Mapping the therapeutic landscape of CRISPR-Cas9 for combating age-related diseases.

IF 4.9 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Frontiers in genome editing Pub Date : 2025-04-04 eCollection Date: 2025-01-01 DOI:10.3389/fgeed.2025.1558432
Qiyu He, Yida Wang, Zhimin Tan, Xian Zhang, Chao Yu, Xiaoqin Jiang
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引用次数: 0

Abstract

CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-associated protein 9) has emerged as a transformative genome-editing tool with significant therapeutic potential for age-related diseases, including Alzheimer's disease, Parkinson's disease, cardiovascular disorders, and osteoporosis. This study presents a bibliometric analysis of CRISPR-Cas9 research in age-related diseases, identifying key contributors, major research hotspots, and critical technological advancements. While promising applications have been demonstrated in gene repair, functional regulation, and molecular interventions, significant barriers persist, including off-target effects, low delivery efficiency, and limited editing in non-dividing cells. Ethical concerns over germline editing and gaps in long-term safety data further complicate clinical translation. Future directions emphasize the development of high-precision Cas9 variants, homology-directed repair-independent tools, and efficient delivery systems, alongside the establishment of international regulatory frameworks and multicenter clinical trials. These efforts are essential to fully realize the potential of CRISPR-Cas9 in addressing the global health challenges of aging.

绘制CRISPR-Cas9对抗年龄相关疾病的治疗前景
CRISPR-Cas9(聚集规律间隔短回文重复相关蛋白9)已成为一种变革性基因组编辑工具,具有治疗年龄相关疾病的显著潜力,包括阿尔茨海默病、帕金森病、心血管疾病和骨质疏松症。本研究对年龄相关疾病中的CRISPR-Cas9研究进行了文献计量分析,确定了关键贡献者、主要研究热点和关键技术进展。虽然在基因修复、功能调节和分子干预方面已经证明了有希望的应用,但仍然存在重大障碍,包括脱靶效应、低传递效率和在非分裂细胞中的有限编辑。对生殖细胞编辑的伦理担忧和长期安全性数据的差距进一步使临床翻译复杂化。未来的方向是开发高精度的Cas9变体、同源定向修复独立工具和高效的递送系统,以及建立国际监管框架和多中心临床试验。这些努力对于充分发挥CRISPR-Cas9在解决全球老龄化健康挑战方面的潜力至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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CiteScore
7.00
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审稿时长
13 weeks
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