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反义疗法:神经退行性疾病治疗中的潜在突破。

Antisense therapy: a potential breakthrough in the treatment of neurodegenerative diseases.

作者信息

Romano Roberta, Bucci Cecilia

机构信息

Department of Biological and Environmental Sciences and Technologies (DiSTeBA), University of Salento, Lecce, Italy.

出版信息

Neural Regen Res. 2024 May;19(5):1027-1035. doi: 10.4103/1673-5374.385285.

Abstract

Neurodegenerative diseases are a group of disorders characterized by the progressive degeneration of neurons in the central or peripheral nervous system. Currently, there is no cure for neurodegenerative diseases and this means a heavy burden for patients and the health system worldwide. Therefore, it is necessary to find new therapeutic approaches, and antisense therapies offer this possibility, having the great advantage of not modifying cellular genome and potentially being safer. Many preclinical and clinical studies aim to test the safety and effectiveness of antisense therapies in the treatment of neurodegenerative diseases. The objective of this review is to summarize the recent advances in the development of these new technologies to treat the most common neurodegenerative diseases, with a focus on those antisense therapies that have already received the approval of the U.S. Food and Drug Administration.

摘要

神经退行性疾病是一组以中枢或周围神经系统中神经元进行性退化为特征的疾病。目前,神经退行性疾病尚无治愈方法,这给全球患者和卫生系统带来了沉重负担。因此,有必要寻找新的治疗方法,反义疗法提供了这种可能性,具有不改变细胞基因组且可能更安全的巨大优势。许多临床前和临床研究旨在测试反义疗法治疗神经退行性疾病的安全性和有效性。本综述的目的是总结这些用于治疗最常见神经退行性疾病的新技术的最新进展,重点关注那些已获得美国食品药品监督管理局批准的反义疗法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cd4d/10749614/af8979cbe1ca/NRR-19-1027-g001.jpg

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