Antisense oligonucleotide therapies in neurodegenerative disease
10.3760/cma.j.issn.1673-4408.2020.05.001
- VernacularTitle:反义寡核苷酸治疗在神经退行性疾病中的应用
- Author:
Jia WANG
1
;
Yujin QU
Author Information
1. 首都儿科研究所遗传室，北京 100020
- From:
International Journal of Pediatrics
2020;47(5):297-301
- CountryChina
- Language:Chinese
-
Abstract:
Antisense oligonucleotides(ASOs)are small sequences of DNA or RNA able to target mRNA transcripts resulting in change or reduction in the expression of target genes, and became an emerging class of therapeutic drugs.With its high specific, adaptable, and low side effects, ASO drugs are widely used in rare diseases, tumor, inflammatory diseases, cardiovascular disease, and infectious diseases.Recent numerous advancements in ASO sequence design, chemical modifications and delivery methods, significant breakthroughs have been made in the treatment and ASOs become an ideal candidate therapies for neurodegenerative diseases.
