期刊
NEUROPHARMACOLOGY
卷 120, 期 -, 页码 56-62出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.neuropharm.2016.12.015
关键词
Antisense oligonucleotides; Drug development; CNS diseases
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.
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