Advances of Antisense Oligonucleotide Technology in the Treatment of Hereditary Neurodegenerative Diseases.
Journal
Evidence-based complementary and alternative medicine : eCAM
ISSN: 1741-427X
Titre abrégé: Evid Based Complement Alternat Med
Pays: United States
ID NLM: 101215021
Informations de publication
Date de publication:
2021
2021
Historique:
received:
05
11
2020
revised:
13
05
2021
accepted:
29
05
2021
entrez:
2
7
2021
pubmed:
3
7
2021
medline:
3
7
2021
Statut:
epublish
Résumé
Antisense nucleic acids are single-stranded oligonucleotides that have been specially chemically modified, which can bind to RNA expressed by target genes through base complementary pairing and affect protein synthesis at the level of posttranscriptional processing or protein translation. In recent years, the application of antisense nucleic acid technology in the treatment of neuromuscular diseases has made remarkable progress. In 2016, the US FDA approved two antisense nucleic acid drugs for the treatment of Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), and the development to treat other neurodegenerative diseases has also entered the clinical stage. Therefore, ASO represents a treatment with great potential. The article will summarize ASO therapies in terms of mechanism of action, chemical modification, and administration methods and analyze their role in several common neurodegenerative diseases, such as SMA, DMD, and amyotrophic lateral sclerosis (ALS). This article systematically summarizes the great potential of antisense nucleic acid technology in the treatment of hereditary neurodegenerative diseases.
Identifiants
pubmed: 34211575
doi: 10.1155/2021/6678422
pmc: PMC8211492
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
6678422Informations de copyright
Copyright © 2021 Mengsi Lin et al.
Déclaration de conflit d'intérêts
The authors declare that they have no conflicts of interest.
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