Antisense oligonucleotide as novel therapies for neurogenetic disorders.
10.3760/cma.j.cn511374-20240821-00451
- Author:
Liyuan FAN
1
,
2
Author Information
1. University of Hong Kong Shenzhen Hospital, Shenzhen, Guangdong 518053, China. fanliyuansophere@
2. com.
- Publication Type:English Abstract
- MeSH:
Humans;
Oligonucleotides, Antisense/therapeutic use*;
Nervous System Diseases/drug therapy*;
Genetic Therapy/methods*
- From:
Chinese Journal of Medical Genetics
2025;42(1):102-113
- CountryChina
- Language:Chinese
-
Abstract:
Antisense oligonucleotide (ASO) was discovered several decades ago and initially used only as a research tool in the laboratory. In recent years, several ASO therapeutics have been developed for neurological disorders. Some of these therapeutics, including eteplirsen, golodirsen, viltolarsen, nusinersen and inotersen, have been approved by the Food and Drug Administration (FDA) and begun to draw the public's attention as an effective therapeutic approach. These novel therapeutics have shown great performance, while many similar therapeutics are under investigation and in clinical trials. This n-of-1 precision medicine may start a new chapter in the paradigm of therapeutics. Clinicians, clinical geneticists, and genetic counselors may know about this novel therapy, but very few may understand the background in details. During genetic counseling, they have the responsibility to convey the effectiveness, side effects and cost of such therapies to patients and their families. As these target therapies will require precise genetic diagnosis before treatment, healthcare professionals and genetic counselors play a vital role in relating the patients to the corresponding ASO drugs. This review has elaborated the mechanism of ASO therapies, including basic rationales, modifications, side effects and delivery routes. It also systemically summarized the FDA-approved ASO therapeutics and their applications for various neurological disorders, and discussed the limitations and challenges the real-world market may face and issues genetic counselor should take into consideration in the near future.
