4.8 Review

Chemistry of Lipid Nanoparticles for RNA Delivery Published as part of the Accounts of Chemical Research special issue mRNA Therapeutics

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Biology

Ionization and structural properties of mRNA lipid nanoparticles influence expression in intramuscular and intravascular administration

Manuel J. Carrasco et al.

Summary: The study reports a method that can predict the pKa of lipid nanoparticles (LNPs) from the structure of the ionizable lipid, and investigates the delivery efficiency and factors influencing LNPs both in vitro and in vivo. It was also found that LNPs with more negative charge lead to higher off-target systemic mRNA expression.

COMMUNICATIONS BIOLOGY (2021)

Review Immunology

Nanomaterial Delivery Systems for mRNA Vaccines

Michael D. Buschmann et al.

Summary: This review highlights the recent success of mRNA vaccines in SARS-CoV-2 clinical trials, emphasizing the crucial role of lipid nanoparticles in the delivery systems and providing an analysis of the determinants of lipid nanoparticles' performance in mRNA vaccines.

VACCINES (2021)

Review Pharmacology & Pharmacy

Self-assembled mRNA vaccines

Jeonghwan Kim et al.

Summary: mRNA vaccines, emerging as front-runners in the COVID-19 vaccine race, utilize advancements in RNA technology, vaccinology, and nanotechnology to design self-assembled nanoparticles for vaccine delivery, showing tremendous promise and readiness for clinical use.

ADVANCED DRUG DELIVERY REVIEWS (2021)

Review Biochemistry & Molecular Biology

Cationic lipids for gene delivery: many players, one goal

Federica Ponti et al.

Summary: Lipid-based carriers are widely used as non-viral vectors for gene expression and gene silencing, due to their ease of synthesis and modification. While significant progress has been made in developing cationic lipids, further research is needed for more effective lipid vectors for gene transfection. This review emphasizes the importance of designing each constituent domain of cationic lipids and how it affects their performance.

CHEMISTRY AND PHYSICS OF LIPIDS (2021)

Article Chemistry, Physical

Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR-Cas gene editing

Shuai Liu et al.

Summary: The study successfully delivered mRNA or single-guide RNA for gene editing in vivo using combinatorially synthesized ionizable phospholipids. Optimized iPhos lipids can change shapes in endosomal acidic environments to facilitate the release of gene editing materials. When working synergistically with helper lipids, iPhos lipids can achieve targeted delivery to specific organs.

NATURE MATERIALS (2021)

Article Multidisciplinary Sciences

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

M. Kim et al.

Summary: Engineered LNPs have been developed for targeted delivery of RNA to hepatocytes and LSECs, showing potential for cell type-specific delivery of RNA to the liver and other tissues.

SCIENCE ADVANCES (2021)

Review Biotechnology & Applied Microbiology

Cytosolic delivery of nucleic acids: The case of ionizable lipid nanoparticles

Michele Schlich et al.

Summary: Ionizable lipid nanoparticles (LNPs) are the most advanced nano-delivery system for therapeutic nucleic acids, with the development of ionizable lipids playing a key role in their success. Overcoming challenges in RNA delivery, such as endosomal escape, is crucial for ensuring the therapeutic activity of RNA molecules. Understanding the mechanisms of endosomal escape and improving cytosolic delivery are important for developing future LNPs with enhanced nucleic acid delivery capabilities.

BIOENGINEERING & TRANSLATIONAL MEDICINE (2021)

Article Chemistry, Multidisciplinary

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

Federica Sebastiani et al.

Summary: This study investigated the structure and lipid distribution of LNPs in the presence of ApoE using small-angle neutron scattering. The binding of ApoE was found to cause a redistribution of lipids at the shell and core of LNPs, impacting their internal structure and mRNA release. The rearrangement of LNP components upon ApoE incubation may have implications for LNP endosomal escape.

ACS NANO (2021)

Article Biophysics

Encapsulation state of messenger RNA inside lipid nanoparticles

Mark L. Brader et al.

Summary: Understanding the structure and microenvironment of mRNA within lipid nanoparticles is crucial for unlocking their biomedical potential. By using the permeating cationic dye thionine as a contrasting agent, cryo-electron microscopy images reveal the location of mRNA within the nanoparticles, suggesting it may be in solvent-filled cavities or associated with lipids.

BIOPHYSICAL JOURNAL (2021)

Article Pharmacology & Pharmacy

Automated high-throughput preparation and characterization of oligonucleotide-loaded lipid nanoparticles

Yuchen Fan et al.

Summary: This study developed an automated high-throughput screening workflow for preparation and analysis of ASO-loaded LNPs, showing that PEGylated lipid content significantly affected particle size distribution, and ionizable lipid/ASO charge ratio impacted ASO encapsulation efficiency. Results from this HTS approach were consistent with those from a state-of-the-art scale-up method, indicating a new avenue for formulation development and design of experiment methods with reduced material usage and improved analytical outputs.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2021)

Article Chemistry, Multidisciplinary

Impact of lipid nanoparticle size on mRNA vaccine immunogenicity

Kimberly J. Hassett et al.

Summary: The size of LNP particles was found to affect the immunogenicity of vaccines in mice, with smaller diameter LNPs being less immunogenic; however, all particle sizes tested yielded robust immune responses in non-human primates (NHP).

JOURNAL OF CONTROLLED RELEASE (2021)

Article Chemistry, Multidisciplinary

Therapeutic Gene Silencing Using Targeted Lipid Nanoparticles in Metastatic Ovarian Cancer

Manu Smriti Singh et al.

Summary: LNPs encapsulating siRNAs demonstrate efficient tumor targeting and gene silencing in ovarian cancer cells, showing promising potential for the treatment of advanced ovarian cancer.
Article Biochemistry & Molecular Biology

Acidic pH-induced changes in lipid nanoparticle membrane packing

Kyoka Koitabashi et al.

Summary: The study investigated the changes in LNPs containing ionizable cationic lipids under acidic pH conditions. It was found that the membrane packing decreased with a decrease in pH value, and the pH-induced variation increased with an increasing percentage of ionizable cationic lipids in the LNPs. These results demonstrate the importance of membrane structure in the fusion of LNPs with endosome membranes.

BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES (2021)

Review Chemistry, Multidisciplinary

Lipids and Lipid Derivatives for RNA Delivery

Yuebao Zhang et al.

Summary: RNA-based therapeutics have shown great promise in treating diseases through various mechanisms, with lipid nanoparticles being the most widely used delivery system. The study of lipids, lipid derivatives, and lipid-derived macromolecules for RNA delivery has also made significant progress in recent years.

CHEMICAL REVIEWS (2021)

Article Chemistry, Multidisciplinary

Scalable mRNA and siRNA Lipid Nanoparticle Production Using a Parallelized Microfluidic Device

Sarah J. Shepherd et al.

Summary: The study presents a scalable, parallelized microfluidic device (PMD) that can simultaneously operate 128 mixing channels, achieving over 100 times production rate compared to traditional methods for lipid nanoparticles (LNPs). Results demonstrate superior delivery of LNPS generated using PMD in mice, showing potential for emerging clinical applications such as RNA therapeutics and vaccines.

NANO LETTERS (2021)

Review Nanoscience & Nanotechnology

Lipid nanoparticles for mRNA delivery

Xucheng Hou et al.

Summary: Lipid nanoparticle-mRNA formulations have successfully entered clinical use as COVID-19 vaccines, representing a major milestone in mRNA therapeutics. This review discusses the design of lipid nanoparticles for mRNA delivery, emphasizing key points for clinical translation and preclinical studies of lipid nanoparticle-mRNA therapeutics for various diseases.

NATURE REVIEWS MATERIALS (2021)

Article Engineering, Biomedical

Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs

Melissa P. Lokugamage et al.

Summary: This study optimized the design of lipid nanoparticles for the efficient delivery of therapeutic mRNA to the lungs via nebulization, resulting in protection of mice from a lethal challenge of H1N1 influenza A virus and more efficient delivery of mRNA compared to LNPs previously optimized for systemic delivery. The cluster approach to LNP design may facilitate the optimization of LNPs for other administration routes and therapeutics.

NATURE BIOMEDICAL ENGINEERING (2021)

Article Materials Science, Biomaterials

Illuminating endosomal escape of polymorphic lipid nanoparticles that boost mRNA delivery

Marco Herrera et al.

Summary: The study utilized a Galectin 8-GFP cell reporter system to visualize the endosomal escape capabilities of lipid-based nanoparticles (LNPs) encapsulating mRNA. LNPs containing beta-sitosterol showed superior ability to escape from endosomal entrapment compared to standard cholesterol LNPs. Live-cell imaging demonstrated a 10-fold increase in detectable endosomal perturbation events with LNPs containing beta-sitosterol.

BIOMATERIALS SCIENCE (2021)

Article Materials Science, Biomaterials

Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver

Rui Zhang et al.

Summary: Nucleic acids hold promise for treating 'undruggable' diseases, but face challenges in delivery due to biological barriers. Research found that LNPs with different formulations preferentially accumulate in the liver and spleen, impacting nucleic acid delivery. Understanding the role of helper lipids and ApoE in LNP distribution may help in designing effective nucleic acid therapeutics.

BIOMATERIALS SCIENCE (2021)

Review Cell Biology

Life in the lumen: The multivesicular endosome

Jean Gruenberg

TRAFFIC (2020)

Article Cell & Tissue Engineering

Naturally Derived Membrane Lipids Impact Nanoparticle-Based Messenger RNA Delivery

Jeonghwan Kim et al.

CELLULAR AND MOLECULAR BIOENGINEERING (2020)

Article Multidisciplinary Sciences

Synergistic lipid compositions for albumin receptor mediated delivery of mRNA to the liver

Lei Miao et al.

NATURE COMMUNICATIONS (2020)

Article Chemistry, Multidisciplinary

Deconvoluting Lipid Nanoparticle Structure for Messenger RNA Delivery

Yulia Eygeris et al.

NANO LETTERS (2020)

Article Nanoscience & Nanotechnology

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

Qiang Cheng et al.

NATURE NANOTECHNOLOGY (2020)

Editorial Material Biotechnology & Applied Microbiology

RNA therapeutics on the rise

Feng Wang et al.

NATURE REVIEWS DRUG DISCOVERY (2020)

Review Biochemical Research Methods

The Biomolecular Corona of Lipid Nanoparticles for Gene Therapy

Valentina Francia et al.

BIOCONJUGATE CHEMISTRY (2020)

Article Engineering, Biomedical

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

Pengxuan Zhao et al.

BIOACTIVE MATERIALS (2020)

Article Chemistry, Physical

Bilayer-Coated Nanoparticles Reveal How Influenza Viral Entry Depends on Membrane Deformability but Not Curvature

Ana M. Villamil Giraldo et al.

JOURNAL OF PHYSICAL CHEMISTRY LETTERS (2020)

Article Multidisciplinary Sciences

The effects of PEGylation on LNP based mRNA delivery to the eye

Renee C. Ryals et al.

PLOS ONE (2020)

Article Chemistry, Medicinal

Property-Driven Design and Development of Lipids for Efficient Delivery of siRNA

Kumar Rajappan et al.

JOURNAL OF MEDICINAL CHEMISTRY (2020)

Review Chemistry, Multidisciplinary

Cubosomes: The Next Generation of Smart Lipid Nanoparticles?

Hanna M. G. Barriga et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2019)

Article Chemistry, Multidisciplinary

Fusion-dependent formation of lipid nanoparticles containing macromolecular payloads

Jayesh A. Kulkarni et al.

NANOSCALE (2019)

Article Chemistry, Multidisciplinary

On the role of helper lipids in lipid nanoparticle formulations of siRNA

Jayesh A. Kulkarni et al.

NANOSCALE (2019)

Review Pharmacology & Pharmacy

Brief update on endocytosis of nanomedicines

Siddharth Patel et al.

ADVANCED DRUG DELIVERY REVIEWS (2019)

Article Chemistry, Physical

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

Jasmine Viger-Gravel et al.

JOURNAL OF PHYSICAL CHEMISTRY B (2018)

Article Multidisciplinary Sciences

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

Marianna Yanez Arteta et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2018)

Article Multidisciplinary Sciences

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

Cory D. Sago et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2018)

Article Nanoscience & Nanotechnology

Achieving long-term stability of lipid nanoparticles: examining the effect of pH, temperature, and lyophilization

Rebecca L. Ball et al.

INTERNATIONAL JOURNAL OF NANOMEDICINE (2017)

Review Chemistry, Multidisciplinary

Next-Generation Lipids in RNA Interference Therapeutics

Stephanie Rietwyk et al.

ACS NANO (2017)

Article Chemistry, Multidisciplinary

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

Sam Chen et al.

JOURNAL OF CONTROLLED RELEASE (2016)

Article Medicine, Research & Experimental

A Glu-urea-Lys Ligand-conjugated Lipid Nanoparticle/siRNA System Inhibits Androgen Receptor Expression In Vivo

Justin B Lee et al.

Molecular Therapy-Nucleic Acids (2016)

Article Chemistry, Multidisciplinary

Cuboplexes: Topologically Active siRNA Delivery

Hojun Kim et al.

ACS NANO (2015)

Article Chemistry, Physical

Microfluidic Mixing: A General Method for Encapsulating Macromolecules in Lipid Nanoparticle Systems

Alex K. K. Leung et al.

JOURNAL OF PHYSICAL CHEMISTRY B (2015)

Article Chemistry, Multidisciplinary

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

Bin Li et al.

NANO LETTERS (2015)

Review Biotechnology & Applied Microbiology

From sewer to saviour - targeting the lymphatic system to promote drug exposure and activity

Natalie L. Trevaskis et al.

NATURE REVIEWS DRUG DISCOVERY (2015)

Article Multidisciplinary Sciences

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

Yizhou Dong et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)

Article Medicine, Research & Experimental

Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy

Varun Kumar et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2014)

Article Medicine, Research & Experimental

Overcoming Nonviral Gene Delivery Barriers: Perspective and Future

Charles H. Jones et al.

MOLECULAR PHARMACEUTICS (2013)

Article Biotechnology & Applied Microbiology

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling

Gaurav Sahay et al.

NATURE BIOTECHNOLOGY (2013)

Article Chemistry, Physical

Lipid-mediated DNA and siRNA transfection efficiency depends on peptide headgroup

Xiao-Xiang Zhang et al.

SOFT MATTER (2013)

Article Medicine, Research & Experimental

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

Barbara L. Mui et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2013)

Article Chemistry, Multidisciplinary

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

Muthusamy Jayaraman et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2012)

Article Chemistry, Multidisciplinary

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

Delai Chen et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2012)

Article Biotechnology & Applied Microbiology

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

Akin Akinc et al.

MOLECULAR THERAPY (2010)

Review Chemistry, Physical

Physical approaches to biomaterial design

Samir Mitragotri et al.

NATURE MATERIALS (2009)

Review Biotechnology & Applied Microbiology

Knocking down barriers: advances in siRNA delivery

Kathryn A. Whitehead et al.

NATURE REVIEWS DRUG DISCOVERY (2009)

Article Chemistry, Multidisciplinary

Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids

J Heyes et al.

JOURNAL OF CONTROLLED RELEASE (2005)