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探索纳米医学跨越物理化学和细胞屏障用于基因治疗的潜力。

Exploring the potential of nanomedicine for gene therapy across the physicochemical and cellular barriers.

机构信息

Faculty of Pharmaceutical Sciences, University of Central Punjab (UCP), Lahore, 54000, Pakistan.

Faculty of Pharmacy, University Paris Saclay, 17 Avenue des sciences, 91190, Orsay, France.

出版信息

Funct Integr Genomics. 2024 Sep 28;24(5):177. doi: 10.1007/s10142-024-01459-z.

Abstract

After COVID-19, a turning point in the way of pharmaceutical technology is gene therapy with beneficial potential to start a new medical era. However, commercialization of such pharmaceuticals would never be possible without the help of nanotechnology. Nanomedicine can fulfill the growing needs linked to safety, efficiency, and site-specific targeted delivery of Gene therapy-based pharmaceuticals. This review's goal is to investigate how nanomedicine may be used to transfer nucleic acids by getting beyond cellular and physicochemical barriers. Firstly, we provide a full description of types of gene therapy, their mechanism, translation, transcription, expression, type, and details of diseases with possible mechanisms that can only be treated with genes-based pharmaceuticals. Additionally, we also reviewed different types of physicochemical barriers, physiological and cellular barriers in nucleic acids (DNA/RNA) based drug delivery. Finally, we highlight the need and importance of cationic lipid-based nanomedicine/nanocarriers in gene-linked drug delivery and how nanotechnology can help to overcome the above-discussed barrier in gene therapy and their biomedical applications.

摘要

在 COVID-19 之后,药物技术的一个转折点是基因治疗,它具有潜在的益处,可以开创一个新的医学时代。然而,如果没有纳米技术的帮助,此类药物的商业化是不可能实现的。纳米医学可以满足与安全性、效率和基因治疗药物的特定部位靶向递送相关的不断增长的需求。本综述的目的是研究纳米医学如何通过克服细胞和物理化学障碍来传递核酸。首先,我们全面描述了基因治疗的类型、它们的机制、翻译、转录、表达、类型以及可能只能用基于基因的药物治疗的疾病的详细信息。此外,我们还回顾了不同类型的物理化学障碍、核酸(DNA/RNA)基于药物传递的生理和细胞障碍。最后,我们强调了阳离子脂质纳米医学/纳米载体在基因相关药物传递中的必要性和重要性,以及纳米技术如何帮助克服基因治疗中的上述障碍及其在生物医学中的应用。

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