特殊(脂质)递送:通过对脂质进行大量头基修饰,研究了可电离脂质 p K a在脂质纳米粒子体内递送 siRNA 中的作用。脂质P的的紧密相关ķ一个 值和鼠标FVII基因(FVII ED的沉默50)被发现,具有最佳p ķ一个范围的6.2-6.5(见图)。本研究中最有效的阳离子脂质的 ED 50水平在小鼠中约为 0.005 mg kg -1,在非人类灵长类动物中低于 0.03 mg kg -1。
LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS
申请人:Manoharan Muthiah
公开号:US20120058144A1
公开(公告)日:2012-03-08
The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structures XIV or XVII.
NOVEL LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS
申请人:MANOHARAN Muthiah
公开号:US20120095075A1
公开(公告)日:2012-04-19
The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structure:
本发明提供了有利于用于体内向细胞传递治疗剂的脂质。具体而言,本发明提供具有以下结构的脂质:
Structure-engineered guide RNA
申请人:GenEdit Inc.
公开号:US11268092B2
公开(公告)日:2022-03-08
The invention provides a modified guide RNA comprising an extension sequence, as well as related compositions and methods.
本发明提供了一种经修饰的引导RNA,其包含延伸序列,以及相关的组合物和方法。
METHODS AND COMPOSITIONS FOR DELIVERY OF NUCLEIC ACIDS
申请人:The University of British Columbia
公开号:EP2512449B1
公开(公告)日:2019-08-07
COMPOSITIONS AND METHODS FOR TARGET NUCLEIC ACID MODIFICATION