中国神经再生研究(英文版) ›› 2024, Vol. 19 ›› Issue (5): 1027-1035.doi: 10.4103/1673-5374.385285

• 综述:退行性病与再生 • 上一篇    下一篇

反义治疗:神经退行性疾病治疗的潜在突破

  

  • 出版日期:2024-05-15 发布日期:2023-10-31

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

Roberta Romano*, Cecilia Bucci*   

  1. Department of Biological and Environmental Sciences and Technologies (DiSTeBA), University of Salento, Lecce, Italy
  • Online:2024-05-15 Published:2023-10-31
  • Contact: Cecilia Bucci, PhD, cecilia.bucci@unisalento.it; Roberta Romano, PhD, roberta.romano@unisalento.it.
  • Supported by:
    This work was partially supported by Association 2HE (Center for Human Health and Environment) and by Regione Puglia - Grant Malattie Rare DUP n. 246 of 2019 (to CB).

摘要: https://orcid.org/0000-0002-6232-6183 (Cecilia Bucci); https://orcid.org/0000-0002-4987-3561 (Roberta Romano) 

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.

Key words: Alzheimer’s disease, amyotrophic lateral sclerosis, antisense oligonucleotide, Huntington’s disease, neurodegenerative disorders, Parkinson’s disease, siRNA