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 …

Chemically modified platforms for better RNA therapeutics

Y Shi, X Zhen, Y Zhang, Y Li, S Koo, Q Saiding… - Chemical …, 2024 - ACS Publications
RNA-based therapies have catalyzed a revolutionary transformation in the biomedical
landscape, offering unprecedented potential in disease prevention and treatment. However …

Strategies to reduce the risks of mRNA drug and vaccine toxicity

D Bitounis, E Jacquinet, MA Rogers… - Nature Reviews Drug …, 2024 - nature.com
Abstract mRNA formulated with lipid nanoparticles is a transformative technology that has
enabled the rapid development and administration of billions of coronavirus disease 2019 …

Recent advances in nanoparticulate RNA delivery systems

J Witten, Y Hu, R Langer… - Proceedings of the …, 2024 - National Acad Sciences
Nanoparticle-based RNA delivery has shown great progress in recent years with the
approval of two mRNA vaccines for Severe Acute Respiratory Syndrome Coronavirus 2 …

A general temperature-guided language model to design proteins of enhanced stability and activity

F Jiang, M Li, J Dong, Y Yu, X Sun, B Wu, J Huang… - Science …, 2024 - science.org
Designing protein mutants with both high stability and activity is a critical yet challenging
task in protein engineering. Here, we introduce PRIME, a deep learning model, which can …

Tailor made: the art of therapeutic mRNA design

M Metkar, CS Pepin, MJ Moore - Nature Reviews Drug Discovery, 2024 - nature.com
Abstract mRNA medicine is a new and rapidly developing field in which the delivery of
genetic information in the form of mRNA is used to direct therapeutic protein production in …

mRNA vaccines for infectious diseases—advances, challenges and opportunities

N Pardi, F Krammer - Nature Reviews Drug Discovery, 2024 - nature.com
The concept of mRNA-based vaccines emerged more than three decades ago.
Groundbreaking discoveries and technological advancements over the past 20 years have …

Rapid generation of long, chemically modified pegRNAs for prime editing

X Lei, A Huang, D Chen, X Wang, R Ji, J Wang… - Nature …, 2024 - nature.com
The editing efficiencies of prime editing (PE) using ribonucleoprotein (RNP) and RNA
delivery are not optimal due to the challenges in solid-phase synthesis of long PE guide …

Advancements of in vitro transcribed mRNA (IVT mRNA) to enable translation into the clinics

DD Kang, H Li, Y Dong - Advanced Drug Delivery Reviews, 2023 - Elsevier
The accelerated progress and approval of two mRNA-based vaccines to address the SARS-
CoV-2 virus were unprecedented. This record-setting feat was made possible through the …

Advances in the design and delivery of RNA vaccines for infectious diseases

AG Lokras, TR Bobak, SS Baghel, F Sebastiani… - Advanced Drug Delivery …, 2024 - Elsevier
RNA medicines represent a paradigm shift in treatment and prevention of critical diseases of
global significance, eg, infectious diseases. The highly successful messenger RNA (mRNA) …