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

IF 2.5 4区 生物学 Q3 BIOCHEMISTRY & MOLECULAR BIOLOGY Molecular Biotechnology Pub Date : 2026-01-01 Epub Date: 2025-02-02 DOI:10.1007/s12033-025-01374-z
Ayesha Shahid, Ambreen Zahra, Sabin Aslam, Amen Shamim, Waqas Rafique Ali, Bilal Aslam, Sultan Habibullah Khan, Muhammad Imran Arshad
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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.

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评价CRISPR技术作为遗传疾病治疗工具包的创新筛选。
遗传性疾病的高频率迫使发展完善的诊断和治疗系统。CRISPR是一种精确的基因组编辑工具,为低资源环境下的即时检测提供了高灵敏度、特异性和灵活性的基因突变检测。CRISPR技术的进步为遗传病的检测带来了新的希望。本文综述了CRISPR在遗传疾病检测和治疗方面的最新进展。它深入研究了下一代CRISPR诊断技术的进步,如纳米生物传感器、数字化CRISPR和基因组学集成CRISPR技术,以提高检测极限,促进“芯片实验室”技术。此外,综述了CRISPR技术的治疗潜力,以评估CRISPR技术在血液病(镰状细胞性贫血和β-地中海贫血)、艾滋病毒、癌症、心血管疾病和神经系统疾病等方面的实施潜力。新兴的CRISPR治疗方法,如碱基/表观遗传编辑和干细胞,用于开发可预见的CRISPR药物,用于开发即时检测。还讨论了人工智能和机器学习的预测模型与不断增长的遗传疾病知识的结合,以了解它们在加速遗传检测中的作用。在审查结束前,将简要讨论伦理问题。这篇综述提供了对CRISPR诊断/治疗方法进展的全面见解,这些进展被认为为可靠、有效和低成本的基因检测铺平了道路。
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来源期刊
Molecular Biotechnology
Molecular Biotechnology 医学-生化与分子生物学
CiteScore
4.10
自引率
3.80%
发文量
165
审稿时长
6 months
期刊介绍: Molecular Biotechnology publishes original research papers on the application of molecular biology to both basic and applied research in the field of biotechnology. Particular areas of interest include the following: stability and expression of cloned gene products, cell transformation, gene cloning systems and the production of recombinant proteins, protein purification and analysis, transgenic species, developmental biology, mutation analysis, the applications of DNA fingerprinting, RNA interference, and PCR technology, microarray technology, proteomics, mass spectrometry, bioinformatics, plant molecular biology, microbial genetics, gene probes and the diagnosis of disease, pharmaceutical and health care products, therapeutic agents, vaccines, gene targeting, gene therapy, stem cell technology and tissue engineering, antisense technology, protein engineering and enzyme technology, monoclonal antibodies, glycobiology and glycomics, and agricultural biotechnology.
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