[HTML][HTML] Drug delivery systems for RNA therapeutics

K Paunovska, D Loughrey, JE Dahlman - Nature Reviews Genetics, 2022 - nature.com
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Nanotechnology-based mRNA vaccines

S Chen, X Huang, Y Xue, E Álvarez-Benedicto… - Nature Reviews …, 2023 - nature.com
Abstract mRNA vaccines have emerged as a revolutionary tool to generate rapid and
precise immune responses against infectious diseases and cancers. Compared with …

An ionizable lipid toolbox for RNA delivery

X Han, H Zhang, K Butowska, KL Swingle… - Nature …, 2021 - nature.com
Recent years have witnessed incredible growth in RNA therapeutics, which has benefited
significantly from decades of research on lipid nanoparticles, specifically its key component …

Lipids and lipid derivatives for RNA delivery

Y Zhang, C Sun, C Wang, KE Jankovic… - Chemical reviews, 2021 - ACS Publications
RNA-based therapeutics have shown great promise in treating a broad spectrum of
diseases through various mechanisms including knockdown of pathological genes …

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

MP Lokugamage, D Vanover, J Beyersdorf… - Nature biomedical …, 2021 - nature.com
Lipid nanoparticles (LNPs) for the efficient delivery of drugs need to be designed for the
particular administration route and type of drug. Here we report the design of LNPs for the …

Lipid nanoparticle (LNP) enables mRNA delivery for cancer therapy

Y Zong, Y Lin, T Wei, Q Cheng - Advanced Materials, 2023 - Wiley Online Library
Messenger RNA (mRNA) has received great attention in the prevention and treatment of
various diseases due to the success of coronavirus disease 2019 (COVID‐19) mRNA …

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

Q Cheng, T Wei, L Farbiak, LT Johnson… - Nature …, 2020 - nature.com
CRISPR–Cas gene editing and messenger RNA-based protein replacement therapy hold
tremendous potential to effectively treat disease-causing mutations with diverse cellular …

Drug delivery systems for CRISPR-based genome editors

V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

RNA drugs and RNA targets for small molecules: principles, progress, and challenges

AM Yu, YH Choi, MJ Tu - Pharmacological reviews, 2020 - ASPET
RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …

Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo

H Ni, MZC Hatit, K Zhao, D Loughrey… - Nature …, 2022 - nature.com
In humans, lipid nanoparticles (LNPs) have safely delivered therapeutic RNA to hepatocytes
after systemic administration and to antigen-presenting cells after intramuscular injection …