THE GENERAL HOSPITAL CORPORATION D/B/A MASSACHUSETTS GENERAL HOSPITAL;RANA THERAPEUTICS INC.
发明人:
KRIEG, ARTHUR M.,SUBRAMANIAN, ROMESH,MCSWIGGEN, JAMES,LEE, JEANNIE T.
申请号：
EP13790819
公开号：
EP2850186A4
申请日:
2013.05.16
申请国别(地区):
EP
年份:
2016
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.
