Delivering a disease-modifying treatment for Huntington's disease. Issue 1 (January 2015)

• Record Type: Journal Article
• Authors: Godinho, Bruno M.D.C.; Malhotra, Meenakshi; O'Driscoll, Caitriona M.; Cryan, John F.
• Is Part Of: Drug discovery today. Volume 20:Issue 1(2015)
• Page Start: 50
• Page End: 64
• Publication Date: 2015-01
• Subjects: Drugs -- Design -- Periodicals; Drugs -- Research -- Periodicals; 615.1
• DOI: 10.1016/j.drudis.2014.09.011
• Languages: English
• ISSNs: 1359-6446

Abstract

<abstract abstract-type="author-highlights" id="abs0005"> <title id="sect0005">Highlights</title> <sec> <p id="spar0005"> <list id="lis0005"> <list-item id="lsti0005"> <label></label> <p id="par0005">Post-transcriptional gene silencing has shown preclinical benefits for the treatment of HD.</p> </list-item> <list-item id="lsti0010"> <label></label> <p id="par0010">Specific design/synthesis of oligonucleotides is essential for on-target silencing of mRNA.</p> </list-item> <list-item id="lsti0015"> <label></label> <p id="par0015">Non-viral nanovectors cater the transport of oligonucleotides across physiological barriers.</p> </list-item> <list-item id="lsti0020"> <label></label> <p id="par0020">Efficient design/synthesis of nanovectors is essential to achieve site-specific targeting, <italic>in vivo.</italic></p> </list-item> <list-item id="lsti0025"> <label></label> <p id="par0025">Allele-specific silencing of mutant HTT is preferable for HD, preserving the wild-type allele.</p> </list-item> </list> </p> </sec> </abstract>