Nucleic acid-based therapeutics for the treatment of central nervous system disorders

Frontiers in Genetics

Genetics

Claes Wahlestedt¹  Robyn McCartan¹  Claude-Henry Volmar¹  Olga Khorkova² 

[1] Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, Florida, United States;Department of Psychiatry and Behavioral Sciences, University of Miami Miller School of Medicine, Miami, Florida, United States;Center for Therapeutic Innovation, University of Miami Miller School of Medicine, Miami, Florida, United States;OPKO Health, Miami, Florida, United States;

关键词: siRNA;    ASO;    oligonucleotide;    blood brain barrier;    drug discovery;   

DOI  :  10.3389/fgene.2023.1250276

 received in 2023-06-29, accepted in 2023-08-07,  发布年份 2023

来源: Frontiers

【 摘 要 】

Nucleic acid-based therapeutics (NBTs) are an emerging class of drugs with potential for the treatment of a wide range of central nervous system conditions. To date, pertaining to CNS indications, there are two commercially available NBTs and a large number of ongoing clinical trials. However, these NBTs are applied directly to the brain due to very low blood brain barrier permeability. In this review, we outline recent advances in chemical modifications of NBTs and NBT delivery techniques intended to promote brain exposure, efficacy, and possible future systemic application.

【 授权许可】

Unknown   
Copyright © 2023 McCartan, Khorkova, Volmar and Wahlestedt.