United States Department of Health and Human Services;The General Hospital Corporation
发明人:
Slaugenhaupt, Susan A.,Johnson, Graham,Paquette, William D.,Zhang, Wei,Marugan, Juan
申请号：
AU2016206589
公开号：
AU2016206589A1
申请日:
2016.01.15
申请国别(地区):
AU
年份:
2017
代理人:
摘要：
Provided herein are compounds useful for improving mRNA splicing in a cell. Exemplary compounds provided herein are useful for improving mRNA splicing in genes comprising at least one exon ending in the nucleotide sequence CAA. Methods for preparing the compounds and methods of treating diseases of the central nervous system are also provided.
