3 Biotech, Год журнала: 2024, Номер 15(1)
Опубликована: Дек. 19, 2024
Язык: Английский
International Journal of Nanomedicine, Год журнала: 2025, Номер Volume 20, С. 25 - 52
Опубликована: Янв. 1, 2025
Abstract: The effective clinical translation of messenger RNA (mRNA), small interfering (siRNA), and microRNA (miRNA) for therapeutic purposes hinges on the development efficient delivery systems. Key challenges include their susceptibility to degradation, limited cellular uptake, inefficient intracellular release. Polymeric drug conjugates (PDCs) offer a promising solution, combining benefits polymeric carriers agents targeted treatment. This comprehensive review explores nucleic acid therapeutics, focusing conjugates. It investigates how these address obstacles, enhance systemic circulation, reduce immunogenicity, provide controlled release, improving safety profiles. delves into conjugation strategies, preparation methods, various classes PDCs, as well strategic design, highlighting role in delivery. Applications PDCs treating diseases such cancer, immune disorders, fibrosis are also discussed. Despite significant advancements, adoption persist. concludes with insights future directions this transformative technology, underscoring potential advance acid-based therapies combat infectious significantly. Keywords: polymer conjugates,
Язык: Английский
Процитировано
1
Frontiers in Chemistry, Год журнала: 2023, Номер 11
Опубликована: Сен. 28, 2023
The use of biomaterials in delivering CRISPR/Cas9 for gene therapy infectious diseases holds tremendous potential. This innovative approach combines the advantages with protective properties biomaterials, enabling accurate and efficient editing while enhancing safety. Biomaterials play a vital role shielding components, such as lipid nanoparticles or viral vectors, from immunological processes degradation, extending their effectiveness. By utilizing flexibility tailored systems can be designed to address specific genetic diseases, paving way personalized therapeutics. Furthermore, this delivery method offers promising avenues combating illnesses by precisely modifying pathogen genomes, reducing pathogenicity. facilitate site-specific modifications, ensuring effective infected cells minimizing off-target effects. However, challenges remain, including optimizing efficiency, effects, long-term safety, establishing scalable production techniques. Thorough research, pre-clinical investigations, rigorous safety evaluations are imperative successful translation laboratory clinical applications. In review, we discussed how using revolutionizes disease treatment, offering precise safe capabilities potential significantly improve human health quality life.
Язык: Английский
Процитировано
21
Micromachines, Год журнала: 2024, Номер 15(4), С. 533 - 533
Опубликована: Апрель 15, 2024
The development of biomaterials for protein delivery is an emerging field that spans materials science, bioengineering, and medicine. In this review, we highlight the immense potential protein-delivering as therapeutic options discuss multifaceted challenges inherent to field. We address current advancements approaches in leverage stimuli-responsive materials, harness advanced fabrication techniques like 3D printing, integrate nanotechnologies greater targeting improved stability, efficacy, tolerability profiles. also demand highly complex systems maintain structural integrity functionality payload. Finally, barriers clinical translation, such biocompatibility, immunogenicity, achieving reliable controlled release, efficient targeted delivery, stability issues, scalability production, navigating regulatory landscape materials. Overall, review summarizes insights from a survey literature sheds light on interplay between innovation practical implementation delivery.
Язык: Английский
Процитировано
7
Theranostics, Год журнала: 2024, Номер 14(7), С. 2777 - 2793
Опубликована: Янв. 1, 2024
Small extracellular vesicles (sEVs) are naturally occurring that have the potential to be manipulated become promising drug delivery vehicles for on-demand in vitro and vivo gene editing.Here, we developed modular safeEXO platform, a prototype sEV vehicle is mostly devoid of endogenous RNA can efficaciously deliver ribonucleoprotein (RNP) complexes their intended intracellular targets manifested by downstream biologic activity.We also successfully engineered producer cells produce contain Cas9 (safeEXO-CAS) effectively efficient (RNP)-mediated CRISPR genome editing machinery organs or diseased vivo.We confirmed safeEXO-CAS sEVs could co-deliver ssDNA, sgRNA siRNA, mediate insertion dose-dependent manner.We demonstrated target engineering express tissue-specific moiety, integrin alpha-6 (safeEXO-CAS-ITGA6), which increased uptake lung epithelial tested ability safeEXO-CAS-ITGA6 loaded with EMX1 sgRNAs induce lung-targeted mice, significant lungs no signs morbidity detectable changes immune cell populations.Our results demonstrate our platform represents targetable, safe, efficacious nucleic acid-based therapeutics reach targets.Furthermore, genetically containing more RNP-mediated editing.This has improve current therapies increase landscape treatment various human diseases using RNAi approaches.
Язык: Английский
Процитировано
6
Journal of Nanoparticle Research, Год журнала: 2024, Номер 26(6)
Опубликована: Июнь 1, 2024
Язык: Английский
Процитировано
6
Cellular & Molecular Biology Letters, Год журнала: 2023, Номер 28(1)
Опубликована: Сен. 28, 2023
Abstract Osteoarthritis (OA), a common joint disorder with articular cartilage degradation as the main pathological change, is major source of pain and disability worldwide. Despite current treatments, overall treatment outcome unsatisfactory. Thus, patients severe OA often require replacement surgery. In recent years, mesenchymal stem cells (MSCs) have emerged promising therapeutic option for preclinical clinical palliation OA. MSC-derived exosomes (MSC-Exos) carrying bioactive molecules parental cells, including non-coding RNAs (ncRNAs) proteins, demonstrated significant impact on modulation various physiological behaviors in cavity, making them candidates cell-free therapy This review provides comprehensive overview biosynthesis composition MSC-Exos their mechanisms action We also discussed potential tool modulating intercellular communication Additionally, we explored bioengineering approaches to enhance MSC-Exos’ potential, which may help overcome challenges achieve clinically meaningful therapies.
Язык: Английский
Процитировано
14
Biomaterials Science, Год журнала: 2023, Номер 11(15), С. 5078 - 5094
Опубликована: Янв. 1, 2023
Stimulus-responsive polymer delivery vectors with modifiable architectures can load and deliver CRISPR gene editing systems in a controlled manner, which improves the effectiveness of towards potential clinical disease treatment.
Язык: Английский
Процитировано
12
Chemical Communications, Год журнала: 2024, Номер 60(23), С. 3098 - 3117
Опубликована: Янв. 1, 2024
DNA nanostructures are easy to design and construct, have good biocompatibility, show great potential in biosensing drug delivery. Numerous distinctive versatile been developed explored for biomedical applications. In addition that completely assembled from DNA, composite obtained by combining with other organic or inorganic materials also widely used related research. The CRISPR/Cas system has attracted attention as a powerful gene editing technology is diagnosis. Many researchers committed exploring new possibilities systems. These explorations provide support the development of detection methods cargo delivery pathways, inspiration improving relevant platforms, further expand application scope This paper mainly reviews principles applications combined based on types nanostructures. Finally, status, challenges prospects summarized. It expected this review will enable better understand current state field insights into systems
Язык: Английский
Процитировано
4
Journal of Drug Delivery Science and Technology, Год журнала: 2024, Номер 95, С. 105607 - 105607
Опубликована: Март 22, 2024
Язык: Английский
Процитировано
4
Communications Materials, Год журнала: 2025, Номер 6(1)
Опубликована: Фев. 22, 2025
Язык: Английский
Процитировано
0