cre reporting and characterization system for the whole mouse brain

(2009) Linda Madisen et al.   NATURE NEUROSCIENCE

Pharmacokinetics and Biodistribution of Nanoparticles

(2008) Shyh-Dar Li et al.   MOLECULAR PHARMACEUTICS

Nonviral approaches for targeted delivery of plasmid DNA and oligonucleotide

(2007) Shigeru Kawakami et al.   JOURNAL OF PHARMACEUTICAL SCIENCES