Current approaches in CRISPR-Cas systems for diabetes.

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology Progress in Molecular Biology and Translational Science Pub Date : 2025-01-01 Epub Date: 2024-08-30 DOI:10.1016/bs.pmbts.2024.08.002
Vishnu Kirthi Arivarasan, Diksha Diwakar, Neethu Kamarudheen, Karthik Loganathan
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Abstract

In the face of advancements in health care and a shift towards healthy lifestyle, diabetes mellitus (DM) still presents as a global health challenge. This chapter explores recent advancements in the areas of genetic and molecular underpinnings of DM, addressing the revolutionary potential of CRISPR-based genome editing technologies. We delve into the multifaceted relationship between genes and molecular pathways contributing to both type1 and type 2 diabetes. We highlight the importance of how improved genetic screening and the identification of susceptibility genes are aiding in early diagnosis and risk stratification. The spotlight then shifts to CRISPR-Cas9, a robust genome editing tool capable of various applications including correcting mutations in type 1 diabetes, enhancing insulin production in T2D, modulating genes associated with metabolism of glucose and insulin sensitivity. Delivery methods for CRISPR to targeted tissues and cells are explored, including viral and non-viral vectors, alongside the exciting possibilities offered by nanocarriers. We conclude by discussing the challenges and ethical considerations surrounding CRISPR-based therapies for DM. These include potential off-target effects, ensuring long-term efficacy and safety, and navigating the ethical implications of human genome modification. This chapter offers a comprehensive perspective on how genetic and molecular insights, coupled with the transformative power of CRISPR, are paving the way for potential cures and novel therapeutic approaches for DM.

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目前治疗糖尿病的CRISPR-Cas系统的方法。
面对医疗保健的进步和向健康生活方式的转变,糖尿病(DM)仍然是一个全球性的健康挑战。本章探讨了DM的遗传和分子基础领域的最新进展,解决了基于crispr的基因组编辑技术的革命性潜力。我们深入研究了导致1型和2型糖尿病的基因和分子途径之间的多方面关系。我们强调如何改进遗传筛查和易感基因的鉴定是帮助早期诊断和风险分层的重要性。然后焦点转移到CRISPR-Cas9,这是一种强大的基因组编辑工具,能够用于各种应用,包括纠正1型糖尿病的突变,增强T2D中的胰岛素产生,调节与葡萄糖代谢和胰岛素敏感性相关的基因。探索了CRISPR到目标组织和细胞的递送方法,包括病毒和非病毒载体,以及纳米载体提供的令人兴奋的可能性。最后,我们讨论了围绕基于crispr的糖尿病治疗的挑战和伦理考虑,包括潜在的脱靶效应,确保长期疗效和安全性,以及人类基因组修饰的伦理影响。本章提供了一个全面的视角,说明基因和分子的见解,加上CRISPR的变革力量,如何为糖尿病的潜在治愈和新的治疗方法铺平道路。
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来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
期刊最新文献
Advances in applications of the CRISPR/Cas9 system for respiratory diseases. Advances in CRISPR-Cas systems for kidney diseases. CRISPR challenges in clinical developments. Current approaches in CRISPR-Cas system for metabolic disorder. Current approaches in CRISPR-Cas systems for diabetes.
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