核酸治疗肝病:十年的技术融合和临床挑战

Yufei Pan , Mingyue Pan , Yuanzheng Huang , Baoping Lian , Yutian Feng , Juan Liu , Xiaoxuan Liu
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引用次数: 0

摘要

本文综述了近十年来核酸治疗肝病的进展,并结合文献计量学分析和转化评价。随着CRISPR-Cas9的进步和非病毒传递系统的创新,该领域已经从基础的病毒载体工程发展到精确的基因组编辑和基于rna的调制。我们的分析强调了对代谢紊乱和肝细胞癌的集中研究,并揭示了对多因素病理的重视。尽管临床里程碑突出了靶向策略的重要进展,但在免疫相容性和临床前转化方面仍存在重大挑战。计算建模、人类相关疾病模型和组合策略的整合,使核酸疗法通过机制驱动的干预措施,在解决不断演变的全球肝病负担方面处于独特的地位。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Nucleic acid therapeutics for liver diseases: A decade of technological convergence and clinical challenges
This review synthesizes a decade of advancements in nucleic acid therapeutics for liver diseases, incorporating bibliometric analysis and translational evaluation. The field has evolved from foundational viral vector engineering to precision genome editing and RNA-based modulation, with advancements in CRISPR-Cas9 and innovations in non-viral delivery systems. Our analysis highlights the concentrated research efforts made in metabolic disorders and hepatocellular carcinoma, and reveals an emerging emphasis on multifactorial pathologies. Although clinical milestones highlight important advancements in targeting strategies, significant challenges remain in immune compatibility and preclinical translation. The integration of computational modeling, human-relevant disease models, and combinatorial strategies places nucleic acid therapies in a unique position to tackle the evolving global liver disease burden through mechanism-driven interventions.
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