Nature Protocols, Journal Year: 2025, Volume and Issue: unknown
Published: March 11, 2025
Language: Английский
Advanced Materials, Journal Year: 2024, Volume and Issue: 36(26)
Published: April 7, 2024
Abstract Brain disorders represent a significant challenge in medical science due to the formidable blood–brain barrier (BBB), which severely limits penetration of conventional therapeutics, hindering effective treatment strategies. This review delves into innovative realm biomimetic nanodelivery systems, including stem cell‐derived nanoghosts, tumor cell membrane‐coated nanoparticles, and erythrocyte membrane‐based carriers, highlighting their potential circumvent BBB's restrictions. By mimicking native properties, these nanocarriers emerge as promising solution for enhancing drug delivery brain, offering strategic advantage overcoming barrier's selective permeability. The unique benefits leveraging membranes from various sources is evaluated advanced technologies fabricating membrane‐encapsulated nanoparticles capable masquerading endogenous cells are examined. enables targeted broad spectrum therapeutic agents, ranging small molecule drugs proteins, thereby providing an approach neurocare. Further, contrasts capabilities limitations with traditional methods, underlining enable targeted, sustained, minimally invasive modalities. concluded perspective on clinical translation underscoring transformative impact landscape intractable brain diseases.
Language: Английский
Citations
27
Advanced Drug Delivery Reviews, Journal Year: 2024, Volume and Issue: 206, P. 115190 - 115190
Published: Feb. 1, 2024
mRNA-based vaccines are emerging as a promising alternative to standard cancer treatments and the conventional vaccines. Moreover, FDA-approval of three nucleic acid based therapeutics (Onpattro, BNT162b2 mRNA-1273) has further increased interest trust on this type therapeutics. In order achieve significant therapeutic efficacy, mRNA needs from drug delivery system. last years, several platforms have been explored, being lipid nanoparticles (LNPs) most well characterized studied. A better understanding how operate (both itself system) will help improve their efficacy safety. review, we provide an overview what mode action highlight advantages challenges different that under investigation.
Language: Английский
Citations
22
Exploration, Journal Year: 2024, Volume and Issue: 4(6)
Published: April 15, 2024
Abstract Over the last two decades, lipid nanoparticles (LNPs) have evolved as an effective biocompatible and biodegradable RNA delivery platform in fields of nanomedicine, biotechnology, drug delivery. They are novel bionanomaterials that can be used to encapsulate a wide range biomolecules, such mRNA, demonstrated by current successes COVID‐19 mRNA vaccines. Therefore, it is important provide perspective on LNPs for delivery, which further offers useful guidance researchers who want work RNA‐based LNP field. This first summarizes approaches preparation LNPs, followed introduction key characterization parameters. Then, vitro cell experiments study performance, including selection, viability, cellular association/uptake, endosomal escape, their efficacy, were summarized. Finally, vivo animal aspects administration, dosing safety, therapeutic efficacy discussed. The authors hope this offer valuable enter field help them understand crucial parameters demand.
Language: Английский
Citations
18
Journal of Controlled Release, Journal Year: 2024, Volume and Issue: 375, P. 366 - 388
Published: Sept. 18, 2024
Recent advancements in RNA therapeutics highlight the critical need for precision gene delivery systems that target specific organs and cells. Lipid nanoparticles (LNPs) have emerged as key vectors delivering mRNA siRNA, offering protection against enzymatic degradation, enabling targeted cellular uptake, facilitating cargo release into cytosol. This review discusses development optimization of organ- cell-specific LNPs, focusing on their design, mechanisms action, therapeutic applications. We explore innovations such DNA/RNA barcoding, which facilitates high-throughput screening precise adjustments formulations. address major challenges, including improving endosomal escape, minimizing off-target effects, enhancing efficiencies. Notable clinical trials recent FDA approvals illustrate practical applications future potential LNP-based therapies. Our findings suggest while considerable progress has been made, continued research is essential to resolve existing limitations bridge gap between pre-clinical evaluation safety efficacy therapeutics. highlights dynamic LNP research. It outlines a roadmap RNA-based medicine.
Language: Английский
Citations
14
Biochimica et Biophysica Acta (BBA) - General Subjects, Journal Year: 2024, Volume and Issue: 1868(3), P. 130558 - 130558
Published: Jan. 5, 2024
Language: Английский
Citations
8
International Journal of Biological Macromolecules, Journal Year: 2025, Volume and Issue: 295, P. 139532 - 139532
Published: Jan. 5, 2025
Language: Английский
Citations
1
Advanced Science, Journal Year: 2025, Volume and Issue: unknown
Published: Feb. 8, 2025
Nanoparticle-based mRNA delivery offers a versatile platform for innovative therapies. However, most of the current systems are limited by poor serum tolerance, suboptimal endosomal escape and efficacy. Herein, highly efficient mRNA-delivering material is identified from library fluoropolymers. The lead FD17 shows exceptional stability escape, enabling into various cell types, surpassing commercial reagents such as Lipofectamine 3000. formed nanoparticles adsorb abundant albumin on surface, which facilitates cellular uptake via scavenger receptor-mediated endocytosis. enables mRNAs encoding CRE, Cas9, base editor hyCBE genome editing. mediates CRISPR/Cas9 gene therapy intraocular injection effectively down-regulates vascular endothelial growth factor A in retinal pigment epithelial cells mice, yielding promising therapeutic responses against laser-induced choroidal neovascularization. discovered this study great promise development therapeutics to combat wide range diseases.
Language: Английский
Citations
1
Heliyon, Journal Year: 2024, Volume and Issue: 10(9), P. e29726 - e29726
Published: April 16, 2024
Hybrid nanostructures exhibit a synergistic combination of features derived from their individual components, showcasing novel characteristics resulting distinctive structure and chemical/physical properties. Surface modifiers play pivotal role in shaping INPs' primary attributes, influencing physicochemical properties, stability, functional applications. Among these modifiers, dendrimers have gained attention as highly effective multifunctional agents for INPs, owing to unique structural qualities, dendritic effects, Dendrimers can be seamlessly integrated with diverse inorganic nanostructures, including metal NPs, carbon silica QDs. Two viable approaches achieving this integration involve either growing or grafting dendrimers, nanostructure-cored dendrimers. The initial step involves functionalizing the nanostructures' surface, followed by generation through stepwise growth attachment pre-synthesized dendrimer branches. This hybridization imparts superior qualities structure, biocompatibility, solubility, high cargo loading capacity, substantial functionalization potential. Combining properties those nanostructure cores creates system suitable applications such theranostics, bio-sensing, component isolation, chemotherapy, cargo-carrying review summarizes recent developments, specific focus on last five years, within realm It delves into INPs explores potential INP-cored biomedical
Language: Английский
Citations
7
ACS Applied Materials & Interfaces, Journal Year: 2024, Volume and Issue: 16(20), P. 25698 - 25709
Published: May 8, 2024
Much of current clinical interest has focused on mRNA therapeutics for the treatment lung-associated diseases, such as infections, genetic disorders, and cancers. However, safe efficient delivery to lungs, especially different pulmonary cell types, is still a formidable challenge. In this paper, we proposed cationic lipid pair (CLP) strategy, which utilized liver-targeted ionizable its derived quaternary ammonium CLP improve liver-to-lung tropism four-component nanoparticles (LNPs) in vivo delivery. Interestingly, structure–activity investigation identified that using lipids with higher performance their counterparts optimal design improving lung-targeted The strategy was also verified be universal suitable clinically available SM-102 ALC-0315 develop LNP systems. Moreover, demonstrated CLP-based LNPs were exhibited potent transfection endothelial epithelial cells. As result, provided powerful shifting preference from liver exhibiting great potential broadening application scenario mRNA-based therapy.
Language: Английский
Citations
7
Signal Transduction and Targeted Therapy, Journal Year: 2024, Volume and Issue: 9(1)
Published: Nov. 14, 2024
Abstract In the last decade, messenger ribonucleic acid (mRNA)-based drugs have gained great interest in both immunotherapy and non-immunogenic applications. This surge can be largely attributed to demonstration of distinct advantages offered by various mRNA molecules, alongside rapid advancements nucleic delivery systems. It is noteworthy that immunogenicity presents a double-edged sword. context immunotherapy, extra supplementation adjuvant generally required for induction robust immune responses. Conversely, non-immunotherapeutic scenarios, activation unwanted considering host tolerability high expression demand mRNA-encoded functional proteins. Herein, mainly focused on linear non-replicating mRNA, we overview preclinical clinical progress prospects medicines encompassing vaccines other therapeutics. We also highlight importance focusing host-specific variations, including age, gender, pathological condition, concurrent medication individual patient, maximized efficacy safety upon administration. Furthermore, deliberate potential challenges may encounter realm disease treatment, current endeavors improvement, as well application future advancements. Overall, this review aims present comprehensive understanding mRNA-based therapies while illuminating prospective development drugs.
Language: Английский
Citations
6