Functionally-modified oligonucleotides and subunits thereof
摘要
Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3' and/or 5'-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
申请公布号
AU2012340390(A1)
申请公布日期
2014.06.26
申请号
AU20120340390
申请日期
2012.11.15
申请人
SAREPTA THERAPEUTICS, INC.
发明人
HANSON, GUNNAR J.;WELLER, DWIGHT D.;CAI, BAO ZHONG;ZHOU, MING