mRNA-based therapies have broad applications in various disease treatments and have been applied in protein replacement therapy, gene editing, and vaccine development. Numerous research studies have been carried out aiming to increase the stability of mRNA, improve its translational efficiency, and reduce its immunogenicity. However, given mRNA's large molecular size and strong electronegativity, the safety and efficient delivery of mRNA into the target cells remains the critical rate-limiting step in current mRNA drug development. Various nanocarriers, such as liposomes, lipid nanoparticles, polyetherimide, and mesoporous silica nanoparticles, have been employed for mRNA delivery in the past few decades. Among them, peptides have demonstrated great potential as promising carrier candidates for mRNA delivery due to their high cell membrane permeability, good biocompatibility, definite chemical structure, and ease of preparation. Here, peptide-based mRNA delivery systems are systematically analyzed, including their construction strategies, mechanisms of action in mRNA delivery, and the application limitations or challenges. It is hoped that this review will guide the design, optimization, and applications of peptide carriers in mRNA-based drug development.
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http://dx.doi.org/10.1039/d4cb00295d | DOI Listing |
RSC Chem Biol
February 2025
Pingshan Translational Medicine Center, Shenzhen Bay Laboratory Shenzhen 518118 China
mRNA-based therapies have broad applications in various disease treatments and have been applied in protein replacement therapy, gene editing, and vaccine development. Numerous research studies have been carried out aiming to increase the stability of mRNA, improve its translational efficiency, and reduce its immunogenicity. However, given mRNA's large molecular size and strong electronegativity, the safety and efficient delivery of mRNA into the target cells remains the critical rate-limiting step in current mRNA drug development.
View Article and Find Full Text PDFNat Protoc
March 2025
Division of Pharmacoengineering and Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
mRNA-based therapies have emerged as a cutting-edge approach for diverse therapeutic applications. However, substantial barriers exist that hinder scientists from entering this research field, including the technical complexity and multiple potential workflows available for formulating and evaluating mRNA lipid nanoparticles (LNPs). Here we present an easy-to-follow and step-by-step guide for mRNA LNP formulation, characterization and in vitro and in vivo evaluation that could lower these barriers, facilitating entry for scientists in academia, industry and clinical settings into this research space.
View Article and Find Full Text PDFAtherosclerosis
January 2025
Division of Endocrinology and Metabolism, Department of Medicine, M0682, University of California San Diego, 9500 Gilman Dr, La Jolla, CA, USA. Electronic address:
Familial chylomicronemia syndrome (FCS) is a rare, recessive monogenic disorder characterized by severely elevated plasma triglyceride (TG) levels due to absent or markedly impaired lipoprotein lipase activity, leading to a greatly increased risk of acute pancreatitis. Naturally occurring very low levels of apoC-III are associated with low TG levels; thus, apoC-III is a target for TG lowering, and therapies have been developed to reduce apoC-III. Strategies to inhibit hepatic apoC-III synthesis include antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs).
View Article and Find Full Text PDFMicrosyst Nanoeng
March 2025
Guangdong Provincial Key Laboratory of Sensor Technology and Biomedical Instrument, School of Biomedical Engineering, Shenzhen Campus of Sun Yat-Sen University, Shenzhen, 518107, PR China.
Hypertrophic scar (HS) is a plaque fibrous and indurated dermal lesion that may cause physical, psychological, and cosmetic challenges for patients. Intralesional injection of triamcinolone acetonide (TA) is commonly used in clinical practice, which cause unbearable pain and uneven drug delivery within HS tissue. Herein, we developed a paper battery powered iontophoresis-driven microneedles patch (PBIMNP) for self-management of HS.
View Article and Find Full Text PDFToxicology
March 2025
Nucleic Acid Medicine Innovation Center, Zhejiang Haichang Biotech Co., Ltd.,Hangzhou, Zhejiang, 310020, China. Electronic address:
mRNA-based technology has been evaluated in a variety of clinical trials for the rapid control and prevention of emergencies and therapeutic indications. HC009 is a mRNA-based vaccine encoding the full-length of the SARS-CoV-2 spike protein and delivered by the QTsome delivery platform. Here, the immunogenicity, toxicity, and biodistribution of HC009 were investigated in rats.
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