基于 CRISPR 的神经退行性疾病基因调控疗法的进展。

IF 3.8 4区 医学 Q2 GENETICS & HEREDITY
Bharat Bhushan, Kuldeep Singh, Shivendra Kumar, Anjali Bhardwaj
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引用次数: 0

摘要

:神经退行性疾病给医疗保健领域带来了重大挑战,因为这些疾病的表现形式复杂多样,通常归因于基因异常。随着CRISPR(Clustered Regularly Interspaced Short Palindromic Repeats,聚类正则间隔短联合重复序列)技术的出现,人们在寻求有针对性的精确遗传操作的过程中发现了一个新的领域。本摘要探讨了基于 CRISPR 的疗法在解决导致各种神经退行性疾病的遗传因素方面的最新进展和潜在应用。这篇综述深入探讨了 CRISPR 技术的基本原理,强调了它以前所未有的精度编辑基因序列的无与伦比的能力。此外,它还介绍了利用 CRISPR 针对与亨廷顿氏病、阿尔茨海默病、肌萎缩性脊髓侧索硬化症(ALS)和帕金森病等神经退行性疾病相关的特定基因突变所取得的最新进展。它讲述了最重要的研究和试验,这些研究和试验显示了基于CRISPR的疗法是如何安全有效地发挥作用。这表明该技术如何改变导致疾病的基因变异。值得注意的是,讨论强调了在临床环境中实施CRISPR所面临的挑战和伦理方面的考虑,包括脱靶效应、传递方法和长期影响。此外,文章还探讨了广泛应用 CRISPR 技术治疗神经退行性疾病的前景和潜在障碍。文章还谈到了继续研究、改进给药机制和伦理框架的必要性,以确保负责任地、公平地使用这些突破性疗法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Advancements in CRISPR-Based Therapies for Genetic Modulation in Neurodegenerative Disorders.

: Neurodegenerative disorders pose significant challenges in the realm of healthcare, as these conditions manifest in complex, multifaceted ways, often attributed to genetic anomalies. With the emergence of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology, a new frontier has been unveiled in the quest for targeted, precise genetic manipulation. This abstract explores the recent advancements and potential applications of CRISPR-based therapies in addressing genetic components contributing to various neurodegenerative disorders. The review delves into the foundational principles of CRISPR technology, highlighting its unparalleled ability to edit genetic sequences with unprecedented precision. In addition, it talks about the latest progress in using CRISPR to target specific genetic mutations linked to neurodegenerative diseases like Huntington's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), and Parkinson's disease. It talks about the most important studies and trials that show how well and safely CRISPR-based therapies work. This shows how this technology can change genetic variants that cause diseases. Notably, the discussion emphasizes the challenges and ethical considerations associated with the implementation of CRISPR in clinical settings, including off-target effects, delivery methods, and long-term implications. Furthermore, the article explores the prospects and potential hurdles in the widespread application of CRISPR technology for treating neurodegenerative disorders. It touches upon the need for continued research, improved delivery mechanisms, and ethical frameworks to ensure responsible and equitable access to these groundbreaking therapies.

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来源期刊
Current gene therapy
Current gene therapy 医学-遗传学
CiteScore
6.70
自引率
2.80%
发文量
46
期刊介绍: Current Gene Therapy is a bi-monthly peer-reviewed journal aimed at academic and industrial scientists with an interest in major topics concerning basic research and clinical applications of gene and cell therapy of diseases. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Current Gene Therapy publishes full-length/mini reviews and original research on the latest developments in gene transfer and gene expression analysis, vector development, cellular genetic engineering, animal models and human clinical applications of gene and cell therapy for the treatment of diseases. Current Gene Therapy publishes reviews and original research containing experimental data on gene and cell therapy. The journal also includes manuscripts on technological advances, ethical and regulatory considerations of gene and cell therapy. Reviews should provide the reader with a comprehensive assessment of any area of experimental biology applied to molecular medicine that is not only of significance within a particular field of gene therapy and cell therapy but also of interest to investigators in other fields. Authors are encouraged to provide their own assessment and vision for future advances. Reviews are also welcome on late breaking discoveries on which substantial literature has not yet been amassed. Such reviews provide a forum for sharply focused topics of recent experimental investigations in gene therapy primarily to make these results accessible to both clinical and basic researchers. Manuscripts containing experimental data should be original data, not previously published.
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