Faculty of Pharmaceutical Sciences, University of Central Punjab, Lahore 54000, Pakistan.
Institute of Clinical and Experimental Pharmacology and Toxicology, University of Lubeck 23566 Lubeck, Germany.
Mini Rev Med Chem. 2024;24(19):1723-1745. doi: 10.2174/0113895575301011240407082559.
Neurological disorders present a formidable challenge in modern medicine due to the intricate obstacles set for the brain and the multipart nature of genetic interventions. This review article delves into the promising realm of nanoparticle-based gene therapy as an innovative approach to addressing the intricacies of neurological disorders. Nanoparticles (NPs) provide a multipurpose podium for the conveyance of therapeutic genes, offering unique properties such as precise targeting, enhanced stability, and the potential to bypass blood-brain barrier (BBB) restrictions. This comprehensive exploration reviews the current state of nanoparticle-mediated gene therapy in neurological disorders, highlighting recent advancements and breakthroughs. The discussion encompasses the synthesis of nanoparticles from various materials and their conjugation to therapeutic genes, emphasizing the flexibility in design that contributes to specific tissue targeting. The abstract also addresses the low immunogenicity of these nanoparticles and their stability in circulation, critical factors for successful gene delivery. While the potential of NP-based gene therapy for neurological disorders is vast, challenges and gaps in knowledge persist. The lack of extensive clinical trials leaves questions about safety and potential side effects unanswered. Therefore, this abstract emphasizes the need for further research to validate the therapeutic applications of NP-mediated gene therapy and to address nanosafety concerns. In conclusion, nanoparticle-based gene therapy emerges as a promising avenue in the pursuit of effective treatments for neurological disorders. This abstract advocates for continued research efforts to bridge existing knowledge gaps, unlocking the full potential of this innovative approach and paving the way for transformative solutions in the realm of neurological health.
神经紊乱在现代医学中是一个巨大的挑战,因为大脑的复杂障碍和遗传干预的多方面性质。这篇综述文章深入探讨了基于纳米粒子的基因治疗作为一种创新方法来解决神经紊乱的复杂性的有前途的领域。纳米粒子(NP)为传递治疗基因提供了一个多功能的平台,具有独特的性质,如精确靶向、增强稳定性和潜在的绕过血脑屏障(BBB)限制。这篇全面的综述回顾了纳米粒子介导的基因治疗在神经紊乱中的现状,强调了最近的进展和突破。讨论包括从各种材料合成纳米粒子及其与治疗基因的结合,强调了设计的灵活性,有助于特定组织的靶向。摘要还提到了这些纳米粒子的低免疫原性及其在循环中的稳定性,这是成功基因传递的关键因素。虽然 NP 基基因治疗对神经紊乱的潜力巨大,但仍存在挑战和知识差距。缺乏广泛的临床试验使得关于安全性和潜在副作用的问题没有答案。因此,摘要强调需要进一步研究来验证 NP 介导的基因治疗的治疗应用,并解决纳米安全问题。总之,NP 基基因治疗作为治疗神经紊乱的有效方法具有广阔的前景。本摘要提倡继续进行研究,以弥合现有知识差距,挖掘这一创新方法的全部潜力,并为神经健康领域的变革性解决方案铺平道路。