{"title":"Recent progress on nanosystems for nucleic acid delivery","authors":"Shanka Walia and Mohit J. Mehta","doi":"10.1039/D4PM00009A","DOIUrl":null,"url":null,"abstract":"<p >Nucleic acid (NA) based therapeutics have witnessed tremendous progress and breakthroughs in treating pathological conditions, including viral infections, neurological disorders, genetic diseases, and metabolic disorders. NAs such as plasmid DNA (pDNA), short interfering RNA (siRNA), microRNA (miRNA), and antisense oligonucleotides (ASOs) can be modified to revolutionize personalized medicine. Despite the great potential of NA-based therapeutics, their clinical transformation is significantly hampered by instability, degradation, and inefficient delivery to the targeted site in the <em>in vivo</em> system. Lipid-based delivery systems hold great potential to overcome these shortcomings to enhance the delivery and bioavailability, improve stability, and increase the therapeutic effect of the NAs by delivering them to the active site. This review emphasized various nucleic acid-based therapeutics and their enhanced and improved delivery using different nanocarriers. Ultimately, the importance of lipid-based nanocarriers for delivering NAs is discussed and provides perspective in this field.</p>","PeriodicalId":101141,"journal":{"name":"RSC Pharmaceutics","volume":" 4","pages":" 645-674"},"PeriodicalIF":0.0000,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://pubs.rsc.org/en/content/articlepdf/2024/pm/d4pm00009a?page=search","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"RSC Pharmaceutics","FirstCategoryId":"1085","ListUrlMain":"https://pubs.rsc.org/en/content/articlelanding/2024/pm/d4pm00009a","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
Abstract
Nucleic acid (NA) based therapeutics have witnessed tremendous progress and breakthroughs in treating pathological conditions, including viral infections, neurological disorders, genetic diseases, and metabolic disorders. NAs such as plasmid DNA (pDNA), short interfering RNA (siRNA), microRNA (miRNA), and antisense oligonucleotides (ASOs) can be modified to revolutionize personalized medicine. Despite the great potential of NA-based therapeutics, their clinical transformation is significantly hampered by instability, degradation, and inefficient delivery to the targeted site in the in vivo system. Lipid-based delivery systems hold great potential to overcome these shortcomings to enhance the delivery and bioavailability, improve stability, and increase the therapeutic effect of the NAs by delivering them to the active site. This review emphasized various nucleic acid-based therapeutics and their enhanced and improved delivery using different nanocarriers. Ultimately, the importance of lipid-based nanocarriers for delivering NAs is discussed and provides perspective in this field.
基于核酸(NA)的疗法在治疗病毒感染、神经系统疾病、遗传疾病和代谢紊乱等病症方面取得了巨大进展和突破。质粒 DNA (pDNA)、短干扰 RNA (siRNA)、微 RNA (miRNA) 和反义寡核苷酸 (ASO) 等 NA 经改良后可彻底改变个性化医疗。尽管基于 NA 的疗法潜力巨大,但由于其不稳定性、降解性以及在体内系统中向靶点递送的效率低下,其临床转化受到严重阻碍。基于脂质的递送系统具有克服这些缺点的巨大潜力,可提高递送和生物利用度,改善稳定性,并通过将 NA 递送到活性位点来提高其治疗效果。本综述强调了各种基于核酸的治疗方法,以及利用不同的纳米载体增强和改善它们的递送。最后,还讨论了脂质纳米载体在递送 NAs 方面的重要性,为这一领域提供了新的视角。