ISU
Home
ISURF/OIPTT
Home
ISURFtech
Home
Advanced
Search
RSS
Feeds
SMN2 Catalytic Core: A Novel Approach for Spinal Muscular Atrophy Treatment
Category(s):
For Information, Contact:
Dario Valenzuela
Senior Commercialization Manager, Life Sciences
515-294-4740
licensing@iastate.edu
Web Published:
5/5/2015
ISURF #
3810
Summary:
Iowa State University researchers have identified a new approach for the development of treatments for spinal muscular atrophy using an antisense oligonucleotide.

Description:
Spinal muscular atrophy (SMA) is a neurodegenerative disorder that affects the control of muscle movement and is a leading genetic cause of infant mortality.  SMA is divided into four subtypes based on disease severity and the age at which it manifests. In humans, there are two nearly identical copies of the Survival Motor Neuron (SMN) gene, SMN1 and SMN2.  Loss of SMN1 in conjunction with skipping of SMN2 exon 7 in pre-mRNA splicing—which results in a truncated, unstable SMN—leads to SMA.  Strategies to correct the splicing aberration in SMN2 are believed to hold promise for a cure for SMA, and have included screening many small compounds as well as the use of short antisense oligonucleotides (ASO).  To date, however, no small compounds or ASO have been identified that can correct the SMN2 splicing defect.  As part of an ongoing research program in SMA, ISU researchers have now identified a novel short antisense oligonucleotide that corrects the aberrant splicing in SMN2.  This ASO may have utility as a potential treatment for SMA since it offers low cost synthesis, high target specificity, ease of manipulation and potential to cross the blood brain barrier.

Advantage:
• Low cost of synthesis due to small size
• High target specificity and low off-target effect
• Effective at very low dose
• Amenable to modifications for transport across blood brain barrier
• Unique mechanism of splicing regulation through modulation of catalytic core of spliceosome
Application:
Development of drugs for the treatment of spinal muscular atrophy

References:
Patent Information:
*To see the full version of the patent(s), follow the link below, then click on "Images" button.
Country Serial No. Patent No. Issued Date
United States 13/093,958 8,802,642* 8/12/2014
United States 14/134,057 9,217,147* 12/22/2015


Direct Link: