Sergey V. Paushkin,Nikolai Naryshkin,Charles Romfo,Ellen Welch
申请号：
US12994517
公开号：
US08633019B2
申请日:
2009.05.27
申请国别(地区):
US
年份:
2014
代理人:
摘要：
The present invention provides nucleic acid constructs, methods for identifying and validating compounds that increase the inclusion of exon 7 of SMN2 into mRNA transcribed from the SMN2 gene, compounds and pharmaceutical compositions that increase levels of SMN protein produced from the SMN2 gene, and methods for use thereof in treating of SMA.
