Author:
McCartan Robyn,Khorkova Olga,Volmar Claude-Henry,Wahlestedt Claes
Abstract
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.
Funder
National Institutes of Health
Subject
Genetics (clinical),Genetics,Molecular Medicine
Cited by
1 articles.
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1. Emerging therapies in hereditary ataxias;Trends in Molecular Medicine;2024-08