Nucleic acid-based therapeutics for the treatment of central nervous system disorders.
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ABSTRACT:
SUBMITTER: McCartan R
PROVIDER: S-EPMC10468602 | biostudies-literature | 2023
REPOSITORIES: biostudies-literature
Publications
Nucleic acid-based therapeutics for the treatment of central nervous system disorders.
McCartan Robyn R Khorkova Olga O Volmar Claude-Henry CH Wahlestedt Claes C
Frontiers in genetics 20230816
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 inten ...[more]
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