Insights from Computational Studies of Polymeric Systems for Nucleic Acid Delivery.

IF 4.5 2区 医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL Molecular Pharmaceutics Pub Date : 2025-03-03 Epub Date: 2025-02-16 DOI:10.1021/acs.molpharmaceut.4c00994
Jesse Dylan Ziebarth, Hossain Shadman, Yongmei Wang
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Abstract

The safe and efficient delivery of nucleic acids into cells is a critical step in the success of gene and cell therapies. Although viral vectors are the predominant tools in current gene and cell therapy practices, they present significant challenges including high costs and safety concerns. Nonviral delivery systems for nucleic acids show immense potential for future medicine, particularly as nucleic acid therapeutics continue to be developed for the treatment of a wide range of diseases, including cancer. Significant research efforts, both experimental and computational, have been devoted to the development, characterization, and understanding of nonviral delivery processes. While numerous reviews have documented these research advancements, few have specifically addressed the contributions from computational studies. In this review, we provide an overview of the insights gained from computational and theoretical studies of polymeric systems for nucleic acid delivery. We also highlight future directions where computational and experimental approaches could synergize to advance the field.

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从核酸传递的聚合系统的计算研究的见解。
安全有效地将核酸输送到细胞中是基因和细胞治疗成功的关键一步。尽管病毒载体是当前基因和细胞治疗实践中的主要工具,但它们面临着包括高成本和安全性问题在内的重大挑战。核酸的非病毒传递系统显示出未来医学的巨大潜力,特别是随着核酸疗法继续被开发用于治疗包括癌症在内的各种疾病。重要的研究工作,实验和计算,已经致力于发展,表征和理解非病毒传递过程。虽然许多评论都记录了这些研究进展,但很少有人特别提到计算研究的贡献。在这篇综述中,我们概述了从计算和理论研究中获得的核酸传递聚合物系统的见解。我们还强调了计算和实验方法可以协同推进该领域的未来方向。
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来源期刊
Molecular Pharmaceutics
Molecular Pharmaceutics 医学-药学
CiteScore
8.00
自引率
6.10%
发文量
391
审稿时长
2 months
期刊介绍: Molecular Pharmaceutics publishes the results of original research that contributes significantly to the molecular mechanistic understanding of drug delivery and drug delivery systems. The journal encourages contributions describing research at the interface of drug discovery and drug development. Scientific areas within the scope of the journal include physical and pharmaceutical chemistry, biochemistry and biophysics, molecular and cellular biology, and polymer and materials science as they relate to drug and drug delivery system efficacy. Mechanistic Drug Delivery and Drug Targeting research on modulating activity and efficacy of a drug or drug product is within the scope of Molecular Pharmaceutics. Theoretical and experimental peer-reviewed research articles, communications, reviews, and perspectives are welcomed.
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