Department of Pharmaceutics, Guru Gobind Singh College of Pharmacy, Yamunanagar, Haryana, 135001, India.
Recent Adv Drug Deliv Formul. 2024;18(4):276-293. doi: 10.2174/0126673878324536240805060143.
Therapeutic gene delivery may be facilitated by the use of polymeric carriers. When combined with nucleic acids to form nanoparticles or polyplexes, a variety of polymers may shield the cargo from in vivo breakdown and clearance while also making it easier for it to enter intracellular compartments.
Polymer synthesis design choices result in a wide variety of compounds and vehicle compositions. Depending on the application, these characteristics may be changed to provide enhanced endosomal escape, longer-lasting distribution, or stronger connection with nucleic acid cargo and cells. Here, we outline current methods for delivering genes in preclinical and clinical settings using polymers.
Significant therapeutic outcomes have previously been attained using genetic material- delivering polymer vehicles in both in-vitro and animal models. When combined with nucleic acids to form nanoparticles or polyplexes, a variety of polymers may shield the cargo from in vivo breakdown and clearance while also making it easier for it to enter intracellular compartments. Many innovative diagnoses for nucleic acids have been investigated and put through clinical assessment in the past 20 years.
Polymer-based carriers have additional delivery issues due to their changes in method and place of biological action, as well as variances in biophysical characteristics. We cover recent custom polymeric carrier architectures that were tuned for nucleic acid payloads such genomemodifying nucleic acids, siRNA, microRNA, and plasmid DNA.
In conclusion, the development of polymeric carriers for gene delivery holds promise for therapeutic applications. Through careful design and optimization, these carriers can overcome various challenges associated with nucleic acid delivery, offering new avenues for treating a wide range of diseases.
治疗性基因传递可以通过使用聚合物载体来实现。当与核酸形成纳米颗粒或多聚物时，各种聚合物可以保护货物免受体内分解和清除的影响，同时也更容易使其进入细胞内隔室。
聚合物合成设计选择导致了广泛的化合物和载体组成。根据应用的不同，这些特性可以改变，以提供增强的内体逃逸、更长时间的分布或与核酸货物和细胞更强的连接。在这里，我们概述了目前使用聚合物在临床前和临床环境中传递基因的方法。
在体外和动物模型中，使用遗传物质传递聚合物载体已经取得了显著的治疗效果。当与核酸形成纳米颗粒或多聚物时，各种聚合物可以保护货物免受体内分解和清除的影响，同时也更容易使其进入细胞内隔室。在过去的 20 年中，已经研究并通过临床评估了许多用于核酸的创新诊断方法。
由于聚合物载体在生物作用的方式和地点上的变化以及生物物理特性的差异，它们还有其他的传递问题。我们介绍了最近为核酸有效载荷（如基因组修饰核酸、siRNA、miRNA 和质粒 DNA）调整的定制聚合物载体架构。
总之，用于基因传递的聚合物载体的开发为治疗应用提供了希望。通过精心设计和优化，这些载体可以克服与核酸传递相关的各种挑战，为治疗广泛的疾病提供新的途径。
