Developing mRNA Nanomedicines with Advanced Targeting Functions

Nano-Micro Letters, Год журнала: 2025, Номер 17(1)

Опубликована: Фев. 21, 2025

The emerging messenger RNA (mRNA) nanomedicines have sprung up for disease treatment. Developing targeted mRNA has become a thrilling research hotspot in recent years, as they can be precisely delivered to specific organs or tissues enhance efficiency and avoid side effects. Herein, we give comprehensive review on the latest progress of with targeting functions. its carriers are first described detail. Then, mechanisms passive targeting, endogenous active outlined, focus various biological barriers that may encounter during vivo delivery. Next, emphasis is placed summarizing mRNA-based organ-targeting strategies. Lastly, advantages challenges clinical translation mentioned. This expected inspire researchers this field drive further development technology.

Язык: Английский

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RNA-based therapies in liver metabolic diseases

Gut, Год журнала: 2025, Номер unknown, С. gutjnl - 331742

Опубликована: Фев. 23, 2025

RNA-based therapeutics have rapidly emerged over the past decade, offering a new class of medicines that differ significantly from conventional drugs. These therapies can be programmed to target or restore defective genes, allowing for more personalised treatments and reducing side effects. Notably, RNA made significant progress in treatment genetic liver diseases, exemplified by small interfering hereditary transthyretin amyloidosis, which use liver-targeting strategies such as GalNAc conjugation improve efficacy safety. gene-editing technologies, base editor prime clustered regularly interspaced short palindromic repeats systems, also show promise with their ability minimise genomic rearrangements cancer risk. While offer high precision, challenges remain optimising delivery methods ensuring long-term safety efficacy. Lipid nanoparticle-mRNA therapeutics, particularly protein replacement rare gained support preclinical successes. Compared viral gene therapies, mRNA present safer profile reduced risks integration oncogene activation. However, clinical trials, especially face limitations sample sizes observation periods. Further studies, including non-human primates, will essential refining trial designs. Despite potential, costs pose challenge require cost–utility models guide pricing accessibility. Here, we discuss fundamental aspects showcase most relevant developments metabolic diseases.

Язык: Английский

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Administration of Alistipes indistinctus prevented the progression from nonalcoholic fatty liver disease to nonalcoholic steatohepatitis by enhancing the gut barrier and increasing Lactobacillus spp.

Biochemical and Biophysical Research Communications, Год журнала: 2024, Номер 741, С. 151033 - 151033

Опубликована: Ноя. 20, 2024

Язык: Английский

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Influence of salt solution on the physicochemical properties and in vitro/ in vivo expression of mRNA/LNP

Journal of Nanobiotechnology, Год журнала: 2025, Номер 23(1)

Опубликована: Март 19, 2025

Lipid nanoparticles (LNPs) have revolutionized nucleic acid delivery, enabling significant advances in mRNA-based therapeutics. While extensive research has focused on lipid composition, the impact of preparation solutions LNP performance remains underexplored. This study systematically investigated effects pH, salt type, and concentration across key solutions—mRNA aqueous, dilution, exchange, storage solutions—on physicochemical properties, stability, expression efficiency SM102-based mRNA/LNPs. Findings revealed that pH mRNA aqueous solution was critical, with a 4 optimizing encapsulation (EE) cellular expression. The exchange solution's significantly influenced biodistribution, particularly liver-specific following intravenous intramuscular administration. Sucrose identified as essential for freeze-thaw 300 mM minimizing aggregation leakage. Furthermore, were shown to influence structural integrity LNPs, impacting their vivo vitro performance. These insights highlight importance conditions formulations clinical applications, offering foundation enhanced therapeutic design delivery.

Язык: Английский

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Histidine Decapeptide‐Incorporating Lipid Nanoparticles with Low Ionizable Lipids Proportion for Efficient Small Interfering RNA Delivery

Macromolecular Bioscience, Год журнала: 2025, Номер unknown

Опубликована: Май 14, 2025

Abstract The diversification of lipid compositions in nanoparticles (LNPs) is crucial for expanding their clinical applications and overcoming current limitations. In this study, LNPs with varying are fabricated using three different mixing processes (pipette, vortex, microfluidic mixing) small interfering RNA (siRNA) delivery. While both siRNA hydrophobic fluorescent dye successfully incorporated within pipette‐ vortex‐mixing, hydrophilic peptides cannot be encapsulated. Following optimization ionizable proportion via cost‐efficient vortex‐mixing method, a lower (27.72%), termed LNP5, selected histidine decapeptide (His10) during formulation method to supplement the function approximately half lipids by simple addition His10. His10‐ LNP5 (LNP5H) exhibited 1.6‐fold increase gene silencing efficiency, compared conventional (cLNPs; 47.95%). Furthermore, LNP5H maintained potency 4 weeks when stored 1% sucrose solution at −70 °C. Taken together, it fabricates potent low fast easy processes, which can applied variety therapeutics efficient intracellular

Язык: Английский

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Nano-enabled regulation of DNA damage in tumor cells to enhance neoantigen-based pancreatic cancer immunotherapy

Biomaterials, Год журнала: 2024, Номер 311, С. 122710 - 122710

Опубликована: Июль 21, 2024

Язык: Английский

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Lipid nanoparticles: A delicate nucleic acid delivery system to be further explored

Nano Today, Год журнала: 2024, Номер 61, С. 102586 - 102586

Опубликована: Дек. 7, 2024

Язык: Английский

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