The Role of CRISPR/Cas9 in Revolutionizing Duchenne's Muscular Dystrophy Treatment: Opportunities and Obstacles.

IF 1.2 Q4 GENETICS & HEREDITY
Global Medical Genetics Pub Date : 2024-10-18 eCollection Date: 2024-12-01 DOI:10.1055/s-0044-1791803
Ahsan Ali, Md Yakeen Rahman, Danish Sheikh
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引用次数: 0

Abstract

Duchenne's muscular dystrophy (DMD) is a severe X-linked disorder characterized by progressive muscle degeneration, leading to loss of ambulation, respiratory failure, and premature death. It affects approximately 1 in 3,500 live male births and is caused by mutations in the dystrophin gene, which impairs muscle fiber stability. Current treatments are limited to managing symptoms and slowing disease progression, with no curative therapies available. The advent of CRISPR/Cas9 gene-editing technology has introduced a promising approach for directly correcting the genetic mutations responsible for DMD. This review explores the potential of CRISPR/Cas9 as a transformative therapy for DMD, highlighting its successes in preclinical models, the challenges associated with its delivery, and the obstacles to its clinical application. While preclinical studies demonstrate the efficacy of CRISPR/Cas9 in restoring dystrophin expression and improving muscle function, significant hurdles remain, including optimizing delivery methods and ensuring long-term safety.

CRISPR/Cas9在杜氏肌肉萎缩症治疗革命中的作用:机遇与障碍。
杜兴氏肌营养不良症(DMD)是一种严重的 X 连锁疾病,以进行性肌肉变性为特征,可导致丧失行动能力、呼吸衰竭和过早死亡。每 3500 名活产男婴中约有 1 人患此病,病因是肌营养不良蛋白基因突变,该基因会损害肌纤维的稳定性。目前的治疗方法仅限于控制症状和延缓疾病进展,尚无根治性疗法。CRISPR/Cas9 基因编辑技术的出现为直接纠正导致 DMD 的基因突变带来了希望。本综述探讨了 CRISPR/Cas9 作为 DMD 的变革性疗法的潜力,重点介绍了它在临床前模型中取得的成功、其应用所面临的挑战以及临床应用的障碍。尽管临床前研究证明了 CRISPR/Cas9 在恢复肌营养不良蛋白表达和改善肌肉功能方面的疗效,但仍存在重大障碍,包括优化给药方法和确保长期安全性。
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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来源期刊
Global Medical Genetics
Global Medical Genetics GENETICS & HEREDITY-
自引率
11.80%
发文量
30
审稿时长
14 weeks
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