毒性聚集的淀粉样β蛋白积累是阿尔茨海默病的关键致病事件。治疗方法侧重于抑制,延期,或淀粉样β纤维和斑块的分散。基因治疗已经发展成为治疗阿尔茨海默病的潜在治疗选择,由于它在最近十年的快速发展。小干扰核糖核酸由于其能够以高序列特异性和几乎无限数量的治疗靶标下调基因的能力,最近在基因治疗中引起了相当大的关注。包括那些曾经被认为是不可用的。然而,细胞摄取不足和小干扰核糖核酸在其生物环境中的不稳定限制了其治疗应用,需要开发一种载体,该载体可以保护遗传物质免受血液中的早期破坏,同时有效地通过血脑屏障传递治疗基因。纳米技术已经成为一种可能的解决方案,和利用纳米颗粒的几种递送系统已被证明可以绕过关于小干扰核糖核酸递送的关键挑战。通过减少遗传成分的酶分解,纳米材料作为基因载体大大提高了基因治疗的效率。脂质体,聚合物纳米颗粒,磁性纳米粒子,树枝状聚合物,胶束是已经设计好的纳米载体的例子,每个都有自己的一组功能。此外,通过基因疗法特异性递送神经营养化合物的最新进展在增强认知能力方面提供了有希望的结果。在本文中,我们强调了在靶向基因递送中使用不同的纳米载体和小干扰核糖核酸介导的基因沉默作为治疗阿尔茨海默病的潜在平台。
Toxic aggregated amyloid-β accumulation is a key pathogenic event in Alzheimer\'s disease. Treatment approaches have focused on the suppression, deferral, or dispersion of amyloid-β fibers and plaques. Gene therapy has evolved as a potential therapeutic option for treating Alzheimer\'s disease, owing to its rapid advancement over the recent decade. Small interfering ribonucleic acid has recently garnered considerable attention in gene therapy owing to its ability to down-regulate genes with high sequence specificity and an almost limitless number of therapeutic targets, including those that were once considered undruggable. However, lackluster cellular uptake and the destabilization of small interfering ribonucleic acid in its biological environment restrict its therapeutic application, necessitating the development of a vector that can safeguard the genetic material from early destruction within the bloodstream while effectively delivering therapeutic genes across the blood-brain barrier. Nanotechnology has emerged as a possible solution, and several delivery systems utilizing nanoparticles have been shown to bypass key challenges regarding small interfering ribonucleic acid delivery. By reducing the enzymatic breakdown of genetic components, nanomaterials as gene carriers have considerably enhanced the efficiency of gene therapy. Liposomes, polymeric nanoparticles, magnetic nanoparticles, dendrimers, and micelles are examples of nanocarriers that have been designed, and each has its own set of features. Furthermore, recent advances in the specific delivery of neurotrophic compounds via gene therapy have provided promising results in relation to augmenting cognitive abilities. In this paper, we highlight the use of different nanocarriers in targeted gene delivery and small interfering ribonucleic acid-mediated gene silencing as a potential platform for treating Alzheimer\'s disease.