Campus Units

Biomedical Sciences, Center for Advanced Host Defenses, Immunobiotics and Translational Comparative Medicine

Document Type

Article

Publication Version

Accepted Manuscript

Publication Date

9-2015

Journal or Book Title

Future Medicinal Chemistry

Volume

7

Issue

13

First Page

1793

Last Page

1808

DOI

10.4155/fmc.15.101

Abstract

Spinal muscular atrophy (SMA) is a major neurodegenerative disorder of children and infants. SMA is primarily caused by low levels of SMN protein owing to deletions or mutations of the SMN1 gene. SMN2, a nearly identical copy of SMN1, fails to compensate for the loss of the production of the functional SMN protein due to predominant skipping of exon 7. Several compounds, including antisense oligonucleotides (ASOs) that elevate SMN protein from SMN2 hold the promise for treatment. An ASO-based drug currently under Phase III clinical trial employs intronic splicing silencer N1 (ISS-N1) as its target. Cumulative studies on ISS-N1 reveal a wealth of information with significance to the overall therapeutic development for SMA. Here, the authors summarize the mechanistic principles behind various antisense targets currently available for SMA therapy.

Comments

This is a manuscript of an article published as Singh, Natalia N., Brian M. Lee, Christine J. DiDonato, and Ravindra N. Singh. "Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy." Future Medicinal Chemistry 7, no. 13 (2015): 1793-1808. DOI: 10.4155/fmc.15.101. Posted with permission.

Copyright Owner

Future Science Group, Ltd.

Language

en

File Format

application/pdf

Published Version

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