The rapid advancement of mRNA as vaccines and therapeutic agents in the biomedical field has sparked hope in the fight against untreatable diseases. Successful clinical application of mRNA therapeutics largely depends on the carriers. Recently, a new and exciting focus has emerged on natural cell-derived vesicles. These nanovesicles offer many functions, including enhanced drug delivery capabilities and immune evasion, thereby presenting a unique and promising platform for the effective and safe delivery of mRNA therapeutics. In this study, we summarize the characteristics and properties of biomimetic delivery systems for mRNA therapeutics. In particular, we discuss the unique features of cellular membrane-derived vesicles (CDVs) and the combination of synthetic nanovesicles with CDVs.
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http://dx.doi.org/10.7150/thno.93755 | DOI Listing |
Nucleosides Nucleotides Nucleic Acids
January 2025
Faculty of Agriculture and Allied Sciences, C.V. Raman Global University, Bhubaneswar, India.
The field of biomedical science has witnessed another milestone with the advent of RNA-based therapeutics. This review explores three major RNA molecules, namely: messenger RNA (mRNA), RNA interference technology (RNAi), and Antisense Oligonucleotide (ASO), and analyses U.S.
View Article and Find Full Text PDFJ Cell Mol Med
January 2025
Department of Hematology, General Hospital, Tianjin Medical University, Tianjin, China.
Single-cell sequencing of lineage negative (Lin-) cells from patients with myelodysplastic syndromes (MDS) revealed a reduction in ferritin heavy chain 1 (FTH1) levels, yet the significance of this decrease in FTH1 in the pathophysiology of MDS remains unclear. In this study, we evaluated the role of FTH1 in patients with MDS. The mRNA expression of FTH1 in GlycoA nucleated erythrocytes from MDS patients was significantly lower than that in control group.
View Article and Find Full Text PDFJ Mater Chem B
January 2025
Chemical and Biological Engineering Department, The Hong Kong University of Science and Technology, Hong Kong SAR, China.
Inefficient endosomal escape has been regarded as the main bottleneck for intracellular nucleic acid delivery. While most research efforts have been spent on designing various nano-sized particles, we took a different path here, investigating micron-sized carriers for direct cytosol entry. Using the spontaneous co-assembly of mRNA and the designer 27 amino acid oligopeptide named pepMAX2, micron-sized co-assemblies were obtained with various sizes by altering the concentration of NaCl salt and time for pre-incubation.
View Article and Find Full Text PDFAm J Cancer Res
December 2024
Department of Biomedical Sciences, Discipline of Pharmacology, Edward Via College of Osteopathic Medicine (VCOM) Monroe, LA 71203, USA.
Prostate cancer (PCa) is the second leading cause of cancer-related deaths among American men. The development of metastatic castration resistant PCa (mCRPC) is the current clinical challenge. Antiandrogens such as Enzalutamide (ENZ) are commonly used for CRPC treatment.
View Article and Find Full Text PDFmRNA delivery offers new opportunities for disease treatment by directing cells to produce therapeutic proteins. However, designing highly stable mRNAs with programmable cell type-specificity remains a challenge. To address this, we measured the regulatory activity of 60,000 5' and 3' untranslated regions (UTRs) across six cell types and developed PARADE (Prediction And RAtional DEsign of mRNA UTRs), a generative AI framework to engineer untranslated RNA regions with tailored cell type-specific activity.
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