Progress in the Treatment of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
10.12376/j.issn.2097-0501.2022.02.015
- VernacularTitle:反义寡核苷酸类药物在Duchenne型肌营养不良中的治疗进展
- Author:
Tingting XU
1
,
2
;
Wei ZUO
1
,
2
;
Xin LIU
1
,
2
;
Qianqian FAN
1
,
2
;
Yang GAO
1
,
2
;
Chunsu LIANG
1
,
2
;
Bo ZHANG
1
,
2
Author Information
1. Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences &
2. Peking Union Medical College, Beijing 100730, China.
- Publication Type:Journal Article
- Keywords:
antisense oligonucleotides;
Duchenne muscular dystrophy;
rare diseases;
pharmacokinetics;
efficacy and safety
- From:
JOURNAL OF RARE DISEASES
2022;1(2):199-205
- CountryChina
- Language:Chinese
-
Abstract:
In recent years, antisense oligonucleotide (ASO) has been very active in the field of rare disease research and development, especially in Duchenne muscular dystrophy, where it made a major breakthrough. Duchenne muscular dystrophy (DMD) is a rare childhood myopathy caused by mutations in the dystrophin gene. Currently, the four ASO drugs approved internationally for DMD are all targeted at dystrophin, including eteplirsen, golodirsen, viltolarsen and casimersen. They all belong to phosphorodiamidate morpholino oligomers (PMO) antisense oligonucleotide drugs, so that their pharmacokinetic characteristics are similar. The drugs quickly spread to other tissues after intravenous administration. Because of the electrical neutrality of the PMO, they have a low binding rate to plasma proteins and are quickly metabolized by the kidney and excreted in the urine as archetypes. In addition, the likelihood of drug-drug interactions of ASO is low. Existing clinical studies have shown that they have certain clinical benefits and good tolerability, bringing new options for DMD treatment. This paper mainly discusses the pharmacological effects, pharmacokinetic characteristics, efficacy, and safety of ASO drugs for the treatment of DMD, hoping to provide scientific reference for the rational and safe clinical use of such drugs.
