Exosome for mRNA delivery: strategies and therapeutic applications

Journal of Nanobiotechnology, Journal Year: 2024, Volume and Issue: 22(1)

Published: July 4, 2024

Abstract Messenger RNA (mRNA) has emerged as a promising therapeutic molecule with numerous clinical applications in treating central nervous system disorders, tumors, COVID-19, and other diseases. mRNA therapies must be encapsulated into safe, stable, effective delivery vehicles to preserve the cargo from degradation prevent immunogenicity. Exosomes have gained growing attention because of their good biocompatibility, low immunogenicity, small size, unique capacity traverse physiological barriers, cell-specific tropism. Moreover, these exosomes can engineered utilize natural carriers target specific cells or tissues. This targeted approach will enhance efficacy reduce side effects mRNAs. However, difficulties such lack consistent reliable methods for exosome purification efficient encapsulation large mRNAs addressed. article outlines current breakthroughs cell-derived vesicle-mediated its biomedical applications. Graphical

Language: Английский

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The Common Hallmarks of Aging, Circadian Rhythms and Cancer: Implications for Therapeutic Strategies

Research, Journal Year: 2025, Volume and Issue: 8

Published: Jan. 1, 2025

The intricate relationship between cancer, circadian rhythms, and aging is increasingly recognized as a critical factor in understanding the mechanisms underlying tumorigenesis cancer progression. Aging well-established primary risk for while disruptions rhythms are intricately associated with progression of various tumors. Moreover, itself disrupts leading to physiological changes that may accelerate development. Despite these connections, specific interplay processes their collective impact on remains inadequately explored literature. In this review, we systematically explore influence We discuss how core genes tumor prognosis, highlighting shared hallmarks such genomic instability, cellular senescence, chronic inflammation. Furthermore, examine aging, focusing crosstalk contributes tumorigenesis, proliferation, apoptosis, well metabolism stability. By elucidating common pathways linking review provides new insights into pathophysiology identifies potential therapeutic strategies. propose targeting regulation could pave way novel treatments, including chronotherapy antiaging interventions, which offer important benefits clinical management cancer.

Language: Английский

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Navigating the landscape of RNA delivery systems in cardiovascular disease therapeutics

Advanced Drug Delivery Reviews, Journal Year: 2024, Volume and Issue: 208, P. 115302 - 115302

Published: April 3, 2024

Cardiovascular diseases (CVDs) stand as the leading cause of death worldwide, posing a significant global health challenge. Consequently, development innovative therapeutic strategies to enhance CVDs treatment is imperative. RNA-based therapies, encompassing non-coding RNAs, mRNA, aptamers, and CRISPR/Cas9 technology, have emerged promising tools for addressing CVDs. However, inherent challenges associated with RNA, such poor cellular uptake, susceptibility RNase degradation, capture by reticuloendothelial system, underscore necessity combining these therapies effective drug delivery systems. Various non-viral systems, including extracellular vesicles, lipid-based carriers, polymeric inorganic nanoparticles, well hydrogels, shown promise in enhancing efficacy RNA therapeutics. In this review, we offer an overview most relevant explored emphasize pivotal role systems augmenting their effectiveness. Additionally, discuss current status that hinder clinical translation.

Language: Английский

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Targeting Strategies for Site-Specific mRNA Delivery

Bioconjugate Chemistry, Journal Year: 2024, Volume and Issue: 35(4), P. 453 - 456

Published: March 16, 2024

mRNA therapeutics hold great promise for disease treatment, yet a key challenge lies in achieving site-specific delivery to maximize therapeutic efficacy while minimizing off-target side effects. This viewpoint delves into multiple complementary targeting strategies achieve precise delivery, covering topics of administration routes, passive targeting, and active targeting. It highlights the critical importance rationally designed nanocarriers obtaining desired effects accelerating clinical translation therapeutics.

Language: Английский

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tRNA-derived small RNAs: their role in the mechanisms, biomarkers, and therapeutic strategies of colorectal cancer

Journal of Translational Medicine, Journal Year: 2025, Volume and Issue: 23(1)

Published: Jan. 13, 2025

Colorectal cancer (CRC) is the third most prevalent malignancy and second leading cause of cancer-related mortality worldwide, with an increasing shift towards younger age onset. In recent years, there has been recognition significance tRNA-derived small RNAs (tsRNAs), encompassing fragments (tRFs) tRNA halves (tiRNAs). Their involvement in regulating translation, gene expression, reverse transcription, epigenetics gradually come to light. Emerging research revealed dysregulation tsRNAs CRC, implicating their role CRC initiation progression, highlighting potential early diagnosis, prognosis, therapeutic strategies. Although clinical application still its stages, findings highlight a close relationship between biogenesis function tsRNAs, chemical modifications, tumor immune microenvironment (TIME). Additionally, similar other RNAs, can be effectively delivered via nanoparticles (NPs). Consequently, future should focus on elucidating concerning base TIME regulation, immunotherapy, NPs delivery systems facilitate translation.

Language: Английский

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Advancements in Nanomedicine for the Diagnosis and Treatment of Kidney Stones

International Journal of Nanomedicine, Journal Year: 2025, Volume and Issue: Volume 20, P. 1401 - 1423

Published: Feb. 1, 2025

Abstract: Kidney stones constitute a common condition impacting the urinary system. In clinical diagnosis and management, traditional surgical interventions pharmacological treatments are primarily utilized; however, these methods possess inherent limitations. Presently, field of nanomedicine is undergoing significant advancements. The application nanomaterials in biosensors enables accurate assessment ion composition. Furthermore, contrast agents developed from materials can improve signal-to-noise ratio enhance image clarity. By mitigating oxidative stress-induced cellular damage, inhibit formation kidney efficacy drug delivery as effective carriers. Additionally, by modifying physical chemical properties bacteria, effectively eliminate bacterial presence, thereby preventing severe complications. This review explores advancements technology related to early detection risk factors, diagnosis, treatment their associated Keywords: nanomaterials, stones, stress, biomaterial

Language: Английский

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Developing mRNA Nanomedicines with Advanced Targeting Functions

Nano-Micro Letters, Journal Year: 2025, Volume and Issue: 17(1)

Published: Feb. 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.

Language: Английский

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Nano-Encapsulation and Conjugation Applied in the Development of Lipid Nanoparticles Delivering Nucleic Acid Materials to Enable Gene Therapies

Applied Nano, Journal Year: 2024, Volume and Issue: 5(3), P. 143 - 161

Published: Aug. 29, 2024

Nano-encapsulation and conjugation are the main strategies employed for drug delivery. Nanoparticles help improve encapsulation targeting efficiency, thus optimizing therapeutic efficacy. Through nanoparticle technology, replacement of a defective gene or delivery new into patient’s genome has become possible. Lipid nanoparticles (LNPs) loaded with genetic materials designed to be delivered specific target sites enable therapy. The lipid shells protect fragile from degradation, then successfully release payload inside cells, where it can integrate subsequently express protein interest. This review focuses on development LNPs nano-pharmaceutical techniques improving potency therapies, reducing toxicities, releasing achieve effects. In addition, we discuss preparation techniques, effects efficacy in delivering nucleic acid materials.

Language: Английский

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Recent Advances and Prospects in RNA Drug Development

International Journal of Molecular Sciences, Journal Year: 2024, Volume and Issue: 25(22), P. 12284 - 12284

Published: Nov. 15, 2024

RNA therapeutics have undergone remarkable evolution since their inception in the late 1970s, revolutionizing medicine by offering new possibilities for treating previously intractable diseases. The field encompasses various modalities, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), and messenger (mRNAs), each with unique mechanisms applications. foundation was laid 1978 discovery that synthetic could inhibit viral replication, followed pivotal developments such as interference's 1998. COVID-19 pandemic marked a crucial turning point, demonstrating potential of mRNA vaccines accelerating interest RNA-based approaches. However, significant challenges remain, stability issues, delivery to target tissues, off-target effects, immunogenicity concerns. Recent advancements chemical modifications, systems, integration AI technologies are addressing these challenges. has seen notable successes, approved treatments spinal muscular atrophy hereditary transthyretin-mediated amyloidosis. Looking ahead, show promise personalized approaches, particularly genetic disorders cancer. continued this field, driven technological innovations deeper understanding biology, suggests transformative impact on future medical treatments. purpose review is provide comprehensive overview evolution, current state, prospects therapeutics.

Language: Английский

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A review on Gaucher disease: therapeutic potential of β-glucocerebrosidase-targeted mRNA/saRNA approach

International Journal of Biological Sciences, Journal Year: 2024, Volume and Issue: 20(6), P. 2111 - 2129

Published: Jan. 1, 2024

Gaucher disease (GD), a rare hereditary lysosomal storage disorder, occurs due to deficiency in the enzyme β-glucocerebrosidase (GCase). This leads buildup of substrate glucosylceramide (GlcCer) macrophages, eventually resulting various complications. Among its three types, GD2 is particularly severe with neurological involvements. Current treatments, such as replacement therapy (ERT), are not effective for and GD3 their inability cross blood-brain barrier (BBB). Other treatment approaches, gene or chaperone therapies still experimental stages. Additionally, GD treatments costly can have certain side effects. The successful use messenger RNA (mRNA)-based vaccines COVID-19 2020 has sparked interest nucleic acid-based therapies. Remarkably, mRNA technology also offers novel approach protein purposes. self-amplifying (saRNA) shows promise, potentially producing more at lower doses. review aims explore potential cost-effective mRNA/saRNA-based therapy. GCase-mRNA/saRNA could offer new promising direction improving quality life extending lifespan individuals GD.

Language: Английский

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