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Article
Mechanistic principles of antisense targets for the treatment of Spinal Muscular Atrophy
Future Medicinal Chemistry
  • Natalia N. Singh, Iowa State University
  • Brian M. Lee, Iowa State University
  • Christine J. DiDonato, Northwestern University
  • Ravindra N. Singh, Iowa State University
Document Type
Article
Publication Version
Accepted Manuscript
Publication Date
9-1-2015
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
Citation Information
Natalia N. Singh, 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 Vol. 7 Iss. 13 (2015) p. 1793 - 1808
Available at: http://works.bepress.com/ravindra-singh/32/