dc.contributor.author |
Klim, Joseph R |
en |
dc.contributor.author |
Vance, Caroline |
en |
dc.contributor.author |
Scotter, Emma |
en |
dc.date.accessioned |
2020-02-11T22:48:11Z |
en |
dc.date.issued |
2019-05 |
en |
dc.identifier.issn |
1357-2725 |
en |
dc.identifier.uri |
http://hdl.handle.net/2292/49916 |
en |
dc.description.abstract |
Amyotrophic lateral sclerosis (ALS) is a disease with highly heterogenous causes, most of which remain unknown, a multitude of possible disease mechanisms, and no therapy currently available that can halt disease progression. However, recent advances in antisense oligonucleotides have made them a viable option for targeted therapeutics for patients. These molecules offer a method of targeting RNA that is highly specific, adaptable, and does not require viral delivery. Antisense oligonucleotides are therefore being developed for several genetic causes of ALS. Furthermore, biological pathways involved in the pathogenesis of disease also offer tantalizing targets for intervention using antisense oligonucleotides. Here we detail existing and potential targets for antisense oligonucleotides in ALS and briefly examine the requirements for these drugs to reach and be effective in clinic. |
en |
dc.format.medium |
Print-Electronic |
en |
dc.language |
eng |
en |
dc.relation.ispartofseries |
The international journal of biochemistry & cell biology |
en |
dc.rights |
Items in ResearchSpace are protected by copyright, with all rights reserved, unless otherwise indicated. Previously published items are made available in accordance with the copyright policy of the publisher. |
en |
dc.rights.uri |
https://researchspace.auckland.ac.nz/docs/uoa-docs/rights.htm |
en |
dc.subject |
Humans |
en |
dc.subject |
Amyotrophic Lateral Sclerosis |
en |
dc.subject |
Oligonucleotides, Antisense |
en |
dc.subject |
Drug Delivery Systems |
en |
dc.subject |
Molecular Targeted Therapy |
en |
dc.title |
Antisense oligonucleotide therapies for Amyotrophic Lateral Sclerosis: Existing and emerging targets. |
en |
dc.type |
Journal Article |
en |
dc.identifier.doi |
10.1016/j.biocel.2019.03.009 |
en |
pubs.begin-page |
149 |
en |
pubs.volume |
110 |
en |
dc.rights.holder |
Copyright: The author |
en |
pubs.end-page |
153 |
en |
pubs.publication-status |
Published |
en |
dc.rights.accessrights |
http://purl.org/eprint/accessRights/RestrictedAccess |
en |
pubs.subtype |
Research Support, Non-U.S. Gov't |
en |
pubs.subtype |
Review |
en |
pubs.subtype |
Journal Article |
en |
pubs.elements-id |
767366 |
en |
pubs.org-id |
Science |
en |
pubs.org-id |
Biological Sciences |
en |
dc.identifier.eissn |
1878-5875 |
en |
pubs.record-created-at-source-date |
2019-03-25 |
en |
pubs.dimensions-id |
30904737 |
en |