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

被引：0
|
作者：
Mccartan, Robyn [1 ,2 ]
Khorkova, Olga [1 ,3 ]
Volmar, Claude-Henry [1 ,2 ]
Wahlestedt, Claes [1 ,2 ]
机构：
[1] Univ Miami, Miller Sch Med, Ctr Therapeut Innovat, Miami, FL 33136 USA
[2] Univ Miami, Miller Sch Med, Dept Psychiat & Behav Sci, Miami, FL 33136 USA
[3] OPKO Hlth, Miami, FL USA
来源：
FRONTIERS IN GENETICS | 2023年 / 14卷
关键词：
siRNA; ASO; oligonucleotide; blood brain barrier; drug discovery; ANTISENSE OLIGONUCLEOTIDE TOFERSEN; INTRANASAL DELIVERY; RNA THERAPEUTICS; CELLULAR UPTAKE; SIRNA; OLIGODEOXYNUCLEOTIDES; NANOPARTICLES; TRAFFICKING; MODULATION; THERAPIES;
D O I：
10.3389/fgene.2023.1250276
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
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.
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页数：13
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