{"title":"利用创新的纳米载体转化rna基因治疗,用于siRNA和miRNA的递送。","authors":"Jitendra Singh Chaudhary, Dilip Kumar Chanchal, Kuldeep Singh, Jeetendra Kumar Gupta, Divya Jain, Anubhav Dubey, Mahima Pandey, Shahbaz Khan","doi":"10.2174/0115665232355294250330002630","DOIUrl":null,"url":null,"abstract":"<p><p>The cells have been given precise instructions proprio to the regulation of gene expression by the main genesis of Ryan-based gene therapy, which has revived cancer treatment and other disorders. The difficulty of delivering small interfering RNA (siRNA) and microRNA (miRNA) to a target cell is an enormous task and is often faced by researchers due to characteristic instabilities of these carriers and their poor uptake by the cell membrane. The new developments from nanocarrier technologies offer opportunities for better effectiveness of RNA therapy for its delivery and the effectiveness of the treatment regimen. The objective of this article is to provide an overview of the existing as well as the newest developments in nanocarrier technology, particularly as related to microRNA and small interfering RNA (siRNA) delivery. Their modes of operation and their uses in gene therapies are also examined as principles of their design. We focus on several nanocarrier technologies, which have shown proof of concept in multiple disciplines such as stability, controlled release profiles, and delivery. Lipid-based nanoparticles, polymeric systems, and hybrid nanocarriers are some of the platforms that fall under this category; however, this list is not exhaustive. We also study the idea that certain nanocarriers could have multiple functionalities, which would make it possible to improve cancer treatment by simultaneously carrying chemotherapy and genes. We aim to shed light on the future of RNA-based gene therapy by providing a thorough overview of recent research in the field. This will help us understand how novel nanocarrier technologies can tackle the delivery issues.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8000,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Transforming RNA-Based Gene Therapy with Innovative Nanocarriers for siRNA and miRNA Delivery.\",\"authors\":\"Jitendra Singh Chaudhary, Dilip Kumar Chanchal, Kuldeep Singh, Jeetendra Kumar Gupta, Divya Jain, Anubhav Dubey, Mahima Pandey, Shahbaz Khan\",\"doi\":\"10.2174/0115665232355294250330002630\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>The cells have been given precise instructions proprio to the regulation of gene expression by the main genesis of Ryan-based gene therapy, which has revived cancer treatment and other disorders. The difficulty of delivering small interfering RNA (siRNA) and microRNA (miRNA) to a target cell is an enormous task and is often faced by researchers due to characteristic instabilities of these carriers and their poor uptake by the cell membrane. The new developments from nanocarrier technologies offer opportunities for better effectiveness of RNA therapy for its delivery and the effectiveness of the treatment regimen. The objective of this article is to provide an overview of the existing as well as the newest developments in nanocarrier technology, particularly as related to microRNA and small interfering RNA (siRNA) delivery. Their modes of operation and their uses in gene therapies are also examined as principles of their design. We focus on several nanocarrier technologies, which have shown proof of concept in multiple disciplines such as stability, controlled release profiles, and delivery. Lipid-based nanoparticles, polymeric systems, and hybrid nanocarriers are some of the platforms that fall under this category; however, this list is not exhaustive. We also study the idea that certain nanocarriers could have multiple functionalities, which would make it possible to improve cancer treatment by simultaneously carrying chemotherapy and genes. We aim to shed light on the future of RNA-based gene therapy by providing a thorough overview of recent research in the field. This will help us understand how novel nanocarrier technologies can tackle the delivery issues.</p>\",\"PeriodicalId\":10798,\"journal\":{\"name\":\"Current gene therapy\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":3.8000,\"publicationDate\":\"2025-04-14\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Current gene therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.2174/0115665232355294250330002630\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"GENETICS & HEREDITY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Current gene therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.2174/0115665232355294250330002630","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
Transforming RNA-Based Gene Therapy with Innovative Nanocarriers for siRNA and miRNA Delivery.
The cells have been given precise instructions proprio to the regulation of gene expression by the main genesis of Ryan-based gene therapy, which has revived cancer treatment and other disorders. The difficulty of delivering small interfering RNA (siRNA) and microRNA (miRNA) to a target cell is an enormous task and is often faced by researchers due to characteristic instabilities of these carriers and their poor uptake by the cell membrane. The new developments from nanocarrier technologies offer opportunities for better effectiveness of RNA therapy for its delivery and the effectiveness of the treatment regimen. The objective of this article is to provide an overview of the existing as well as the newest developments in nanocarrier technology, particularly as related to microRNA and small interfering RNA (siRNA) delivery. Their modes of operation and their uses in gene therapies are also examined as principles of their design. We focus on several nanocarrier technologies, which have shown proof of concept in multiple disciplines such as stability, controlled release profiles, and delivery. Lipid-based nanoparticles, polymeric systems, and hybrid nanocarriers are some of the platforms that fall under this category; however, this list is not exhaustive. We also study the idea that certain nanocarriers could have multiple functionalities, which would make it possible to improve cancer treatment by simultaneously carrying chemotherapy and genes. We aim to shed light on the future of RNA-based gene therapy by providing a thorough overview of recent research in the field. This will help us understand how novel nanocarrier technologies can tackle the delivery issues.
期刊介绍:
Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of diseases. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Current Gene Therapy publishes full-length/mini reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of diseases.
Current Gene Therapy publishes reviews and original research containing experimental data on gene and cell therapy. The journal also includes manuscripts on technological advances, ethical and regulatory considerations of gene and cell therapy. Reviews should provide the reader with a comprehensive assessment of any area of experimental biology applied to molecular medicine that is not only of significance within a particular field of gene therapy and cell therapy but also of interest to investigators in other fields. Authors are encouraged to provide their own assessment and vision for future advances. Reviews are also welcome on late breaking discoveries on which substantial literature has not yet been amassed. Such reviews provide a forum for sharply focused topics of recent experimental investigations in gene therapy primarily to make these results accessible to both clinical and basic researchers. Manuscripts containing experimental data should be original data, not previously published.