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用CRISPR/Cas9针对LncRNA用于疗法:一篇评论

Bhupendra Puri¹, Anil Bhanudas Gaikwad¹

¹Department of Pharmacy, Birla Institute of Technology and Science, Pilani, Pilani Campus, Vidya Vihar, Pilani, Rajasthan 333031, India.

International journal of biological macromolecules

|December 28, 2025

概括

长非编码RNAs (lncRNAs) 在病的发病过程中至关重要. 在脏疾病中,CRISPR/Cas9基因编辑显示出针对lncRNAs的前景,但临床翻译需要进一步的研究和克服生物学挑战.

科学领域:

分子生物学分子生物学
遗传学是一个遗传学.
腎臟病學 (nephrology) 是一種醫學專業.

背景情况:

长非编码RNA (lncRNAs) 在各种病的发展和进展中起着重要作用.
目前对病病原体中的lncRNA机制的理解是有限的.
基于lncRNA的治疗方法的治疗潜力在临床环境中基本上尚未被探索.

研究的目的:

审查CRISPR/Cas9技术的进展,以针对脏疾病中的lncRNAs.
突出将基于CRISPR/Cas9的lncRNA疗法转化为临床应用的局限性和挑战.
讨论未来开发有效的CRISPR/Cas9基IncRNA疗法治疗脏疾病的方向.

主要方法:

在病中对lncRNAs和CRISPR/Cas9进行临床前和临床研究的文献综述.
对当前的CRISPR/Cas9基因编辑策略进行分析.
评估生物挑战和临床翻译的潜在解决方案.

主要成果:

在临床前脏疾病模型中,CRISPR/Cas9技术已证明在向lncRNAs方面具有有效性.
克里斯普尔/卡斯9在脏疾病,特别是AKI和CKD中准lncRNA的临床应用仍处于早期阶段.
为了成功的临床转化,需要解决重要的生物障碍.

结论:

通过CRISPR/Cas9介导的lncRNAs向是治疗脏疾病的一个有前途的治疗策略.
为了克服现有的局限性并促进这些疗法的临床转化,进一步的研究是必不可少的.
推进用于IncRNA向的CRISPR/Cas9技术,有可能为脏疾病提供新的治疗方法.

关键词:

这就是CRISPR/Cas9的作用.脏疾病脏疾病治疗目标治疗目标在 lncRNAs 中.

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