Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy.

Abstract excerpt

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...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Exons
• Share to SMN2 gene
• Share to Models, Molecular