mRNA-based therapeutics: powerful and versatile tools to combat diseases

S Qin, X Tang, Y Chen, K Chen, N Fan… - Signal transduction and …, 2022 - nature.com
The therapeutic use of messenger RNA (mRNA) has fueled great hope to combat a wide
range of incurable diseases. Recent rapid advances in biotechnology and molecular …

Amplifying gene expression with RNA-targeted therapeutics

O Khorkova, J Stahl, A Joji, CH Volmar… - Nature Reviews Drug …, 2023 - nature.com
Many diseases are caused by insufficient expression of mutated genes and would benefit
from increased expression of the corresponding protein. However, in drug development, it …

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 …

Optimization of storage conditions for lipid nanoparticle-formulated self-replicating RNA vaccines

B Kim, RR Hosn, T Remba, D Yun, N Li… - Journal of Controlled …, 2023 - Elsevier
The recent clinical success of multiple mRNA-based SARS-CoV-2 vaccines has proven the
potential of RNA formulated in lipid nanoparticles (LNPs) in humans, and products based on …

Quality by Design for enabling RNA platform production processes

S Daniel, Z Kis, C Kontoravdi, N Shah - Trends in biotechnology, 2022 - cell.com
RNA-based products have emerged as one of the most promising and strategic
technologies for global vaccination, infectious disease control, and future therapy …

Recent advances in the development of toll-like receptor agonist-based vaccine adjuvants for infectious diseases

JX Yang, JC Tseng, GY Yu, Y Luo, CYF Huang… - Pharmaceutics, 2022 - mdpi.com
Vaccines are powerful tools for controlling microbial infections and preventing epidemic
diseases. Efficient inactive, subunit, or viral-like particle vaccines usually rely on a safe and …

mRNA in the context of protein replacement therapy

T Vavilis, E Stamoula, A Ainatzoglou, A Sachinidis… - Pharmaceutics, 2023 - mdpi.com
Protein replacement therapy is an umbrella term used for medical treatments that aim to
substitute or replenish specific protein deficiencies that result either from the protein being …

Nucleic acid degradation as barrier to gene delivery: a guide to understand and overcome nuclease activity

H Zhang, J Vandesompele, K Braeckmans… - Chemical Society …, 2024 - pubs.rsc.org
Gene therapy is on its way to revolutionize the treatment of both inherited and acquired
diseases, by transferring nucleic acids to correct a disease-causing gene in the target cells …

Complete substitution with modified nucleotides in self-amplifying RNA suppresses the interferon response and increases potency

JE McGee, JR Kirsch, D Kenney, F Cerbo… - Nature …, 2024 - nature.com
The use of modified nucleotides to suppress the interferon response and maintain
translation of self-amplifying RNA (saRNA), which has been achieved for mRNA, has not yet …

[HTML][HTML] mRNA: A promising platform for cancer immunotherapy

B Kong, Y Kim, EH Kim, JS Suk, Y Yang - Advanced Drug Delivery Reviews, 2023 - Elsevier
Messenger RNA (mRNA) is now in the limelight as a powerful tool for treating various
human diseases, especially malignant tumors, thanks to the remarkable clinical outcomes of …