Unlocking the promise of mRNA therapeutics

E Rohner, R Yang, KS Foo, A Goedel… - Nature biotechnology, 2022 - nature.com
The extraordinary success of mRNA vaccines against coronavirus disease 2019 (COVID-19)
has renewed interest in mRNA as a means of delivering therapeutic proteins. Early clinical …

[HTML][HTML] Lipid nanoparticles for mRNA delivery

X Hou, T Zaks, R Langer, Y Dong - Nature Reviews Materials, 2021 - nature.com
Messenger RNA (mRNA) has emerged as a new category of therapeutic agent to prevent
and treat various diseases. To function in vivo, mRNA requires safe, effective and stable …

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 …

Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

Non-viral COVID-19 vaccine delivery systems

KS Park, X Sun, ME Aikins, JJ Moon - Advanced drug delivery reviews, 2021 - Elsevier
The novel corona virus termed severe acute respiratory syndrome coronavirus 2 (SARS-
CoV-2) has spread throughout the globe at a formidable speed, causing tens of millions of …

[HTML][HTML] Delivering the messenger: advances in technologies for therapeutic mRNA delivery

PS Kowalski, A Rudra, L Miao, DG Anderson - Molecular Therapy, 2019 - cell.com
mRNA has broad potential as a therapeutic. Current clinical efforts are focused on
vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical …

Ionizable lipid nanoparticles deliver mRNA to pancreatic β cells via macrophage-mediated gene transfer

JR Melamed, SS Yerneni, ML Arral, ST LoPresti… - Science …, 2023 - science.org
Systemic messenger RNA (mRNA) delivery to organs outside the liver, spleen, and lungs
remains challenging. To overcome this issue, we hypothesized that altering nanoparticle …

Block copolymer micelles in nanomedicine applications

H Cabral, K Miyata, K Osada, K Kataoka - Chemical reviews, 2018 - ACS Publications
Polymeric micelles are demonstrating high potential as nanomedicines capable of
controlling the distribution and function of loaded bioactive agents in the body, effectively …

Strategies for non-viral vectors targeting organs beyond the liver

J Kim, Y Eygeris, RC Ryals, A Jozić, G Sahay - Nature nanotechnology, 2024 - nature.com
In recent years, nanoparticles have evolved to a clinical modality to deliver diverse nucleic
acids. Rising interest in nanomedicines comes from proven safety and efficacy profiles …

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

KA Hajj, KA Whitehead - Nature Reviews Materials, 2017 - nature.com
In recent years, messenger RNA (mRNA) has come into the spotlight as a versatile
therapeutic with the potential to prevent and treat a staggering range of diseases. Billions of …