Advanced drug delivery reviews最新文献_第8页

Model-informed drug development in pediatric, pregnancy and geriatric drug development: States of the art and future 儿科、妊娠和老年病药物开发中的模型信息药物开发：技术现状与未来。

IF 15.2 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-25 DOI: 10.1016/j.addr.2024.115364

Yue-E Wu , Yuan-Yuan Zheng , Qiu-Yue Li , Bu-Fan Yao , Jing Cao , Hui-Xin Liu , Guo-Xiang Hao , John van den Anker , Yi Zheng , Wei Zhao

{"title":"Model-informed drug development in pediatric, pregnancy and geriatric drug development: States of the art and future","authors":"Yue-E Wu , Yuan-Yuan Zheng , Qiu-Yue Li , Bu-Fan Yao , Jing Cao , Hui-Xin Liu , Guo-Xiang Hao , John van den Anker , Yi Zheng , Wei Zhao","doi":"10.1016/j.addr.2024.115364","DOIUrl":"10.1016/j.addr.2024.115364","url":null,"abstract":"<div><p>The challenges of drug development in pediatric, pregnant and geriatric populations are a worldwide concern shared by regulatory authorities, pharmaceutical companies, and healthcare professionals. Model-informed drug development (MIDD) can integrate and quantify real-world data of physiology, pharmacology, and disease processes by using modeling and simulation techniques to facilitate decision-making in drug development. In this article, we reviewed current MIDD policy updates, reflected on the integrity of physiological data used for MIDD and the effects of physiological changes on the drug PK, as well as summarized current MIDD strategies and applications, so as to present the state of the art of MIDD in pediatric, pregnant and geriatric populations. Some considerations are put forth for the future improvements of MIDD including refining regulatory considerations, improving the integrity of physiological data, applying the emerging technologies, and exploring the application of MIDD in new therapies like gene therapies for special populations.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115364"},"PeriodicalIF":15.2,"publicationDate":"2024-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141465513","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}

引用次数: 0

Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications 向大脑输送腺相关病毒载体：技术进步与临床应用。

IF 15.2 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-19 DOI: 10.1016/j.addr.2024.115363

Dezhuang Ye , Chinwendu Chukwu , Yaoheng Yang , Zhongtao Hu , Hong Chen

{"title":"Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications","authors":"Dezhuang Ye , Chinwendu Chukwu , Yaoheng Yang , Zhongtao Hu , Hong Chen","doi":"10.1016/j.addr.2024.115363","DOIUrl":"10.1016/j.addr.2024.115363","url":null,"abstract":"<div><p>Adeno-associated virus (AAV) vectors have emerged as a promising tool in the development of gene therapies for various neurological diseases, including Alzheimer’s disease and Parkinson’s disease. However, the blood–brain barrier (BBB) poses a significant challenge to successfully delivering AAV vectors to the brain. Strategies that can overcome the BBB to improve the AAV delivery efficiency to the brain are essential to successful brain-targeted gene therapy. This review provides an overview of existing strategies employed for AAV delivery to the brain, including direct intraparenchymal injection, intra-cerebral spinal fluid injection, intranasal delivery, and intravenous injection of BBB-permeable AAVs. Focused ultrasound has emerged as a promising technology for the noninvasive and spatially targeted delivery of AAV administered by intravenous injection. This review also summarizes each strategy’s current preclinical and clinical applications in treating neurological diseases. Moreover, this review includes a detailed discussion of the recent advances in the emerging focused ultrasound-mediated AAV delivery. Understanding the state-of-the-art of these gene delivery approaches is critical for future technology development to fulfill the great promise of AAV in neurological disease treatment.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115363"},"PeriodicalIF":15.2,"publicationDate":"2024-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141436547","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}

引用次数: 0

Cytoskeleton-modulating nanomaterials and their therapeutic potentials 细胞骨架调节纳米材料及其治疗潜力。

IF 15.2 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-19 DOI: 10.1016/j.addr.2024.115362

Jinwon Park , Yina Wu , Jung Suk Kim , Junho Byun, Jaiwoo Lee, Yu-Kyoung Oh

{"title":"Cytoskeleton-modulating nanomaterials and their therapeutic potentials","authors":"Jinwon Park , Yina Wu , Jung Suk Kim , Junho Byun, Jaiwoo Lee, Yu-Kyoung Oh","doi":"10.1016/j.addr.2024.115362","DOIUrl":"10.1016/j.addr.2024.115362","url":null,"abstract":"<div><p>The cytoskeleton, an intricate network of protein fibers within cells, plays a pivotal role in maintaining cell shape, enabling movement, and facilitating intracellular transport. Its involvement in various pathological states, ranging from cancer proliferation and metastasis to the progression of neurodegenerative disorders, underscores its potential as a target for therapeutic intervention. The exploration of nanotechnology in this realm, particularly the use of nanomaterials for cytoskeletal modulation, represents a cutting-edge approach with the promise of novel treatments. Inorganic nanomaterials, including those derived from gold, metal oxides, carbon, and black phosphorus, alongside organic variants such as peptides and proteins, are at the forefront of this research. These materials offer diverse mechanisms of action, either by directly interacting with cytoskeletal components or by influencing cellular signaling pathways that, in turn, modulate the cytoskeleton. Recent advancements have introduced magnetic field-responsive and light-responsive nanomaterials, which allow for targeted and controlled manipulation of the cytoskeleton. Such precision is crucial in minimizing off-target effects and enhancing therapeutic efficacy. This review explores the importance of research into cytoskeleton-targeting nanomaterials for developing therapeutic interventions for a range of diseases. It also addresses the progress made in this field, the challenges encountered, and future directions for using nanomaterials to modulate the cytoskeleton. The continued exploration of nanomaterials for cytoskeleton modulation holds great promise for advancing therapeutic strategies against a broad spectrum of diseases, marking a significant step forward in the intersection of nanotechnology and medicine.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115362"},"PeriodicalIF":15.2,"publicationDate":"2024-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141436548","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}

引用次数: 0

Navigating cancer therapy induced cardiotoxicity: From pathophysiology to treatment innovations 癌症治疗诱发的心脏毒性：从病理生理学到治疗创新。

IF 15.2 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-18 DOI: 10.1016/j.addr.2024.115361

Jessica Tetterton-Kellner , Brian C. Jensen , Juliane Nguyen

{"title":"Navigating cancer therapy induced cardiotoxicity: From pathophysiology to treatment innovations","authors":"Jessica Tetterton-Kellner , Brian C. Jensen , Juliane Nguyen","doi":"10.1016/j.addr.2024.115361","DOIUrl":"10.1016/j.addr.2024.115361","url":null,"abstract":"<div><p>Every year, more than a million people in the United States undergo chemotherapy or radiation therapy for cancer, as estimated by the CDC. While chemotherapy has been an instrumental tool for treating cancer, it also causes severe adverse effects. The more commonly acknowledged adverse effects include hair loss, fatigue, and nausea, but a more severe and longer lasting side effect is cardiotoxicity. Cardiotoxicity, or heart damage, is a common complication of cancer treatments. It can range from mild to severe, and it can affect some patients temporarily or others permanently, even after they are cured of cancer. Dexrazoxane is the only FDA-approved drug for treating anthracycline induced cardiotoxicity, but it also has drawbacks and adverse effects. There is no other type of chemotherapy induced cardiotoxicity that has an approved treatment option. In this review, we discuss the pathophysiology of chemotherapeutic-induced cardiotoxicity, methods and guidelines of diagnosis, methods of treatment and mitigation, and current drug delivery approaches in therapeutic development.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115361"},"PeriodicalIF":15.2,"publicationDate":"2024-06-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141431126","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}

引用次数: 0

Preface: Image-assisted organoid research and application 前言：图像辅助类器官研究与应用。

IF 15.2 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-17 DOI: 10.1016/j.addr.2024.115360

Hyuk Sang Yoo, Nathaniel S. Hwang, Kam W. Leong

引用次数: 0

Nanoassemblies designed for efficient nuclear targeting 设计用于高效核打靶的纳米组件。

IF 16.1 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-09 DOI: 10.1016/j.addr.2024.115354

Michal Skowicki , Shabnam Tarvirdipour , Manuel Kraus , Cora-Ann Schoenenberger , Cornelia G. Palivan

{"title":"Nanoassemblies designed for efficient nuclear targeting","authors":"Michal Skowicki , Shabnam Tarvirdipour , Manuel Kraus , Cora-Ann Schoenenberger , Cornelia G. Palivan","doi":"10.1016/j.addr.2024.115354","DOIUrl":"10.1016/j.addr.2024.115354","url":null,"abstract":"<div><p>One of the key aspects of coping efficiently with complex pathological conditions is delivering the desired therapeutic compounds with precision in both space and time. Therefore, the focus on nuclear-targeted delivery systems has emerged as a promising strategy with high potential, particularly in gene therapy and cancer treatment. Here, we explore the design of supramolecular nanoassemblies as vehicles to deliver specific compounds to the nucleus, with the special focus on polymer and peptide-based carriers that expose nuclear localization signals. Such nanoassemblies aim at maximizing the concentration of genetic and therapeutic agents within the nucleus, thereby optimizing treatment outcomes while minimizing off-target effects. A complex scenario of conditions, including cellular uptake, endosomal escape, and nuclear translocation, requires fine tuning of the nanocarriers’ properties. First, we introduce the principles of nuclear import and the role of nuclear pore complexes that reveal strategies for targeting nanosystems to the nucleus. Then, we provide an overview of cargoes that rely on nuclear localization for optimal activity as their integrity and accumulation are crucial parameters to consider when designing a suitable delivery system. Considering that they are in their early stages of research, we present various cargo-loaded peptide- and polymer nanoassemblies that promote nuclear targeting, emphasizing their potential to enhance therapeutic response. Finally, we briefly discuss further advancements for more precise and effective nuclear delivery.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115354"},"PeriodicalIF":16.1,"publicationDate":"2024-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0169409X24001765/pdfft?md5=4da3d0e8d1d1c8fb9b5da3be9f7743bb&pid=1-s2.0-S0169409X24001765-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141299749","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}

引用次数: 0

Delivery of nucleic acid based genome editing platforms via lipid nanoparticles: Clinical applications 通过脂质纳米颗粒传递基于核酸的基因组编辑平台：临床应用。

IF 16.1 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-08 DOI: 10.1016/j.addr.2024.115359

Razan Masarwy , Lior Stotsky-Oterin , Aviad Elisha , Inbal Hazan-Halevy , Dan Peer

{"title":"Delivery of nucleic acid based genome editing platforms via lipid nanoparticles: Clinical applications","authors":"Razan Masarwy , Lior Stotsky-Oterin , Aviad Elisha , Inbal Hazan-Halevy , Dan Peer","doi":"10.1016/j.addr.2024.115359","DOIUrl":"10.1016/j.addr.2024.115359","url":null,"abstract":"<div><p>CRISPR/Cas technology presents a promising approach for treating a wide range of diseases, including cancer and genetic disorders. Despite its potential, the translation of CRISPR/Cas into effective in-vivo gene therapy encounters challenges, primarily due to the need for safe and efficient delivery mechanisms. Lipid nanoparticles (LNPs), FDA-approved for RNA delivery, show potential for delivering also CRISPR/Cas, offering the capability to efficiently encapsulate large mRNA molecules with single guide RNAs. However, achieving precise targeting in-vivo remains a significant obstacle, necessitating further research into optimizing LNP formulations. Strategies to enhance specificity, such as modifying LNP structures and incorporating targeting ligands, are explored to improve organ and cell type targeting. Furthermore, the development of base and prime editing technology presents a potential breakthrough, offering precise modifications without generating double-strand breaks (DSBs). Prime editing, particularly when delivered via targeted LNPs, holds promise for treating diverse diseases safely and precisely. This review assesses both the progress made and the persistent challenges faced in using LNP-encapsulated CRISPR-based technologies for therapeutic purposes, with a particular focus on clinical translation.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115359"},"PeriodicalIF":16.1,"publicationDate":"2024-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141299748","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}

引用次数: 0

Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads 探索细胞衍生囊泡瞬时传递基因编辑有效载荷的潜力。

IF 15.2 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-06 DOI: 10.1016/j.addr.2024.115346

Kevin Leandro , David Rufino-Ramos , Koen Breyne , Emilio Di Ianni , Sara M. Lopes , Rui Jorge Nobre , Benjamin P. Kleinstiver , Pedro R.L. Perdigão , Xandra O. Breakefield , Luís Pereira de Almeida

{"title":"Exploring the potential of cell-derived vesicles for transient delivery of gene editing payloads","authors":"Kevin Leandro , David Rufino-Ramos , Koen Breyne , Emilio Di Ianni , Sara M. Lopes , Rui Jorge Nobre , Benjamin P. Kleinstiver , Pedro R.L. Perdigão , Xandra O. Breakefield , Luís Pereira de Almeida","doi":"10.1016/j.addr.2024.115346","DOIUrl":"10.1016/j.addr.2024.115346","url":null,"abstract":"<div><p>Gene editing technologies have the potential to correct genetic disorders by modifying, inserting, or deleting specific DNA sequences or genes, paving the way for a new class of genetic therapies. While gene editing tools continue to be improved to increase their precision and efficiency, the limited efficacy of <em>in vivo</em> delivery remains a major hurdle for clinical use. An ideal delivery vehicle should be able to target a sufficient number of diseased cells in a transient time window to maximize on-target editing and mitigate off-target events and immunogenicity.</p><p>Here, we review major advances in novel delivery platforms based on cell-derived vesicles − extracellular vesicles and virus-like particles − for transient delivery of gene editing payloads. We discuss major findings regarding packaging, <em>in vivo</em> biodistribution, therapeutic efficacy, and safety concerns of cell-derived vesicles delivery of gene editing cargos and their potential for clinical translation.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115346"},"PeriodicalIF":15.2,"publicationDate":"2024-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141287593","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}

引用次数: 0

In vivo deposition of poorly soluble drugs 难溶性药物的体内沉积。

IF 16.1 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-06 DOI: 10.1016/j.addr.2024.115358

Zhaohuan Lou , Chaofeng Mu , Clairissa D. Corpstein , Tonglei Li

{"title":"In vivo deposition of poorly soluble drugs","authors":"Zhaohuan Lou , Chaofeng Mu , Clairissa D. Corpstein , Tonglei Li","doi":"10.1016/j.addr.2024.115358","DOIUrl":"10.1016/j.addr.2024.115358","url":null,"abstract":"<div><p>Administered drug molecules, whether dissolved or solubilized, have the potential to precipitate and accumulate as solid forms in tissues and cells within the body. This phase transition can significantly impact the pharmacokinetics of treatment. It is thus crucial to gain an understanding of how drug solubility/permeability, drug formulations and routes of administration affect <em>in vivo</em> behaviors of drug deposition. This review examines literature reports on the drug deposition in tissues and cells of poorly water-soluble drugs, as well as underlying physical mechanisms that lead to precipitation. Our work particularly highlights drug deposition in macrophages and the subcellular fate of precipitated drugs. We also propose a tissue permeability-based classification framework to evaluate precipitation potentials of poorly soluble drugs in major organs and tissues. The impact on pharmacokinetics is further discussed and needs to be considered in developing drug delivery systems. Finally, bioimaging techniques that are used to examine aggregated states and the intracellular trafficking of absorbed drugs are summarized.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115358"},"PeriodicalIF":16.1,"publicationDate":"2024-06-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141293001","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}

引用次数: 0

Mitochondrial endogenous substance transport-inspired nanomaterials for mitochondria-targeted gene delivery 用于线粒体靶向基因递送的线粒体内源性物质转运纳米材料。

IF 16.1 1区医学

Advanced drug delivery reviews Pub Date : 2024-06-05 DOI: 10.1016/j.addr.2024.115355

Yi Wang , Jing-Song Yang , Min Zhao , Jia-Qi Chen , Hai-Xin Xie , Hao-Yuan Yu , Na-Hui Liu , Zi-Juan Yi , Hui-Lin Liang , Lei Xing , Hu-Lin Jiang

{"title":"Mitochondrial endogenous substance transport-inspired nanomaterials for mitochondria-targeted gene delivery","authors":"Yi Wang , Jing-Song Yang , Min Zhao , Jia-Qi Chen , Hai-Xin Xie , Hao-Yuan Yu , Na-Hui Liu , Zi-Juan Yi , Hui-Lin Liang , Lei Xing , Hu-Lin Jiang","doi":"10.1016/j.addr.2024.115355","DOIUrl":"10.1016/j.addr.2024.115355","url":null,"abstract":"<div><p>Mitochondrial genome (mtDNA) independent of nuclear gene is a set of double-stranded circular DNA that encodes 13 proteins, 2 ribosomal RNAs and 22 mitochondrial transfer RNAs, all of which play vital roles in functions as well as behaviors of mitochondria. Mutations in mtDNA result in various mitochondrial disorders without available cures. However, the manipulation of mtDNA via the mitochondria-targeted gene delivery faces formidable barriers, particularly owing to the mitochondrial double membrane. Given the fact that there are various transport channels on the mitochondrial membrane used to transfer a variety of endogenous substances to maintain the normal functions of mitochondria, mitochondrial endogenous substance transport-inspired nanomaterials have been proposed for mitochondria-targeted gene delivery. In this review, we summarize mitochondria-targeted gene delivery systems based on different mitochondrial endogenous substance transport pathways. These are categorized into mitochondrial steroid hormones import pathways-inspired nanomaterials, protein import pathways-inspired nanomaterials and other mitochondria-targeted gene delivery nanomaterials. We also review the applications and challenges involved in current mitochondrial gene editing systems. This review delves into the approaches of mitochondria-targeted gene delivery, providing details on the design of mitochondria-targeted delivery systems and the limitations regarding the various technologies. Despite the progress in this field is currently slow, the ongoing exploration of mitochondrial endogenous substance transport and mitochondrial biological phenomena may act as a crucial breakthrough in the targeted delivery of gene into mitochondria and even the manipulation of mtDNA.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":"211 ","pages":"Article 115355"},"PeriodicalIF":16.1,"publicationDate":"2024-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141287594","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}

引用次数: 0