Akbar Amna, Haider Rida, Agnello Luisa, Noor Bushra, Maqsood Nida, Atif Fatima, Ali Wajeeha, Ciaccio Marcello, Tariq Hira
Institute of Molecular Biology and Biotechnology, Bahauddin Zakariya University, Bosan Road, Multan 60800, Punjab, Pakistan.
Department of Precision Medicine in Medical, Surgical, and Critical Care (Me.Pre.C.C.), University of Palermo, 90100 Palermo, Italy.
Genes (Basel). 2025 Jul 22;16(8):850. doi: 10.3390/genes16080850.
Neurodegenerative diseases (NDs) pose a major challenge to global healthcare systems owing to their devastating effects and limited treatment options. These disorders are characterized by progressive loss of neuronal structure and function, resulting in cognitive and motor impairments. Current therapies primarily focus on symptom management rather than on targeting the underlying causes. However, clustered regularly interspaced short palindromic repeat (CRISPR) technology offers a promising alternative by enabling precise genetic modifications that could halt or even reverse ND progression. CRISPR-Cas9, the most widely used CRISPR system, acts as a molecular scissor targeting specific DNA sequences for editing. By designing guide RNAs (gRNAs) to match sequences in genes associated with NDs, researchers can leverage CRISPR to knockout harmful genes, correct mutations, or insert protective genes. This review explores the potential of CRISPR-based therapies in comparison with traditional treatments for NDs. As research advances, CRISPR has the potential to revolutionize ND treatment by addressing its genetic underpinnings. Ongoing clinical trials and preclinical studies continue to expand our understanding and application of this powerful tool to fight debilitating conditions.
神经退行性疾病(NDs)因其具有毁灭性影响且治疗选择有限,对全球医疗保健系统构成了重大挑战。这些疾病的特征是神经元结构和功能的逐渐丧失,导致认知和运动障碍。目前的治疗主要集中在症状管理上,而非针对根本原因。然而,成簇规律间隔短回文重复序列(CRISPR)技术提供了一种有前景的替代方法,通过实现精确的基因修饰,有可能阻止甚至逆转ND的进展。CRISPR-Cas9是使用最广泛的CRISPR系统,充当靶向特定DNA序列进行编辑的分子剪刀。通过设计与ND相关基因中的序列相匹配的引导RNA(gRNA),研究人员可以利用CRISPR敲除有害基因、纠正突变或插入保护性基因。本综述探讨了基于CRISPR的疗法与传统ND治疗方法相比的潜力。随着研究的进展,CRISPR有潜力通过解决ND的遗传基础来彻底改变其治疗方式。正在进行的临床试验和临床前研究不断扩展我们对这一强大工具的理解和应用,以对抗使人衰弱的疾病。
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