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.
Copyright Owner
Future Science Group, Ltd.
Copyright Date
2015
Language
en
File Format
application/pdf
Recommended Citation
Singh, Natalia N.; Lee, Brian M.; DiDonato, Christine J.; and Singh, Ravindra N., "Mechanistic principles of antisense targets for the treatment of Spinal Muscular Atrophy" (2015). Biomedical Sciences Publications. 59.
https://lib.dr.iastate.edu/bms_pubs/59
Included in
Amino Acids, Peptides, and Proteins Commons, Medical Biochemistry Commons, Medical Genetics Commons
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.