Nanotechnology strategies to address challenges in topical and cellular delivery of siRNAs in skin disease therapy

IF 15.2 1区 医学 Q1 PHARMACOLOGY & PHARMACY
Ana Vitoria Pupo Silvestrini, Milena Finazzi Morais, Bryan Wender Debiasi, Fabíola Garcia Praça, Maria Vitória Lopes Badra Bentley
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Abstract

Gene therapy is one of the most advanced therapies in current medicine. In particular, interference RNA-based therapy by small interfering RNA (siRNA) has gained attention in recent years as it is a highly versatile, selective and specific therapy. In dermatological conditions, topical delivery of siRNA offers numerous therapeutic advantages, mainly by inhibiting the expression of target transcripts directly in the skin. However, crossing the stratum corneum and overcoming intracellular barriers is an inherent challenge. Substantial efforts by scientists have moved towards the use of multimodal and multifunctional nanoparticles to overcome these barriers and achieve greater bioavailability in their site of action, the cytoplasm. In this review the most innovative strategies based on nanoparticle and physical methods are presented, as well as the design principles and the main factors that contribute to the performance of these systems. This review also highlights the synergistic contributions of medicine, nanotechnology, and molecular biology to advancing translational research into siRNA-based therapeutics for skin diseases.

Abstract Image

采用纳米技术策略应对皮肤病治疗中局部和细胞递送 siRNA 的挑战。
基因疗法是目前医学界最先进的疗法之一。特别是以小干扰 RNA(siRNA)为基础的干扰 RNA 疗法,因其用途广泛、选择性强、特异性高而近年来备受关注。在皮肤病方面,siRNA 的局部给药具有许多治疗优势,主要是通过直接抑制皮肤中目标转录本的表达。然而,穿过角质层和克服细胞内屏障是一项固有的挑战。科学家们正努力利用多模式和多功能纳米粒子来克服这些障碍,并在其作用部位--细胞质--获得更高的生物利用度。本综述介绍了基于纳米粒子和物理方法的最具创新性的策略,以及设计原则和影响这些系统性能的主要因素。本综述还强调了医学、纳米技术和分子生物学在推动基于 siRNA 的皮肤病治疗转化研究方面的协同贡献。
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来源期刊
CiteScore
28.10
自引率
5.00%
发文量
294
审稿时长
15.1 weeks
期刊介绍: The aim of the Journal is to provide a forum for the critical analysis of advanced drug and gene delivery systems and their applications in human and veterinary medicine. The Journal has a broad scope, covering the key issues for effective drug and gene delivery, from administration to site-specific delivery. In general, the Journal publishes review articles in a Theme Issue format. Each Theme Issue provides a comprehensive and critical examination of current and emerging research on the design and development of advanced drug and gene delivery systems and their application to experimental and clinical therapeutics. The goal is to illustrate the pivotal role of a multidisciplinary approach to modern drug delivery, encompassing the application of sound biological and physicochemical principles to the engineering of drug delivery systems to meet the therapeutic need at hand. Importantly the Editorial Team of ADDR asks that the authors effectively window the extensive volume of literature, pick the important contributions and explain their importance, produce a forward looking identification of the challenges facing the field and produce a Conclusions section with expert recommendations to address the issues.
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