Progress and promise of antisense oligonucleotide therapeutics for central nervous system diseases

被引：19

作者：

Bishop, Kathie M. ^{[1
]}

机构：

[1] Otonomy, 6275 Nancy Ridge Dr, San Diego, CA 92121 USA

来源：

NEUROPHARMACOLOGY | 2017年 / 120卷

关键词：

Antisense oligonucleotides; Drug development; CNS diseases; SPINAL MUSCULAR-ATROPHY; POSTDURAL PUNCTURE HEADACHE; SINGLE-NUCLEOTIDE POLYMORPHISMS; ANGELMAN-SYNDROME; THERAPY; SOD1; HUNTINGTIN; INHIBITION; PREVENTION; CHILDREN;

D O I：

10.1016/j.neuropharm.2016.12.015

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

Antisense oligonucleotide (ASO) drugs are an emerging class of therapeutics that have recently demonstrated progress and promise to treat diseases of the central nervous system (CNS). ASOs for a variety of targets and mechanisms are currently being investigated in clinical trials and pre-clinically for a number of CNS diseases. This review examines the available data regarding central ASO delivery, distribution, pharmacokinetics, pharmacodynamics and therapeutic opportunities. This article is part of the Special Issue entitled "Beyond small molecules for neurological disorders". (C) 2016 Elsevier Ltd. All rights reserved.

引用

页码：56 / 62

页数：7

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