摘要
背景:基因治疗由于其在医学领域的巨大应用潜力,近年来受到越来越多的关注。它可以被定义为基因或遗传物质(DNA,RNA,使用寡核苷酸)来治疗或预防疾病的状态,一般一个geneticbased一。 应用:其他应用,如治疗病毒,细菌或寄生虫感染或疫苗的开发也越来越有兴趣。有效的基因治疗主要依赖于高度不稳定的遗传物质达到治疗靶点的能力。为此,设计并广泛研究了不同的输送系统。纳米颗粒提供了广泛的可能性,在设计,正在准备使用生物相容性和生物可降解的赋形剂,因此通常被认为是安全的。 结论:由于这种特殊的生物屏障的复杂性,遗传物质的口服给药也是一个很大的挑战。在粒子设计过程中,必须特别注意由于屏障造成的基因传递的内在危害。粒子设计也允许靶向胃肠道的特定部位。固体脂质纳米粒已广泛应用于口服给药领域,也通过其他给药途径进行基因传递,但在口服基因传递方面尚未得到研究。在这篇手稿中,设计考虑和颗粒细胞相互作用机制将被广泛审查,侧重于口服途径,以鼓励科学界探索这些宝贵的载体口服基因的交付。
关键词: 基因治疗,脂质纳米粒,基因传递,口服途径,口服,控释
Current Gene Therapy
Title:Lipid Nanoparticles as Potential Gene Therapeutic Delivery Systems for Oral Administration
Volume: 17 Issue: 2
关键词: 基因治疗,脂质纳米粒,基因传递,口服途径,口服,控释
摘要: Background: Gene therapy has experimented an increasing attention in the last decades, due to its enormous potential applications in the medical field. It can be defined as the use of genes or genetic material (DNA, RNA, oligonucleotides) to treat or prevent a disease state, generally a geneticbased one.
Application: Other applications, like treating viral, bacterial or parasite infections or development of vaccines are gaining also interest. Efficient gene therapy is mainly dependent on the ability of the highly labile genetic material to reach the therapeutic target. For this purpose, different delivery systems have been designed and extensively investigated. Nanoparticles offer a broad range of possibilities in design, being prepared using biocompatible and biodegradable excipients, being therefore generally considered as safe. Conclusion: Oral delivery of the genetic material is also a great challenge, due to the complexity of this specific biological barrier. Special attention to all the intrinsic hazards for gene delivery due to the barrier must be taken into account during the particle design process. Particle design will also allow targeting to specific sites of the gastrointestinal tract. Solid lipid nanoparticles have been extensively studied in the oral drug delivery field, and also in gene delivery through other administration routes, but still not explored in oral gene delivery. In this manuscript, design considerations and particle-cell interaction mechanisms will be extensively reviewed, focusing on the oral route to encourage the scientific community to explore these valuable carriers for oral gene delivery.Export Options
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Cite this article as:
Lipid Nanoparticles as Potential Gene Therapeutic Delivery Systems for Oral Administration, Current Gene Therapy 2017; 17 (2) . https://dx.doi.org/10.2174/1566523217666170510163038
DOI https://dx.doi.org/10.2174/1566523217666170510163038 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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