FUNCTIONALLY-MODIFIED OLIGONUCLEOTIDES AND SUBUNITS THEREOF
申请人:Sarepta Therapeutics, Inc.
公开号:US20140330006A1
公开(公告)日:2014-11-06
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.
Disclosed are a compound represented by formula (I) and a pharmaceutically acceptable salt thereof,
wherein R
1
, R
2
, R
3
, R
4
, R
5
, W, n are defined as in the present application.
The present invention relates to xanthone analogs. Such compounds may be used in the treatment of bacterial infections.
本发明涉及黄酮类似物。这些化合物可用于治疗细菌感染。
Amine Cationic Lipids and Uses Thereof
申请人:Dicerna Pharmaceuticals, Inc.
公开号:US20140371293A1
公开(公告)日:2014-12-18
The present invention relates to lipid compounds and uses thereof. In particular, the compounds include a class of cationic lipids having an amine moiety, such as an amino-amine or an amino-amide moiety. The lipid compounds are useful for in vivo or in vitro delivery of one or more agents (e.g., a polyanionic payload or an antisense payload, such as an RNAi agent).
In 1,4‐Stellung unsymmetrisch disubstituierte Piperazine lassen sich N‐(bzw. N')‐mono‐ oder N,N'‐bisalkylieren. Bei ausreichendem Größenunterschied zwischen beiden Substituenten verlaufen Monoalkylierungen regiospezifisch.