United States Department of Health and Human Services;The General Hospital Corporation
发明人:
Susan A. Slaugenhaupt,Graham Johnson,William D. Paquette,Wei Zhang,Juan Marugan
申请号：
US15543826
公开号：
US20180118748A1
申请日:
2016.01.15
申请国别(地区):
US
年份:
2018
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.
