THE GENERAL HOSPITAL CORPORATION D/B/A MASSACHUSETTS GENERAL HOSPITAL;RANA THERAPEUTICS; INC.;RANA THERAPEUTICS, INC.
发明人:
KRIEG, ARTHUR M.,SUBRAMANIAN, ROMESH,MCSWIGGEN, JAMES,LEE, JEANNIE T.
申请号：
SG11201407483Y
公开号：
SG11201407483YA
申请日:
2013.05.16
申请国别(地区):
SG
年份:
2014
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.
