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。
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。
STRUCTURE-ENGINEERED GUIDE RNA
申请人:GenEdit Inc.
公开号:US20190218547A1
公开(公告)日:2019-07-18
The invention provides a modified guide RNA comprising an extension sequence, as well as related compositions and methods.
COMPOSITIONS AND METHODS FOR TARGET NUCLEIC ACID MODIFICATION
申请人:GenEdit Inc.
公开号:US20200017852A1
公开(公告)日:2020-01-16
The present disclosure provides methods and compositions utilizing CRISPR systems wherein the guide RNA and the donor polynucleotide are modified.
MODIFIED CPF1 GUIDE RNA
申请人:GenEdit Inc.
公开号:US20200299689A1
公开(公告)日:2020-09-24
The invention provides a nucleic acid comprising a Cpf1 crRNA, a processing sequence 5′ of the Cpf1 crRNA, and an extension sequence 5′ of the processing sequence. The invention also provides a composition comprising the nucleic acid, a carrier, and optionally Cpf1. Additionally, the invention provides method of genetically modifying a eukaryotic target cell, comprising contacting the eukaryotic target cell with the nucleic acid or the composition to genetically modify a target nucleic acid in the cell.