{"title":"A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines","authors":"Namratha Turuvekere Vittala Murthy , Kseniia Vlasova , Jonas Renner , Antony Jozic , Gaurav Sahay","doi":"10.1016/j.addr.2024.115305","DOIUrl":"https://doi.org/10.1016/j.addr.2024.115305","url":null,"abstract":"<div><p>Cystic fibrosis (CF) is a complex genetic respiratory disorder that necessitates innovative gene delivery strategies to address the mutations in the gene. This review delves into the promises and challenges of non-viral gene delivery for CF therapy and explores strategies to overcome these hurdles. Several emerging technologies and nucleic acid cargos for CF gene therapy are discussed. Novel formulation approaches including lipid and polymeric nanoparticles promise enhanced delivery through the CF mucus barrier, augmenting the potential of non-viral strategies. Additionally, safety considerations and regulatory perspectives play a crucial role in navigating the path toward clinical translation of gene therapy.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140605447","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}
Yisi Tang , Bao Liu , Yuting Zhang , Yuling Liu , Yongzhuo Huang , Wufa Fan
{"title":"Interactions between nanoparticles and lymphatic systems: Mechanisms and applications in drug delivery","authors":"Yisi Tang , Bao Liu , Yuting Zhang , Yuling Liu , Yongzhuo Huang , Wufa Fan","doi":"10.1016/j.addr.2024.115304","DOIUrl":"10.1016/j.addr.2024.115304","url":null,"abstract":"<div><p>The lymphatic system has garnered significant attention in drug delivery research due to the advantages it offers, such as enhancing systemic exposure and enabling lymph node targeting for nanomedicines via the lymphatic delivery route. The journey of drug carriers involves transport from the administration site to the lymphatic vessels, traversing the lymph before entering the bloodstream or targeting specific lymph nodes. However, the anatomical and physiological barriers of the lymphatic system play a pivotal role in influencing the behavior and efficiency of carriers. To expedite research and subsequent clinical translation, this review begins by introducing the composition and classification of the lymphatic system. Subsequently, we explore the routes and mechanisms through which nanoparticles enter lymphatic vessels and lymph nodes. The review further delves into the interactions between nanomedicine and body fluids at the administration site or within lymphatic vessels. Finally, we provide a comprehensive overview of recent advancements in lymphatic delivery systems, addressing the challenges and opportunities inherent in current systems for delivering macromolecules and vaccines.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140542240","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}
Jacob D. Bonadio, Ghazal Bashiri, Patrick Halligan, Michael Kegel, Fatima Ahmed, Karin Wang
{"title":"Delivery technologies for therapeutic targeting of fibronectin in autoimmunity and fibrosis applications","authors":"Jacob D. Bonadio, Ghazal Bashiri, Patrick Halligan, Michael Kegel, Fatima Ahmed, Karin Wang","doi":"10.1016/j.addr.2024.115303","DOIUrl":"https://doi.org/10.1016/j.addr.2024.115303","url":null,"abstract":"<div><p>Fibronectin (FN) is a critical component of the extracellular matrix (ECM) contributing to various physiological processes, including tissue repair and immune response regulation. FN regulates various cellular functions such as adhesion, proliferation, migration, differentiation, and cytokine release. Alterations in FN expression, deposition, and molecular structure can profoundly impact its interaction with other ECM proteins, growth factors, cells, and associated signaling pathways, thus influencing the progress of diseases such as fibrosis and autoimmune disorders. Therefore, developing therapeutics that directly target FN or its interaction with cells and other ECM components can be an intriguing approach to address autoimmune and fibrosis pathogenesis.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-04-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140649560","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}
Paula Gil-Cabrerizo , Teresa Simon-Yarza , Elisa Garbayo , María J. Blanco Prieto
{"title":"Navigating the landscape of RNA delivery systems in cardiovascular disease therapeutics","authors":"Paula Gil-Cabrerizo , Teresa Simon-Yarza , Elisa Garbayo , María J. Blanco Prieto","doi":"10.1016/j.addr.2024.115302","DOIUrl":"https://doi.org/10.1016/j.addr.2024.115302","url":null,"abstract":"<div><p>Cardiovascular diseases (CVDs) stand as the leading cause of death worldwide, posing a significant global health challenge. Consequently, the development of innovative therapeutic strategies to enhance CVDs treatment is imperative. RNA-based therapies, encompassing non-coding RNAs, mRNA, aptamers, and CRISPR/Cas9 technology, have emerged as promising tools for addressing CVDs. However, inherent challenges associated with RNA, such as poor cellular uptake, susceptibility to RNase degradation, and capture by the reticuloendothelial system, underscore the necessity of combining these therapies with effective drug delivery systems.</p><p>Various non-viral delivery systems, including extracellular vesicles, lipid-based carriers, polymeric and inorganic nanoparticles, as well as hydrogels, have shown promise in enhancing the efficacy of RNA therapeutics. In this review, we offer an overview of the most relevant RNA-based therapeutic strategies explored for addressing CVDs and emphasize the pivotal role of delivery systems in augmenting their effectiveness. Additionally, we discuss the current status of these therapies and the challenges that hinder their clinical translation.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0169409X24001248/pdfft?md5=b7d72182b1c54ed96276ba384add1421&pid=1-s2.0-S0169409X24001248-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140533311","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}
Neil Mathias , Sylvain Huille , Marie Picci , Robert P. Mahoney , Ronald J. Pettis , Brian Case , Bernhard Helk , David Kang , Ronak Shah , Junchi Ma , Deep Bhattacharya , Yogita Krishnamachari , Dany Doucet , Nathalie Maksimovikj , Sahab Babaee , Patrick Garidel , Reza Esfandiary , Rajesh Gandhi
{"title":"Towards more tolerable subcutaneous administration: Review of contributing factors for improving combination product design","authors":"Neil Mathias , Sylvain Huille , Marie Picci , Robert P. Mahoney , Ronald J. Pettis , Brian Case , Bernhard Helk , David Kang , Ronak Shah , Junchi Ma , Deep Bhattacharya , Yogita Krishnamachari , Dany Doucet , Nathalie Maksimovikj , Sahab Babaee , Patrick Garidel , Reza Esfandiary , Rajesh Gandhi","doi":"10.1016/j.addr.2024.115301","DOIUrl":"https://doi.org/10.1016/j.addr.2024.115301","url":null,"abstract":"<div><p>Subcutaneous (SC) injections can be associated with local pain and discomfort that is subjective and may affect treatment adherence and overall patient experience. With innovations increasingly focused on finding ways to deliver higher doses and volumes (≥2 mL), there is a need to better understand the multiple intertwined factors that influence pain upon SC injection. As a priority for the SC Drug Development & Delivery Consortium, this manuscript provides a comprehensive review of known attributes from published literature that contribute to pain/discomfort upon SC injection from three perspectives: (1) device and delivery factors that cause physical pain, (2) formulation factors that trigger pain responses, and (3) human factors impacting pain perception. Leveraging the Consortium’s collective expertise, we provide an assessment of the comparative and interdependent factors likely to impact SC injection pain. In addition, we offer expert insights and future perspectives to fill identified gaps in knowledge to help advance the development of patient-centric and well tolerated high-dose/high-volume SC drug delivery solutions.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140639372","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}
Fabiola A. Chapa-Villarreal , Madeleine Stephens , Rachel Pavlicin , Micaela Beussman , Nicholas A. Peppas
{"title":"Therapeutic delivery systems for rheumatoid arthritis based on hydrogel carriers","authors":"Fabiola A. Chapa-Villarreal , Madeleine Stephens , Rachel Pavlicin , Micaela Beussman , Nicholas A. Peppas","doi":"10.1016/j.addr.2024.115300","DOIUrl":"10.1016/j.addr.2024.115300","url":null,"abstract":"<div><p>Rheumatoid arthritis (RA) is an autoimmune disease suffered by millions of people worldwide. It can significantly affect the patient’s quality of life by damaging not only the joints but also organs such as the lungs and the heart. RA is normally treated using nonsteroidal anti-inflammatory drugs (NSAIDs), glucocorticoids, disease-modifying antirheumatic drugs (DMARDs), and biologics. These active agents often cause side effects and offer low efficacy due to their lack of specificity and limited retention time. In an attempt to improve RA treatments, hydrogel-based systems have been proposed as drug delivery carriers. Due to their exceptional adaptability and biocompatibility, hydrogels have the potential of enhancing the delivery of RA therapy through different administration routes in an efficient and effective manner. In this review, we explore the application of hydrogel systems as potential carriers in RA treatment. Additionally, we discuss recent work in the field and highlight the required hydrogel properties, depending on the administration route. The outstanding potential of hydrogel systems as carriers for RA was demonstrated; however, there is extensive research yet to be done to improve available treatments for RA.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-03-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140317557","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}
Petya G. Popova , Sunny P. Chen , Suiyang Liao , Manish Sadarangani , Anna K. Blakney
{"title":"Clinical perspective on topical vaccination strategies","authors":"Petya G. Popova , Sunny P. Chen , Suiyang Liao , Manish Sadarangani , Anna K. Blakney","doi":"10.1016/j.addr.2024.115292","DOIUrl":"10.1016/j.addr.2024.115292","url":null,"abstract":"<div><p>Vaccination is one of the most successful measures in modern medicine to combat diseases, especially infectious diseases, and saves millions of lives every year. Vaccine design and development remains critical and involves many aspects, including the choice of platform, antigen, adjuvant, and route of administration. Topical vaccination, defined herein as the introduction of a vaccine to any of the three layers of the human skin, has attracted interest in recent years as an alternative vaccination approach to the conventional intramuscular administration because of its potential to be needle-free and induce a superior immune response against pathogens. In this review, we describe recent progress in developing topical vaccines, highlight progress in the development of delivery technologies for topical vaccines, discuss potential factors that might impact the topical vaccine efficacy, and provide an overview of the current clinical landscape of topical vaccines.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-03-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0169409X24001145/pdfft?md5=449b741c00ad281bb70eb2cfad9bce6e&pid=1-s2.0-S0169409X24001145-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140206101","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}
Josefina Piñón Hofbauer , Christina Guttmann-Gruber , Verena Wally , Anshu Sharma , Iris K. Gratz , Ulrich Koller
{"title":"Challenges and progress related to gene editing in rare skin diseases","authors":"Josefina Piñón Hofbauer , Christina Guttmann-Gruber , Verena Wally , Anshu Sharma , Iris K. Gratz , Ulrich Koller","doi":"10.1016/j.addr.2024.115294","DOIUrl":"10.1016/j.addr.2024.115294","url":null,"abstract":"<div><p>Genodermatoses represent a large group of inherited skin disorders encompassing clinically-heterogeneous conditions that manifest in the skin and other organs. Depending on disease variant, associated clinical manifestations and secondary complications can severely impact patients’ quality of life and currently available treatments are transient and not curative. Multiple emerging approaches using CRISPR-based technologies offer promising prospects for therapy. Here, we explore current advances and challenges related to gene editing in rare skin diseases, including different strategies tailored to mutation type and structural organization of the affected gene, considerations for <em>in vivo</em> and <em>ex vivo</em> applications, the critical issue of delivery into the skin, and immune aspects of therapy. Against the backdrop of a landmark FDA approval for the first re-dosable gene replacement therapy for a rare genetic skin disorder, gene editing approaches are inching closer to the clinics and the possibility of a local permanent cure for patients affected by these disorders.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0169409X24001169/pdfft?md5=a335309f305324e00e9ff9132baf2b18&pid=1-s2.0-S0169409X24001169-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140287948","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":"Bridging the gap between testing and clinics exploring alternative pre-clinical models in melanoma research","authors":"Juliana Viegas , Bruno Sarmento","doi":"10.1016/j.addr.2024.115295","DOIUrl":"10.1016/j.addr.2024.115295","url":null,"abstract":"<div><p>Melanoma, the deadliest form of skin cancer, poses a significant clinical challenge for the development of effective treatments. Conventional <em>in vivo</em> animal studies have shown limited translational relevance to humans, raising strength to pre-clinical models for melanoma research. This review provides an in-depth analysis of alternative pre-clinical models including <em>in vitro</em> and <em>ex vivo</em> platforms such as reconstructed skin, spheroids, organoids, organotypic models, skin-on-a-chip, and bioprinting. Through a comprehensive analysis, the specific attributes, advantages, and limitations of each model are elucidated. It discusses the points related to the uniqueness advantages, from capturing complex interactions between melanoma cells and their microenvironment to enabling high-throughput drug screening and personalized medicine approaches. This review is structured covering firstly the roadmap to identify the co-occurrence of discovering new melanoma treatments and the development of its models, secondly it covers a comparative between the most used models followed by a section discussing each of them: the <em>in vitro</em> and <em>ex vivo</em> models. It intends to serve as an asset for researchers of melanoma field and clinicians involved in melanoma therapy, offering insights into the diverse preclinical models available for optimizing their integration into the translational pipeline.</p></div>","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-03-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0169409X24001170/pdfft?md5=55953a4192df28308f7c0297bf5b6ac7&pid=1-s2.0-S0169409X24001170-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140287947","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":"Emerging Voices in Drug Delivery – Harnessing and Modulating Complex Biological Systems (Issue 2)","authors":"Shawn C. Owen, Juliane Nguyen","doi":"10.1016/j.addr.2024.115293","DOIUrl":"10.1016/j.addr.2024.115293","url":null,"abstract":"","PeriodicalId":7254,"journal":{"name":"Advanced drug delivery reviews","volume":null,"pages":null},"PeriodicalIF":16.1,"publicationDate":"2024-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140193083","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}