Polymeric Vehicles for Nucleic Acid Delivery: Enhancing the Therapeutic Efficacy and Cellular Uptake.

Parul Gupta, Anjali Sharma, Vishnu Mittal
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Abstract

Background: 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.

Aim and objectives: 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.

Methodology: 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.

Results: 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.

Conclusion: 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.

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用于核酸递送的聚合物载体:增强疗效和细胞吸收。
背景:使用聚合物载体可促进治疗性基因递送。当与核酸结合形成纳米颗粒或多聚体时,各种聚合物可以保护货物不被体内分解和清除,同时也使其更容易进入细胞内:目的和目标:聚合物合成设计的选择会产生各种各样的化合物和载体成分。根据不同的应用,可以改变这些特性,以提高内体逸出率、延长分布时间或加强与核酸货物和细胞的连接。在此,我们概述了目前在临床前和临床环境中使用聚合物递送基因的方法:方法:在体外和动物模型中,使用递送遗传物质的聚合物载体已经取得了显著的治疗效果。当与核酸结合形成纳米颗粒或多聚体时,各种聚合物可以防止货物在体内分解和清除,同时也使其更容易进入细胞内。在过去的 20 年中,许多创新的核酸诊断方法都得到了研究,并通过了临床评估:结果:基于聚合物的载体因其生物作用方式和场所的变化以及生物物理特性的差异而存在额外的递送问题。我们介绍了最近针对核酸有效载荷(如基因修饰核酸、siRNA、microRNA 和质粒 DNA)调整的定制聚合物载体结构:总之,用于基因递送的聚合物载体的开发为治疗应用带来了希望。通过精心设计和优化,这些载体可以克服与核酸递送相关的各种挑战,为治疗各种疾病提供新的途径。
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