Audrey Marie WINKELSAS,Kenneth Henry FISCHBECK,Christopher GRUNSEICH,George Geltmyier HARMISON, II
申请号：
US16750531
公开号：
US20200237931A1
申请日:
2020.01.23
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The present disclosure relates to agents, such as antisense oligonucleotides (oligomers) capable of binding to 5′； untranslated region (5′；UTR) sequence of survival motor neuron 2 (SMN2) transcripts, and thereby cause the cell to increase the production of SMN2 mRNA and total SMN protein.
