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CRISPR-Cas9靶向前蛋白转化酶枯草溶菌素9:一种有前景的动脉粥样硬化治疗方法。

CRISPR-Cas9 Targeting PCSK9: A Promising Therapeutic Approach for Atherosclerosis.

作者信息

Gu Bin, Li Min, Li Dan, Huang Kaisen

机构信息

Department of Cardiology, Affiliated Hospital of Southwest Medical University, No.1 Section 1, Xiang Lin Road, Longmatan District, Luzhou, Sichuan, 646000, China.

Department of Cardiology, Neijiang Dongxing District People's Hospital, Neijiang, Sichuan, 641300, China.

出版信息

J Cardiovasc Transl Res. 2025 Apr;18(2):424-441. doi: 10.1007/s12265-024-10587-7. Epub 2025 Jan 13.

Abstract

CRISPR-Cas9 gene editing technology, as an innovative biomedical tool, holds significant potential in the prevention and treatment of atherosclerosis. By precisely editing key genes such as PCSK9, CRISPR-Cas9 offers the possibility of long-term regulation of low-density lipoprotein cholesterol (LDL-C), which may reduce the risk of cardiovascular diseases. Early clinical studies of gene editing therapies like VERVE-101 have yielded encouraging results, highlighting both the feasibility and potential efficacy of this technology. However, clinical applications still face challenges such as off-target effects, immunogenicity, and long-term safety. Future research should focus on enhancing the specificity and efficiency of gene editing, optimizing delivery systems, and improving personalized treatment strategies. Additionally, the establishment of ethical and legal regulatory frameworks will be critical for the safe adoption of this technology. With the continued advancement of gene editing technology, CRISPR-Cas9 may become an important tool for treating atherosclerosis and other complex diseases.

摘要

CRISPR-Cas9基因编辑技术作为一种创新的生物医学工具,在动脉粥样硬化的预防和治疗方面具有巨大潜力。通过精确编辑PCSK9等关键基因,CRISPR-Cas9为长期调节低密度脂蛋白胆固醇(LDL-C)提供了可能性,这可能降低心血管疾病的风险。像VERVE-101这样的基因编辑疗法的早期临床研究已取得令人鼓舞的结果,凸显了该技术的可行性和潜在疗效。然而,临床应用仍面临脱靶效应、免疫原性和长期安全性等挑战。未来的研究应专注于提高基因编辑的特异性和效率、优化递送系统以及改进个性化治疗策略。此外,建立伦理和法律监管框架对于安全采用该技术至关重要。随着基因编辑技术的不断进步,CRISPR-Cas9可能成为治疗动脉粥样硬化和其他复杂疾病的重要工具。

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