4.7 Article

Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) using multiple technical methods for tissue-specific mRNA delivery

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 Chemistry, Multidisciplinary

Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery

Kelsey L. Swingle et al.

Summary: In utero treatment of congenital disorders using mRNA delivery is a promising strategy to minimize disease burden before irreversible disease onset. LNP stability in amniotic fluid is crucial for efficient intra-amniotic mRNA delivery and potential treatment of congenital disorders prenatally.

JOURNAL OF CONTROLLED RELEASE (2022)

Article Materials Science, Biomaterials

Optimization of phospholipid chemistry for improved lipid nanoparticle (LNP) delivery of messenger RNA (mRNA)

Ester Alvarez-Benedicto et al.

Summary: Research has shown that manipulating the identity of the phospholipid in LNPs can enhance mRNA delivery efficacy, improve endosomal escape, and alter the organ tropism of LNPs. The choice of phospholipid is crucial for designing and optimizing LNPs for improved mRNA delivery and more effective therapeutics.

BIOMATERIALS SCIENCE (2022)

Article Multidisciplinary Sciences

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Jeong Pil Han et al.

Summary: This study developed lipid nanoparticles (LNPs) to deliver CRISPR-Cas9 for gene editing in hemophilia treatment. The inhibition of the antithrombin gene led to improved thrombin generation and recovery of bleeding-associated phenotypes in mouse models. The LNP-mediated CRISPR-Cas9 delivery was found to be safe and efficient.

SCIENCE ADVANCES (2022)

Article Chemistry, Multidisciplinary

Design of SARS-CoV-2 hFc-Conjugated Receptor-Binding Domain mRNA Vaccine Delivered via Lipid Nanoparticles

Uri Elia et al.

Summary: This study presents the design of an mRNA vaccine based on lipid nanoparticles (LNPs) encapsulated SARS-CoV-2 human Fc-conjugated receptor-binding domain (RBD-hFc), demonstrating its potential to induce strong immune responses and produce neutralizing antibodies, making it a promising candidate for a COVID-19 vaccine.

ACS NANO (2021)

Article Chemistry, Multidisciplinary

A Systematic Study of Unsaturation in Lipid Nanoparticles Leads to Improved mRNA Transfection In Vivo

Sang M. Lee et al.

Summary: The study identified an ionizable lipid series (4A3) with a citronellol-based (Cit) periphery as the best performer out of 91 amino lipids, showing superior lipid fusion and improved mRNA delivery efficacy in vivo through Selective ORgan Targeting (SORT). These findings demonstrate how lipid unsaturation promotes mRNA delivery and how lipid mixing can enhance efficacy.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2021)

Review Biotechnology & Applied Microbiology

Engineering precision nanoparticles for drug delivery

Michael J. Mitchell et al.

Summary: The development of nanoparticles has shown promising clinical applications, overcoming the limitations of therapeutic interventions and biological barriers. Precision therapies have enhanced treatment efficacy through personalized interventions. Advancements in nanoparticle design aim to optimize drug delivery, improving efficacy in general applications and enabling tailored designs for precision applications.

NATURE REVIEWS DRUG DISCOVERY (2021)

Article Immunology

Lipid nanoparticles enhance the efficacy of mRNA and protein subunit vaccines by inducing robust T follicular helper cell and humoral responses

Mohamad-Gabriel Alameh et al.

Summary: Lipid nanoparticle (LNP)-encapsulated nucleoside-modified mRNA vaccines have intrinsic adjuvant activity that enhances durable and protective antibody responses. Compared to the widely used MF59-like adjuvant AddaVax, this LNP formulation outperforms and relies on specific components and cytokine induction for its adjuvant activity.

IMMUNITY (2021)

Article Multidisciplinary Sciences

On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles

Sean A. Dilliard et al.

Summary: SORT nanoparticles have been developed to deliver genetic medicines to non-liver tissues by incorporating a supplemental SORT molecule and recruiting specific proteins to the nanoparticle surface, bypassing the liver accumulation barrier typically seen with traditional LNPs.

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

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

Min Qiu et al.

Summary: Loss-of-function mutations in Angiopoietin-like 3 (Angptl3) are linked to reduced blood lipid levels, making it a potential therapeutic target for lipid metabolism disorders. A lipid nanoparticle delivery system successfully edited Angptl3 gene in vivo, leading to significant reductions in lipid levels without off-target effects or toxicity, highlighting the potential of this platform for safe and effective gene editing therapies.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2021)

Article Multidisciplinary Sciences

Delivery of mRNA vaccine with a lipid-like material potentiates antitumor efficacy through Toll-like receptor 4 signaling

Hongxia Zhang et al.

Summary: The C1 lipid nanoparticle-based mRNA cancer nanovaccine efficiently delivers mRNA to antigen presenting cells, activates TLR4, induces inflammatory cytokines in dendritic cells, and shows significant antitumor efficacy.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2021)

Article Chemistry, Multidisciplinary

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

Lukas Farbiak et al.

Summary: The study demonstrates the use of dendrimer-based lipid nanoparticles for increasing HDR efficiency in gene editing. By optimizing component ratios, over 91% of cells were edited in vitro, with over 20% HDR efficiency achieved in xenograft tumors in vivo.

ADVANCED MATERIALS (2021)

Review Engineering, Biomedical

Microfluidic formulation of nanoparticles for biomedical applications

Sarah J. Shepherd et al.

Summary: Nanomedicine has advanced significantly in clinical applications, with microfluidic devices showing potential for generating reproducible nanoparticle formulations. These formulations enhance biomedical applications with controlled physical properties, pointing towards future production scale-independent formulations through parallelization and multi-step reactions.

BIOMATERIALS (2021)

Review Nanoscience & Nanotechnology

The current landscape of nucleic acid therapeutics

Jayesh A. Kulkarni et al.

Summary: The increasing number of approved nucleic acid therapeutics demonstrates their potential for long-lasting or curative effects by targeting genetic blueprints; however, their clinical translation relies on advances in delivery technologies. Four platform technologies, including antisense oligonucleotides, small interfering RNA conjugates, lipid nanoparticles, and adeno-associated virus vectors, have enabled the clinical translation of nucleic acid therapeutics, leading to the approval of cutting-edge genetic drugs.

NATURE NANOTECHNOLOGY (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 Medicine, Research & Experimental

Vitamin E Scaffolds of pH-Responsive Lipid Nanoparticles as DNA Vaccines in Cancer and Protozoan Infection

Mio Maeta et al.

MOLECULAR PHARMACEUTICS (2020)

Article Chemistry, Multidisciplinary

Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering

Margaret M. Billingsley et al.

NANO LETTERS (2020)

Article Pharmacology & Pharmacy

Charge-reversible lipid derivative: A novel type of pH-responsive lipid for nanoparticle-mediated siRNA delivery

Yusuke Hirai et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (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)

Article Multidisciplinary Sciences

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

Ugur Sahin et al.

NATURE (2020)

Article Materials Science, Biomaterials

Adjuvant incorporated lipid nanoparticles for enhanced mRNA-mediated cancer immunotherapy

Kyuri Lee et al.

BIOMATERIALS SCIENCE (2020)

Review Nanoscience & Nanotechnology

Nanoscale platforms for messenger RNA delivery

Bin Li et al.

WILEY INTERDISCIPLINARY REVIEWS-NANOMEDICINE AND NANOBIOTECHNOLOGY (2019)

Review Biotechnology & Applied Microbiology

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Piotr S. Kowalski et al.

MOLECULAR THERAPY (2019)

Article Medicine, Research & Experimental

Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines

Kimberly J. Hassett et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

Article Chemistry, Multidisciplinary

Lipid nanoparticles for delivery of messenger RNA to the back of the eye

Siddharth Patel et al.

JOURNAL OF CONTROLLED RELEASE (2019)

Article Nanoscience & Nanotechnology

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

Akin Akinc et al.

NATURE NANOTECHNOLOGY (2019)

Article Biotechnology & Applied Microbiology

Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

Ema Robinson et al.

MOLECULAR THERAPY (2018)

Review Biotechnology & Applied Microbiology

mRNA vaccines - a new era in vaccinology

Norbert Pardi et al.

NATURE REVIEWS DRUG DISCOVERY (2018)

Article Multidisciplinary Sciences

Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

Nuphar Veiga et al.

NATURE COMMUNICATIONS (2018)

Article Chemistry, Multidisciplinary

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

Matthias A. Oberli et al.

NANO LETTERS (2017)

Review Nanoscience & Nanotechnology

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

Khalid A. Hajj et al.

NATURE REVIEWS MATERIALS (2017)

Review Pharmacology & Pharmacy

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

Xinwei Cheng et al.

ADVANCED DRUG DELIVERY REVIEWS (2016)

Article Multidisciplinary Sciences

Modular degradable dendrimers enable small RNAs to extend survival in an aggressive liver cancer model

Kejin Zhou et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)

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

mRNA-based therapeutics - developing a new class of drugs

Ugur Sahin et al.

NATURE REVIEWS DRUG DISCOVERY (2014)

Review Pharmacology & Pharmacy

Liposomal drug delivery systems: From concept to clinical applications

Theresa M. Allen et al.

ADVANCED DRUG DELIVERY REVIEWS (2013)

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 Medicine, Research & Experimental

Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA

Nathan M. Belliveau et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2012)

Review Biotechnology & Applied Microbiology

Nanoparticle PEGylation for imaging and therapy

Jesse V. Jokerst et al.

NANOMEDICINE (2011)

Review Pharmacology & Pharmacy

Conventional and Dense Gas Techniques for the Production of Liposomes: A Review

Louise A. Meure et al.

AAPS PHARMSCITECH (2008)

Review Chemistry, Multidisciplinary

Lipid-based nanoparticles for nucleic acid delivery

Weijun Li et al.

PHARMACEUTICAL RESEARCH (2007)

Article Multidisciplinary Sciences

Chaotic mixer for microchannels

AD Stroock et al.

SCIENCE (2002)