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

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...

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Published inFrontiers in genetics Vol. 14; p. 1250276
Main Authors McCartan, Robyn, Khorkova, Olga, Volmar, Claude-Henry, Wahlestedt, Claes
Format Journal Article
LanguageEnglish
Published Switzerland Frontiers Media S.A 16.08.2023
Subjects
ASO
blood brain barrier
drug discovery
Genetics
oligonucleotide
siRNA
blood brain barrier
siRNA
drug discovery
ASO
oligonucleotide
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Summary: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.
Bibliography:ObjectType-Article-1
SourceType-Scholarly Journals-1
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ObjectType-Review-3
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Edited by: Yadong Zheng, Zhejiang Agriculture and Forestry University, China
Selene Ingusci, University of Pittsburgh, United States
Reviewed by: Shahidee Zainal Abidin, University of Malaysia Terengganu, Malaysia
ISSN:1664-8021
1664-8021
DOI:10.3389/fgene.2023.1250276