{"title":"Next-Generation Nucleic Acid Delivery: A Review of Nanobiosystem Design and Applications.","authors":"Ashish Kumar Parashar, Anu Hardenia, Sunil Kumar Dwivedi, Gaurav Kant Saraogi, Shiv Hardenia","doi":"10.2174/0115665232367377250519114910","DOIUrl":null,"url":null,"abstract":"<p><p>The increasing approval of nucleic acid therapeutics has led to a significant advancement in medicines, demonstrating their potential to revolutionize the prevention and treatment of numerous diseases. However, challenges like nuclease degradation and difficult cellular delivery hinder their use as therapeutic agents. The rising demand for precise gene therapy delivery has positioned nanobiosystems as a groundbreaking solution, with their customizable properties enabling targeted and efficient delivery. Nucleic Acid therapeutics, encompassing antisense DNA, mRNA, small interfering RNA (siRNA), and microRNA (miRNA), have been rigorously investigated for their capacity to modulate gene expression. Notably, integrating these gene therapies with nanoscale delivery platforms has significantly broadened their scope, facilitating sophisticated advancements in bioanalysis, gene silencing, protein replacement therapies, and the development of vaccines. This review provides a thorough review of recent advancements in nanobiosystems for therapeutic nucleic acid delivery. We explore the unique characteristics of various nanobiosystems, including gene therapy-based delivery, nanoparticles, stimuli-responsive systems, smart nanocarriers, and extracellular vesicle-based delivery. We offer a detailed overview of their applications in nucleic acid delivery. Furthermore, we address biological barriers and strategies for the therapeutic delivery of nucleic acids. Ultimately, this review provides critical insights into the strategic development of nextgeneration delivery vectors for nucleic acid therapeutics.</p>","PeriodicalId":10798,"journal":{"name":"Current gene therapy","volume":" ","pages":""},"PeriodicalIF":3.8000,"publicationDate":"2025-05-27","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/10.2174/0115665232367377250519114910","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
引用次数: 0
Abstract
The increasing approval of nucleic acid therapeutics has led to a significant advancement in medicines, demonstrating their potential to revolutionize the prevention and treatment of numerous diseases. However, challenges like nuclease degradation and difficult cellular delivery hinder their use as therapeutic agents. The rising demand for precise gene therapy delivery has positioned nanobiosystems as a groundbreaking solution, with their customizable properties enabling targeted and efficient delivery. Nucleic Acid therapeutics, encompassing antisense DNA, mRNA, small interfering RNA (siRNA), and microRNA (miRNA), have been rigorously investigated for their capacity to modulate gene expression. Notably, integrating these gene therapies with nanoscale delivery platforms has significantly broadened their scope, facilitating sophisticated advancements in bioanalysis, gene silencing, protein replacement therapies, and the development of vaccines. This review provides a thorough review of recent advancements in nanobiosystems for therapeutic nucleic acid delivery. We explore the unique characteristics of various nanobiosystems, including gene therapy-based delivery, nanoparticles, stimuli-responsive systems, smart nanocarriers, and extracellular vesicle-based delivery. We offer a detailed overview of their applications in nucleic acid delivery. Furthermore, we address biological barriers and strategies for the therapeutic delivery of nucleic acids. Ultimately, this review provides critical insights into the strategic development of nextgeneration delivery vectors for nucleic acid therapeutics.
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.
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