The General Hospital Corporation;The United States of America, as Represented by the Secretary, Department of Health and Human Servic
发明人:
Susan A. Slaugenhaupt,Graham Johnson,William D. Paquette,Wei Zhang,Juan Marugan
申请号：
US16877254
公开号：
US20200283441A1
申请日:
2020.05.18
申请国别(地区):
US
年份:
2020
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.
