Dominika Berdecka , Stefaan C. De Smedt , Winnok H. De Vos , Kevin Braeckmans
{"title":"Non-viral delivery of RNA for therapeutic T cell engineering","authors":"Dominika Berdecka , Stefaan C. De Smedt , Winnok H. De Vos , Kevin Braeckmans","doi":"10.1016/j.addr.2024.115215","DOIUrl":"10.1016/j.addr.2024.115215","url":null,"abstract":"<div><p>Adoptive T cell transfer has shown great success in treating blood cancers, resulting in a growing number of FDA-approved therapies using chimeric antigen receptor (CAR)-engineered T cells. However, the effectiveness of this treatment for solid tumors is still not satisfactory, emphasizing the need for improved T cell engineering strategies and combination approaches. Currently, CAR T cells are mainly manufactured using gammaretroviral and lentiviral vectors due to their high transduction efficiency. However, there are concerns about their safety, the high cost of producing them in compliance with current Good Manufacturing Practices (cGMP), regulatory obstacles, and limited cargo capacity, which limit the broader use of engineered T cell therapies. To overcome these limitations, researchers have explored non-viral approaches, such as membrane permeabilization and carrier-mediated methods, as more versatile and sustainable alternatives for next-generation T cell engineering. Non-viral delivery methods can be designed to transport a wide range of molecules, including RNA, which allows for more controlled and safe modulation of T cell phenotype and function. In this review, we provide an overview of non-viral RNA delivery in adoptive T cell therapy. We first define the different types of RNA therapeutics, highlighting recent advancements in manufacturing for their therapeutic use. We then discuss the challenges associated with achieving effective RNA delivery in T cells. Next, we provide an overview of current and emerging technologies for delivering RNA into T cells. Finally, we discuss ongoing preclinical and clinical studies involving RNA-modified T cells.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139943292","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ting Lei , Zixiao Yang , Hanmei Li , Meng Qin , Huile Gao
{"title":"Interactions between nanoparticles and pathological changes of vascular in Alzheimer’s disease","authors":"Ting Lei , Zixiao Yang , Hanmei Li , Meng Qin , Huile Gao","doi":"10.1016/j.addr.2024.115219","DOIUrl":"10.1016/j.addr.2024.115219","url":null,"abstract":"<div><p>Emerging evidence suggests that vascular pathological changes play a pivotal role in the pathogenesis of Alzheimer's disease (AD). The dysfunction of the cerebral vasculature occurs in the early course of AD, characterized by alterations in vascular morphology, diminished cerebral blood flow (CBF), impairment of the neurovascular unit (NVU), vasculature inflammation, and cerebral amyloid angiopathy. Vascular dysfunction not only facilitates the influx of neurotoxic substances into the brain, triggering inflammation and immune responses but also hampers the efflux of toxic proteins such as Aβ from the brain, thereby contributing to neurodegenerative changes in AD. Furthermore, these vascular changes significantly impact drug delivery and distribution within the brain. Therefore, developing targeted delivery systems or therapeutic strategies based on vascular alterations may potentially represent a novel breakthrough in AD treatment. This review comprehensively examines various aspects of vascular alterations in AD and outlines the current interactions between nanoparticles and pathological changes of vascular.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139943328","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Hussein H. Genedy , Paul Humbert , Bilel Laoulaou , Brian Le Moal , Marion Fusellier , Catherine Passirani , Catherine Le Visage , Jérôme Guicheux , Élise Lepeltier , Johann Clouet
{"title":"MicroRNA-targeting nanomedicines for the treatment of intervertebral disc degeneration","authors":"Hussein H. Genedy , Paul Humbert , Bilel Laoulaou , Brian Le Moal , Marion Fusellier , Catherine Passirani , Catherine Le Visage , Jérôme Guicheux , Élise Lepeltier , Johann Clouet","doi":"10.1016/j.addr.2024.115214","DOIUrl":"10.1016/j.addr.2024.115214","url":null,"abstract":"<div><p>Low back pain stands as a pervasive global health concern, afflicting almost 80% of adults at some point in their lives with nearly 40% attributable to intervertebral disc degeneration (IVDD). As only symptomatic relief can be offered to patients there is a dire need for innovative treatments.<!--> <!-->Given the accumulating evidence that multiple microRNAs (miRs) are dysregulated during IVDD, they could have a huge potential against this debilitating condition. The way miRs can profoundly modulate signaling pathways and influence several cellular processes at once is particularly exciting to tackle this multifaceted disorder. However, miR delivery encounters extracellular and intracellular biological barriers. A promising technology to address this challenge is the vectorization of miRs within nanoparticles, providing both protection and enhancing their uptake within the scarce target cells of the degenerated IVD. This comprehensive review presents the diverse spectrum of miRs’ connection with IVDD and demonstrates their therapeutic potential when vectorized in nanomedicines.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139937777","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Xiaotong Li , Jiahui Zou , Zhongshan He , Yanhua Sun , Xiangrong Song , Wei He
{"title":"The interaction between particles and vascular endothelium in blood flow","authors":"Xiaotong Li , Jiahui Zou , Zhongshan He , Yanhua Sun , Xiangrong Song , Wei He","doi":"10.1016/j.addr.2024.115216","DOIUrl":"10.1016/j.addr.2024.115216","url":null,"abstract":"<div><p>Particle-based drug delivery systems have shown promising application potential to treat human diseases; however, an incomplete understanding of their interactions with vascular endothelium in blood flow prevents their inclusion into mainstream clinical applications. The flow performance of nano/micro-sized particles in the blood are disturbed by many external/internal factors, including blood constituents, particle properties, and endothelium bioactivities, affecting the fate of particles <em>in vivo</em> and therapeutic effects for diseases. This review highlights how the blood constituents, hemodynamic environment and particle properties influence the interactions and particle activities <em>in vivo</em>. Moreover, we briefly summarized the structure and functions of endothelium and simulated devices for studying particle performance under blood flow conditions. Finally, based on particle-endothelium interactions, we propose future opportunities for novel therapeutic strategies and provide solutions to challenges in particle delivery systems for accelerating their clinical translation. This review helps provoke an increasing in-depth understanding of particle-endothelium interactions and inspires more strategies that may benefit the development of particle medicine.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139917276","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jieqiong Wang , Yuanyuan Wang , Lin Zhong , Fei Yan , Hairong Zheng
{"title":"Nanoscale contrast agents: A promising tool for ultrasound imaging and therapy","authors":"Jieqiong Wang , Yuanyuan Wang , Lin Zhong , Fei Yan , Hairong Zheng","doi":"10.1016/j.addr.2024.115200","DOIUrl":"10.1016/j.addr.2024.115200","url":null,"abstract":"<div><p>Nanoscale contrast agents have emerged as a versatile platform in the field of biomedical research, offering great potential for ultrasound imaging and therapy. Various kinds of nanoscale contrast agents have been extensively investigated in preclinical experiments to satisfy diverse biomedical applications. This paper provides a comprehensive review of the structure and composition of various nanoscale contrast agents, as well as their preparation and functionalization, encompassing both chemosynthetic and biosynthetic strategies. Subsequently, we delve into recent advances in the utilization of nanoscale contrast agents in various biomedical applications, including ultrasound molecular imaging, ultrasound-mediated drug delivery, and cell acoustic manipulation. Finally, the challenges and prospects of nanoscale contrast agents are also discussed to promote the development of this innovative nanoplatform in the field of biomedicine.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139745780","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Encapsulation and immune protection for type 1 diabetes cell therapy","authors":"Sophia Kioulaphides , Andrés J. García","doi":"10.1016/j.addr.2024.115205","DOIUrl":"10.1016/j.addr.2024.115205","url":null,"abstract":"<div><p>Type 1 Diabetes (T1D) involves the autoimmune destruction of insulin-producing β-cells in the pancreas. Exogenous insulin injections are the current therapy but are user-dependent and cannot fully recapitulate physiological insulin secretion dynamics. Since the emergence of allogeneic cell therapy for T1D, the Edmonton Protocol has been the most promising immunosuppression protocol for cadaveric islet transplantation, but the lack of donor islets, poor cell engraftment, and required chronic immunosuppression have limited its application as a therapy for T1D. Encapsulation in biomaterials on the nano-, micro-, and macro-scale offers the potential to integrate islets with the host and protect them from immune responses. This method can be applied to different cell types, including cadaveric, porcine, and stem cell-derived islets, mitigating the issue of a lack of donor cells. This review covers progress in the efforts to integrate insulin-producing cells from multiple sources to T1D patients as a form of cell therapy.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139740124","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ajay S. Thatte , Margaret M. Billingsley , Drew Weissman , Jilian R. Melamed , Michael J. Mitchell
{"title":"Emerging strategies for nanomedicine in autoimmunity","authors":"Ajay S. Thatte , Margaret M. Billingsley , Drew Weissman , Jilian R. Melamed , Michael J. Mitchell","doi":"10.1016/j.addr.2024.115194","DOIUrl":"10.1016/j.addr.2024.115194","url":null,"abstract":"<div><p>Autoimmune disorders have risen to be among the most prevalent chronic diseases across the globe, affecting approximately 5–7% of the population. As autoimmune diseases steadily rise in prevalence, so do the number of potential therapeutic strategies to combat them. In recent years, fundamental research investigating autoimmune pathologies has led to the emergence of several cellular targets that provide new therapeutic opportunities. However, key challenges persist in terms of accessing and specifically combating the dysregulated, self-reactive cells while avoiding systemic immune suppression and other off-target effects. Fortunately, the continued advancement of nanomedicines may provide strategies to address these challenges and bring innovative autoimmunity therapies to the clinic. Through precise engineering and rational design, nanomedicines can possess a variety of physicochemical properties, surface modifications, and cargoes, allowing for specific targeting of therapeutics to pathological cell and organ types. These advances in nanomedicine have been demonstrated in cancer therapies and have the broad potential to advance applications in autoimmunity therapies as well. In this review, we focus on leveraging the power of nanomedicine for prevalent autoimmune disorders throughout the body. We expand on three key areas for the development of autoimmunity therapies – avoiding systemic immunosuppression, balancing interactions with the immune system, and elevating current platforms for delivering complex cargoes – and emphasize how nanomedicine-based strategies can overcome these barriers and enable the development of next-generation, clinically relevant autoimmunity therapies.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139717254","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Beatriz da Silva Gomes , Ana Cláudia Paiva-Santos , Francisco Veiga , Filipa Mascarenhas-Melo
{"title":"Beyond the adverse effects of the systemic route: Exploiting nanocarriers for the topical treatment of skin cancers","authors":"Beatriz da Silva Gomes , Ana Cláudia Paiva-Santos , Francisco Veiga , Filipa Mascarenhas-Melo","doi":"10.1016/j.addr.2024.115197","DOIUrl":"10.1016/j.addr.2024.115197","url":null,"abstract":"<div><p>Skin cancer is a heterogeneous disease that can be divided into two main groups, melanoma and nonmelanoma skin cancers. Conventional therapies for skin cancer have numerous systemic side effects and a high recurrence rate. Topical treatment is an alternative approach, but drug permeability remains a challenge. Therefore, nanocarriers appear as important nanotechnology tools that reduces both the side effects and improves clinical outcomes. This is why they are attracting growing interest. In this review, scientific articles on the use of nanocarriers for the topical treatment of skin cancer were collected. Despite the promising results of the presented nanocarriers and considering that some of them are already on the market, there is an urgent need for investment in the development of manufacturing methods, as well as of suitable toxicological and regulatory evaluations, since the conventional methods currently used to develop these nanocarriers-based products are more time-consuming and expensive than conventional products.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0169409X2400019X/pdfft?md5=0c5b62e3daa0674101043ef6939fde0a&pid=1-s2.0-S0169409X2400019X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139717253","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Extracellular vesicles are key players in mesenchymal stem cells’ dual potential to regenerate and modulate the immune system","authors":"Martijn van Griensven , Elizabeth R. Balmayor","doi":"10.1016/j.addr.2024.115203","DOIUrl":"10.1016/j.addr.2024.115203","url":null,"abstract":"<div><p>MSCs are used for treatment of inflammatory conditions or for regenerative purposes. MSCs are complete cells and allogenic transplantation is in principle possible, but mostly autologous use is preferred. In recent years, it was discovered that cells secrete extracellular vesicles. These are active budded off vesicles that carry a cargo. The cargo can be miRNA, protein, lipids etc. The extracellular vesicles can be transported through the body and fuse with target cells. Thereby, they influence the phenotype and modulate the disease. The extracellular vesicles have, like the MSCs, immunomodulatory or regenerative capacities. This review will focus on those features of extracellular vesicles and discuss their dual role. Besides the immunomodulation, the regeneration will concentrate on bone, cartilage, tendon, vessels and nerves. Current clinical trials with extracellular vesicles for immunomodulation and regeneration that started in the last five years are highlighted as well. In summary, extracellular vesicles have a great potential as disease modulating entity and treatment. Their dual characteristics need to be taken into account and often are both important for having the best effect.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0169409X24000255/pdfft?md5=8a672f38dd65752f4f6c918a1db99291&pid=1-s2.0-S0169409X24000255-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139717255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ana Vitoria Pupo Silvestrini, Milena Finazzi Morais, Bryan Wender Debiasi, Fabíola Garcia Praça, Maria Vitória Lopes Badra Bentley
{"title":"Nanotechnology strategies to address challenges in topical and cellular delivery of siRNAs in skin disease therapy","authors":"Ana Vitoria Pupo Silvestrini, Milena Finazzi Morais, Bryan Wender Debiasi, Fabíola Garcia Praça, Maria Vitória Lopes Badra Bentley","doi":"10.1016/j.addr.2024.115198","DOIUrl":"10.1016/j.addr.2024.115198","url":null,"abstract":"<div><p>Gene therapy is one of the most advanced therapies in current medicine. In particular, interference RNA-based therapy by small interfering RNA (siRNA) has gained attention in recent years as it is a highly versatile, selective and specific therapy. In dermatological conditions, topical delivery of siRNA offers numerous therapeutic advantages, mainly by inhibiting the expression of target transcripts directly in the skin. However, crossing the stratum corneum and overcoming intracellular barriers is an inherent challenge. Substantial efforts by scientists have moved towards the use of multimodal and multifunctional nanoparticles to overcome these barriers and achieve greater bioavailability in their site of action, the cytoplasm. In this review the most innovative strategies based on nanoparticle and physical methods are presented, as well as the design principles and the main factors that contribute to the performance of these systems. This review also highlights the synergistic contributions of medicine, nanotechnology, and molecular biology to advancing translational research into siRNA-based therapeutics for skin diseases.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-02-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139715719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}