Nano Today, Journal Year: 2024, Volume and Issue: 61, P. 102586 - 102586
Published: Dec. 7, 2024
Language: Английский
ACS Nano, Journal Year: 2025, Volume and Issue: unknown
Published: Jan. 21, 2025
Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these face numerous challenges practical applications, particularly regarding their stability, effectiveness, cellular uptake efficiency, and limitations delivering them specifically to target tissues. To overcome obstacles, researchers have developed various innovative delivery systems, including viral vectors, lipid nanoparticles, polymer inorganic protein carriers, exosomes, antibody oligonucleotide conjugates, DNA nanostructure-based systems. These systems enhance therapeutic efficacy of nucleic drugs by improving targeting specificity, half-life vivo. In this review, we systematically discuss different types drugs, analyze major barriers encountered delivery, summarize current research progress emerging We also highlight latest advancements application for diseases, infectious cancer, brain wound healing. This review aims provide comprehensive overview drug systems' status future directions integrating nanotechnology, biomaterials science, gene editing technologies, emphasizing transformative potential precision medicine.
Language: Английский
Citations
2
Tissue Engineering and Regenerative Medicine, Journal Year: 2025, Volume and Issue: unknown
Published: Feb. 24, 2025
Language: Английский
Citations
1
International Journal of Pharmaceutics X, Journal Year: 2024, Volume and Issue: 8, P. 100283 - 100283
Published: Sept. 10, 2024
Language: Английский
Citations
6
ACS Chemical Biology, Journal Year: 2025, Volume and Issue: unknown
Published: March 13, 2025
Glioblastoma multiforme (GBM) is a WHO grade 4 glioma and the most common malignant primary brain tumor. Addressing clinical management of GBM presents an exceptionally daunting intricate challenge, particularly in overcoming blood–brain barrier (BBB) to deliver effective therapies brain. Nanotechnology-based drug delivery systems have exhibited considerable promise tackling this aggressive cancer. However, BBB remains key challenge achieving nanocarriers. Here, we optimized lipid nanoparticle (LNP) formulation (C2) modified LNP with Angiopep-2 peptide, which exhibits significant improvements penetration accumulation (about 2.23% injection dose). Using Ang-2-coupled C2 formulation, researched therapeutic effect Polo-like Kinase 1(PLK1)-targeted siRNA treat mouse model GBM. The was demonstrated significantly inhibit growth extend median survival mice (2.18-fold). This work demonstrates efficacy brain-targeted system treatment. As understanding role RNAs deepens innovative methods are continually developed refined, RNA-based could emerge as crucial breakthrough advancement tumor
Language: Английский
Citations
0
Acta Pharmaceutica Sinica B, Journal Year: 2025, Volume and Issue: unknown
Published: March 1, 2025
Language: Английский
Citations
0
Heart Failure Reviews, Journal Year: 2025, Volume and Issue: unknown
Published: March 8, 2025
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive, life-threatening disease caused by the pathological deposition of misfolded transthyretin (TTR) protein in myocardium, leading to restrictive and heart failure. While TTR stabilizers such as tafamidis acoramidis are only FDA-approved treatments, novel gene-modulating therapies emerging transformative approaches. Small interfering RNA (siRNA) antisense oligonucleotide (ASO) effectively reduce production have demonstrated promising clinical outcomes, though their use cardiac amyloidosis remains investigational. CRISPR-Cas9 represent paradigm shift, offering potential one-time treatment permanently silencing gene. Recent trials shown significant reduction stabilization biomarkers, although long-term safety efficacy require further evaluation. Despite lack direct comparisons among these modalities, emergence highlights future for ATTR-CM management. This review discusses pathogenesis ATTR-CM, mechanisms therapies, evidence, challenges, outlook advancing options.
Language: Английский
Citations
0
Pharmaceutics, Journal Year: 2025, Volume and Issue: 17(4), P. 459 - 459
Published: April 2, 2025
Background/Objectives: Lipid nanoparticles (LNPs) are leading mRNA delivery vehicles, with ionizable lipids (ILs) as their key component. However, the relationship between IL structure and LNP endogenous organ-targeting is not well understood. In this study, we developed a novel library of biodegradable ILs featuring beta-propionate linkers, which, when incorporated into four-component formulation, show excellent extrahepatic selectivity high protein expression. Methods: We explored impact structural modifications in hydrophobic chains polar-head groups while keeping linkers unchanged. vivo results were evaluated to examine how changes influence biodistribution spleen or lungs. formulations assessed for expression levels organ-specific targeting. Additionally, corona formation by best-performing LNPs was examined provide further mechanistic insights. Results: Organ targeting significantly influenced ILs, allowing precise control Branched demonstrated higher propensity targeting, group could drastically shift from lung spleen. This led identification LNPs’ zeta potential determinant properties. Notably, lipid A3T2C7, also known CP-LC-1495, displayed strong (97%) tissue (1.21 × 108 p/s). Similarly, several promising candidates spleen-targeting exceeding 1 107 p/s (selectivity >80%). Conclusions: study elucidates structure–function relationships passive delivery, highlighting fine-tuning chains, groups, surface charge (zeta potential) allows biodistribution, mechanism formation. These findings enable rational design targeted systems, enhancing therapeutic specific organs, such
Language: Английский
Citations
0
Scientific Reports, Journal Year: 2024, Volume and Issue: 14(1)
Published: July 31, 2024
Self-amplifying RNAs (saRNAs) are versatile vaccine platforms that take advantage of a viral RNA-dependent RNA polymerase (RdRp) to amplify the messenger (mRNA) an antigen interest encoded within backbone genome once inside target cell. In recent years, more saRNA vaccines have been clinically tested with hope reducing vaccination dose compared conventional mRNA approach. The use N1-methyl-pseudouridine (1mΨ), which enhances stability and reduces innate immune response triggered by RNAs, is among improvements included in current vaccines. present study, we evaluated effects this modified nucleoside on various based different viruses. results showed stages replication process were affected depending virus. For TNCL, insect virus Alphanodavirus genus, was impaired poor recognition RdRp. contrast, translation step severely abrogated coxsackievirus B3 (CVB3), member Picornaviridae family. Finally, 1mΨ Semliki forest (SFV), not detrimental vitro studies, but no advantages observed when immunogenicity vivo.
Language: Английский
Citations
1
Pharmaceutics, Journal Year: 2024, Volume and Issue: 16(11), P. 1366 - 1366
Published: Oct. 25, 2024
The COVID-19 pandemic has significantly accelerated progress in RNA-based therapeutics, particularly through the successful development and global rollout of mRNA vaccines. This review delves into transformative impact on RNA with a strong focus lipid nanoparticles (LNPs) as pivotal delivery platform. LNPs have proven to be critical enhancing stability, bioavailability, targeted mRNA, facilitating unprecedented success vaccines like those developed by Pfizer-BioNTech Moderna. Beyond vaccines, LNP technology is being explored for broader therapeutic applications, including treatments cancer, rare genetic disorders, infectious diseases. also discusses emerging systems, such polymeric viral vectors, which offer alternative strategies overcome existing challenges related immune responses, tissue-specific targeting. Additionally, we examine pandemic's influence regulatory processes, fast-tracked approvals therapies, surge research funding that spurred further innovation field. Public acceptance grown, laying groundwork future developments personalized medicine. By providing an in-depth analysis these advancements, this highlights long-term evolution therapeutics precision drug technologies.
Language: Английский
Citations
1
Cytotherapy, Journal Year: 2024, Volume and Issue: unknown
Published: Aug. 1, 2024
Language: Английский
Citations
0