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2014 Fiscal Year Final Research Report

Development of spinal muscular atrophy treatment by SMN2 gene splicing modification

Research Project

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Project/Area Number 24791060
Research Category

Grant-in-Aid for Young Scientists (B)

Allocation TypeMulti-year Fund
Research Field Pediatrics
Research InstitutionKobe University

Principal Investigator

NAKAGAWA Taku  神戸大学, 医学部附属病院, 助教 (40457073)

Co-Investigator(Renkei-kenkyūsha) NISHIO Hisahide  神戸大学, 大学院医学研究科, 教授 (80189258)
Project Period (FY) 2012-04-01 – 2015-03-31
Keywordsspinal muscular atrophy / SMA / splicing / SMN2
Outline of Final Research Achievements

Spinal muscular atrophy is a fatal congenital neuromuscular disease caused by SMN1 gene mutation. Effective treatment has not established yet. As a strategy of the treatment, modification for SMN2 splicing is thought to be a promising approach. We previously discovered compound A is effective to modify the SMN2 splicing using mini gene system. Subsequently, we tried to search for similar compound that would modify the SMN2 splicing safely. However, we didn't find the effective compounds for SMN2 splicing correction during the study period.

Free Research Field

小児科学

URL: 

Published: 2016-06-03  

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