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Generated: September 22, 2019

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Patent: 8,586,559

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Summary for Patent: 8,586,559
Title:Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
Abstract: The present invention is directed to methods and compositions capable of blocking the inhibitory effect of a newly-identified intronic inhibitory sequence element, named ISS-N1 (for \"intronic splicing silencer\"), located in the SMN2 gene. The compositions and methods of the instant invention include oligonucleotide reagents (e.g., oligoribonucleotides) that effectively target the SMN2 ISS-N1 site in the SMN2 pre-mRNA, thereby modulating the splicing of SMN2 pre-mRNA to include exon 7 in the processed transcript. The ISS-N1 blocking agents of the invention cause elevated expression of SMN protein, thus compensating for the loss of SMN protein expression commonly observed in subjects with spinal muscular atrophy (SMA).
Inventor(s): Singh; Ravindra N. (Shrewsbury, MA), Singh; Natalia N. (Shrewsbury, MA), Singh; Nirmal K. (Temple, TX), Androphy; Elliot J. (Natick, MA)
Assignee: University of Massachusetts (Boston, MA)
Application Number:13/329,926
Patent Claims:see list of patent claims

Details for Patent 8,586,559

Applicant Tradename Biologic Ingredient Dosage Form BLA Number Approval Date Patent No. Assignee Estimated Patent Expiration Status Orphan Source
Schering INTRON A interferon alfa-2b VIAL 103132 001 1986-06-04   Try a Free Trial University of Massachusetts (Boston, MA) 2024-12-03 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 002 1986-06-04   Try a Free Trial University of Massachusetts (Boston, MA) 2024-12-03 RX search
Schering INTRON A interferon alfa-2b VIAL 103132 003 1986-06-04   Try a Free Trial University of Massachusetts (Boston, MA) 2024-12-03 RX search
>Applicant >Tradename >Biologic Ingredient >Dosage Form >BLA >Number >Approval Date >Patent No. >Assignee >Estimated Patent Expiration >Status >Orphan >Source

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