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Pharm Res 2022 Jan 26;39(1):105-114. Epub 2022 Jan 26.

School of Pharmacy, Shanghai Jiao Tong University, Shanghai, China.

Purpose: Lipid nanoparticles (LNPs) are widely utilized as means to deliver mRNA molecules. However, metric connections between biodistribution and pharmacokinetics (PK) of the nanoparticle carrier and transgene expression dynamics remain largely unknown.

Methods: LNPs containing mRNAs encoding the firefly luciferase gene were prepared with varying sizes. Read More

J Control Release 2022 03 22;343:207-216. Epub 2022 Jan 22.

CDL Research, UMC Utrecht, Utrecht, the Netherlands. Electronic address:

Lipid Nanoparticles (LNPs) are a promising drug delivery vehicle for clinical siRNA delivery. Modified mRNA (modRNA) has recently gained great attention as a therapeutic molecule in cardiac regeneration. However, for mRNA to be functional, it must first reach the diseased myocardium, enter the target cell, escape from the endosomal compartment into the cytosol and be translated into a functional protein. Read More

Mol Ther Nucleic Acids 2021 Sep 10;25:708-715. Epub 2021 Sep 10.

Department of Immunology, Yokohama City University Graduate School of Medicine, Yokohama 236-0004, Japan.

RNA interference-based gene silencing drugs are attracting attention for treating various diseases. Lipid nanoparticles (LNPs) are carriers that efficiently deliver small interfering RNA (siRNA) to the cytoplasm of target cells. Recently, we developed potent and well-tolerated biodegradable LNPs with asymmetric ionizable lipids. Read More

Vaccines (Basel) 2021 Sep 10;9(9). Epub 2021 Sep 10.

Hematopoietic Stem Cell Research Center, Shahid Beheshti University of Medical Sciences, Tehran 1983969411, Iran.

Among the vaccines have been developed thus far against SARS-CoV-2, the mRNA-based ones have demonstrated more promising results regarding both safety and efficacy. Two remarkable features of the mRNA vaccines introduced by the Pfizer/BioNTech and Moderna companies are the use of (N-methyl-pseudouridine-) modified mRNA and the microfluidics-based production of lipid nanoparticles (LNPs) as the carrier. In the present study, except Anti-Reverse Cap Analog (ARCA), no other nucleoside analogs were employed to synthesize Spike-encoding mRNA using the in vitro transcription (IVT) method. Read More

Drug Des Devel Ther 2021 18;15:3953-3963. Epub 2021 Sep 18.

GenePharma Co., Ltd., Suzhou, 215125, Jiangsu, People's Republic of China.

Purpose: The purpose of our research was to identify and evaluate synthetic phosphorothioate-modified CPG oligodeoxynucleotides (CPG-ODNs) activating innate and adaptive immune responses. Furthermore, combined treatment with CpG and an mRNA cancer vaccine was evaluated in melanoma models as a therapeutic approach.

Methods: A molecular assay was used to screen new CpG molecules; mouse modeling and pathological analysis were used to confirm the antitumor effect of CpG alone or in combination with an mRNA vaccine. Read More