Translate Bio MA, Inc.;The General Hospital Corporation
发明人:
Arthur M. Krieg,Romesh Subramanian,James McSwiggen,Jeannie T. Lee
申请号：
US15872684
公开号：
US20180298384A1
申请日:
2018.01.16
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2.
