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Spinal muscular atrophy treatment via targeting smn2 catalytic core
Iowa State University Patents
  • Ravindra N. Singh, Iowa State University
  • Natalia N. Singh, Iowa State University
Document Type
Patent Application
Publication Date
The present invention is directed to methods and compositions for blocking the effect of the intronic inhibitory splicing region of intron 7 of the SMN2 gene. The compositions and methods of the instant invention include short oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target sites in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The short target regions are 8-mers and 5-mers and also include the identification of a single nucleotide base that is essential for initiating a long distance stearic inhibitory interactions as well as novel targets distant from intron 7 which block the intronic inhibitory splicing of the same. These short target regions and concomitant inhibitory blocking oligonucleotides are less expensive and easier to manufacture and are small enough to cross the blood brain barrier.
Patent Number
US 2011/0269820 A1
Iowa State University Research Foundation, Inc.
Application Number
Date Filed
File Format
Citation Information
Ravindra N. Singh and Natalia N. Singh. "Spinal muscular atrophy treatment via targeting smn2 catalytic core" (2011)
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