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CRISPR技术作为遗传疾病创新筛查和治疗工具的评估。

Appraisal of CRISPR Technology as an Innovative Screening to Therapeutic Toolkit for Genetic Disorders.

作者信息

Shahid Ayesha, Zahra Ambreen, Aslam Sabin, Shamim Amen, Ali Waqas Rafique, Aslam Bilal, Khan Sultan Habibullah, Arshad Muhammad Imran

机构信息

National Center for Genome Editing, Center for Advanced Studies/D-8 Research Center, University of Agriculture, Faisalabad, 38000, Pakistan.

Center for Agricultural Biochemistry and Biotechnology, University of Agriculture, Faisalabad, 38000, Pakistan.

出版信息

Mol Biotechnol. 2025 Feb 2. doi: 10.1007/s12033-025-01374-z.

Abstract

The high frequency of genetic diseases compels the development of refined diagnostic and therapeutic systems. CRISPR is a precise genome editing tool that offers detection of genetic mutation with high sensitivity, specificity and flexibility for point-of-care testing in low resource environment. Advancements in CRISPR ushered new hope for the detection of genetic diseases. This review aims to explore the recent advances in CRISPR for the detection and treatment of genetic disorders. It delves into the advances like next-generation CRISPR diagnostics like nano-biosensors, digitalized CRISPR, and omics-integrated CRISPR technologies to enhance the detection limits and to facilitate the "lab-on-chip" technologies. Additionally, therapeutic potential of CRISPR technologies is reviewed to evaluate the implementation potential of CRISPR technologies for the treatment of hematological diseases, (sickle cell anemia and β-thalassemia), HIV, cancer, cardiovascular diseases, and neurological disorders, etc. Emerging CRISPR therapeutic approaches such as base/epigenetic editing and stem cells for the development of foreseen CRIPSR drugs are explored for the development of point-of-care testing. A combination of predictive models of artificial intelligence and machine learning with growing knowledge of genetic disorders has also been discussed to understand their role in acceleration of genetic detection. Ethical consideration are briefly discussed towards to end of review. This review provides the comprehensive insights into advances in the CRISPR diagnostics/therapeutics which are believed to pave the way for reliable, effective, and low-cost genetic testing.

摘要

遗传疾病的高发性促使人们开发精准的诊断和治疗系统。CRISPR是一种精确的基因组编辑工具,能够以高灵敏度、特异性和灵活性检测基因突变,适用于资源匮乏环境下的即时检测。CRISPR技术的进步为遗传疾病的检测带来了新希望。本综述旨在探讨CRISPR在遗传疾病检测和治疗方面的最新进展。它深入研究了诸如纳米生物传感器等下一代CRISPR诊断技术、数字化CRISPR以及组学整合CRISPR技术等进展,以提高检测限并推动“芯片实验室”技术发展。此外,还综述了CRISPR技术的治疗潜力,以评估CRISPR技术在治疗血液疾病(镰状细胞贫血和β地中海贫血)、HIV、癌症、心血管疾病和神经系统疾病等方面的应用潜力。为了开发即时检测,还探索了新兴的CRISPR治疗方法,如碱基/表观遗传编辑和用于开发预期CRISPR药物的干细胞。还讨论了人工智能和机器学习的预测模型与对遗传疾病不断增长的认识相结合,以了解它们在加速基因检测中的作用。综述结尾简要讨论了伦理考量。本综述全面深入地介绍了CRISPR诊断/治疗的进展,相信这些进展将为可靠、有效且低成本的基因检测铺平道路。

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