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细胞外囊泡与 CRISPR 基因治疗:在阿尔茨海默病、帕金森病、肌萎缩性侧索硬化症和亨廷顿病中的应用现状。

Extracellular vesicle and CRISPR gene therapy: Current applications in Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, and Huntington's disease.

机构信息

Department of Biophysics, International School of Medicine, University of Health Sciences, Istanbul, Türkiye.

School of Medicine, Marmara University, Istanbul, Türkiye.

出版信息

Eur J Neurosci. 2024 Oct;60(8):6057-6090. doi: 10.1111/ejn.16541. Epub 2024 Sep 19.


DOI:10.1111/ejn.16541
PMID:39297377
Abstract

Neurodegenerative diseases are characterized by progressive deterioration of the nervous system. Alzheimer's disease (AD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS), and Huntington's disease (HD) are prominently life-threatening examples of neurodegenerative diseases. The complexity of the pathophysiology in neurodegenerative diseases causes difficulties in diagnosing. Although the drugs temporarily help to correct specific symptoms including memory loss and degeneration, a complete treatment has not been found yet. New therapeutic approaches have been developed to understand and treat the underlying pathogenesis of neurodegenerative diseases. With this purpose, clustered-regularly interspaced short palindromic repeats/CRISPR-associated protein (CRISPR/Cas) technology has recently suggested a new treatment option. Editing of the genome is carried out by insertion and deletion processes on DNA. Safe delivery of the CRISPR/Cas system to the targeted cells without affecting surrounding cells is frequently investigated. Extracellular vesicles (EVs), that is exosomes, have recently been used in CRISPR/Cas studies. In this review, CRISPR/Cas and EV approaches used for diagnosis and/or treatment in AD, PD, ALS, and HD are reviewed. CRISPR/Cas and EV technologies, which stand out as new therapeutic approaches, may offer a definitive treatment option in neurodegenerative diseases.

摘要

神经退行性疾病的特征是神经系统的进行性恶化。阿尔茨海默病(AD)、帕金森病(PD)、肌萎缩侧索硬化症(ALS)和亨廷顿病(HD)是神经退行性疾病中突出的危及生命的例子。神经退行性疾病的病理生理学的复杂性导致诊断困难。尽管药物暂时有助于纠正包括记忆丧失和退化在内的特定症状,但尚未找到完全的治疗方法。已经开发了新的治疗方法来了解和治疗神经退行性疾病的潜在发病机制。为此,最近簇状规则间隔短回文重复序列/CRISPR 相关蛋白(CRISPR/Cas)技术提出了一种新的治疗选择。通过在 DNA 上的插入和缺失过程来进行基因组编辑。经常研究将 CRISPR/Cas 系统安全地递送到靶细胞而不影响周围细胞。细胞外囊泡(EVs),即外泌体,最近已用于 CRISPR/Cas 研究。在这篇综述中,综述了用于 AD、PD、ALS 和 HD 的诊断和/或治疗的 CRISPR/Cas 和 EV 方法。作为新的治疗方法脱颖而出的 CRISPR/Cas 和 EV 技术,可能为神经退行性疾病提供明确的治疗选择。

相似文献

[1]
Extracellular vesicle and CRISPR gene therapy: Current applications in Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, and Huntington's disease.

Eur J Neurosci. 2024-10

[2]
Targeted Delivery for Neurodegenerative Disorders Using Gene Therapy Vectors: Gene Next Therapeutic Goals.

Curr Gene Ther. 2021

[3]
Next Generation Precision Medicine: CRISPR-mediated Genome Editing for the Treatment of Neurodegenerative Disorders.

J Neuroimmune Pharmacol. 2019-4-23

[4]
Common Factors in Neurodegeneration: A Meta-Study Revealing Shared Patterns on a Multi-Omics Scale.

Cells. 2020-12-8

[5]
CRISPR-Based Genome-Editing Tools for Huntington's Disease Research and Therapy.

Neurosci Bull. 2022-11

[6]
CRISPR/Cas9 novel therapeutic road for the treatment of neurodegenerative diseases.

Life Sci. 2020-7-29

[7]
Applications of CRISPR-Cas9 in Alzheimer's Disease and Related Disorders.

Int J Mol Sci. 2022-8-5

[8]
The potential of gene editing for Huntington's disease.

Trends Neurosci. 2023-5

[9]
Unraveling Molecular and Genetic Insights into Neurodegenerative Diseases: Advances in Understanding Alzheimer's, Parkinson's, and Huntington's Diseases and Amyotrophic Lateral Sclerosis.

Int J Mol Sci. 2023-6-28

[10]
microRNAs and Neurodegenerative Diseases.

Adv Exp Med Biol. 2015

引用本文的文献

[1]
Deciphering Molecular and Signaling Pathways of Extracellular Vesicles-Based Therapeutics for Alzheimer's Disease.

Mol Neurobiol. 2025-7-18

[2]
Novel Pyrido[3,4-]pyrimidin-4-one and Pyrimido[5,4-]pyrimidin-4-one Derivatives as TREM2 Agonists for Treating Parkinson's Disease.

ACS Med Chem Lett. 2025-4-2

[3]
Exosome-powered neuropharmaceutics: unlocking the blood-brain barrier for next-gen therapies.

J Nanobiotechnology. 2025-5-3

[4]
Pathophysiology, Clinical Heterogeneity, and Therapeutic Advances in Amyotrophic Lateral Sclerosis: A Comprehensive Review of Molecular Mechanisms, Diagnostic Challenges, and Multidisciplinary Management Strategies.

Life (Basel). 2025-4-14

[5]
CRISPR/Cas9-Based therapeutics as a promising strategy for management of Alzheimer's disease: progress and prospects.

Front Cell Neurosci. 2025-4-7

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