Invention Grant
US08802642B2 Spinal muscular atrophy treatment via targeting SMN2 catalytic core
有权
通过靶向SMN2催化核心的脊髓肌萎缩治疗
- Patent Title: Spinal muscular atrophy treatment via targeting SMN2 catalytic core
- Patent Title (中): 通过靶向SMN2催化核心的脊髓肌萎缩治疗
-
Application No.: US13093958Application Date: 2011-04-26
-
Publication No.: US08802642B2Publication Date: 2014-08-12
- Inventor: Ravindra Singh , Natalia Singh
- Applicant: Ravindra Singh , Natalia Singh
- Applicant Address: US IA Ames
- Assignee: Iowa State University Research Foundation, Inc.
- Current Assignee: Iowa State University Research Foundation, Inc.
- Current Assignee Address: US IA Ames
- Agency: McKee, Voorhees & Sease
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C12N15/85 ; C07H21/02 ; C07H21/04

Abstract:
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.
Public/Granted literature
- US20110269820A1 SPINAL MUSCULAR ATROPHY TREATMENT VIA TARGETING SMN2 CATALYTIC CORE Public/Granted day:2011-11-03
Information query