通过超可塑囊泡高效递送 siRNA 增强基因疗法。

Q2 Pharmacology, Toxicology and Pharmaceutics
Chintan Aundhia, Nirmal Shah, Chitrali Talele, Aarti Zanwar, Mamta Kumari, Sapana Patil
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引用次数: 0

摘要

基因疗法是一种革命性的方法,旨在通过操纵特定基因的表达来治疗各种疾病。超变形囊泡的成分和配方在决定其作为 siRNA 递送载体的特性和性能方面起着至关重要的作用。在开发用于递送 siRNA 的超变形囊泡时,仔细选择和优化脂质对于获得理想的囊泡特性以及高效的 siRNA 封装和递送至关重要。角质层起着保护屏障的作用,限制了包括 siRNA 在内的分子向皮肤深层的渗透。超微可变形囊泡为克服这一屏障、促进 siRNA 向皮肤靶细胞的高效递送提供了一种很有前景的解决方案。角质层是皮肤的最外层,是 siRNA 穿透皮肤的重要屏障。这些工程方法能生产出均匀、轮廓清晰的囊泡,并能增强其变形能力,提高 siRNA 的封装效率。展望未来,超变形囊泡设计和优化方面的进步,以及对组合策略和监管框架的不断探索,将进一步推动基于超变形囊泡的 siRNA 递送领域的发展。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Enhancing Gene Therapy through Ultradeformable Vesicles for Efficient siRNA Delivery.

Gene therapy is a revolutionary approach aimed at treating various diseases by manipulating the expression of specific genes. The composition and formulation of ultra-deformable vesicles play a crucial role in determining their properties and performance as siRNA delivery vectors. In the development of ultra-deformable vesicles for siRNA delivery, careful lipid selection and optimization are crucial for achieving desirable vesicle characteristics and efficient siRNA encapsulation and delivery. The stratum corneum acts as a protective barrier, limiting the penetration of molecules, including siRNA, into the deeper layers of the skin. Ultradeformable vesicles offer a promising solution to overcome this barrier and facilitate efficient siRNA delivery to target cells in the skin. The stratum corneum, the outermost layer of the skin, acts as a significant barrier to the penetration of siRNA.These engineering approaches enable the production of uniform and well-defined vesicles with enhanced deformability and improved siRNA encapsulation efficiency. Looking ahead, advancements in ultra-deformable vesicle design and optimization, along with continued exploration of combination strategies and regulatory frameworks, will further drive the field of ultra-deformable vesicle-based siRNA delivery.

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来源期刊
Pharmaceutical nanotechnology
Pharmaceutical nanotechnology Pharmacology, Toxicology and Pharmaceutics-Pharmaceutical Science
CiteScore
4.20
自引率
0.00%
发文量
46
期刊介绍: Pharmaceutical Nanotechnology publishes original manuscripts, full-length/mini reviews, thematic issues, rapid technical notes and commentaries that provide insights into the synthesis, characterisation and pharmaceutical (or diagnostic) application of materials at the nanoscale. The nanoscale is defined as a size range of below 1 µm. Scientific findings related to micro and macro systems with functionality residing within features defined at the nanoscale are also within the scope of the journal. Manuscripts detailing the synthesis, exhaustive characterisation, biological evaluation, clinical testing and/ or toxicological assessment of nanomaterials are of particular interest to the journal’s readership. Articles should be self contained, centred around a well founded hypothesis and should aim to showcase the pharmaceutical/ diagnostic implications of the nanotechnology approach. Manuscripts should aim, wherever possible, to demonstrate the in vivo impact of any nanotechnological intervention. As reducing a material to the nanoscale is capable of fundamentally altering the material’s properties, the journal’s readership is particularly interested in new characterisation techniques and the advanced properties that originate from this size reduction. Both bottom up and top down approaches to the realisation of nanomaterials lie within the scope of the journal.
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