公开(公告)号：US20170080107A1

公开(公告)日：2017-03-23

申请号：US15272107

申请日：2016-09-21

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh Chivukula ,  Rachel Wilkie-Grantham ,  Kiyoshi Tachikawa

IPC: A61K48/00

CPC classification number: A61K48/005 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/323 ,  C12N2320/34

Abstract: This invention encompasses compounds, structures, compositions and methods for therapeutic guide molecules that direct CRISPR gene editing. A guide molecule for directing gene editing can be allele selective, or disease allele selective, and can exhibit reduced off target activity. A guide molecule can be composed of monomers, including UNA monomers, nucleic acid monomers, and modified nucleotides, wherein the compound is targeted to a genomic DNA. The guide molecules of this invention can be used as active ingredients for editing or disrupting a gene in vitro, ex vivo, or in vivo.

公开(公告)号：US10369232B2

公开(公告)日：2019-08-06

申请号：US15272107

申请日：2016-09-21

Applicant: Arcturus Therapeutics, Inc.

Inventor： Padmanabh Chivukula ,  Rachel Wilkie-Grantham ,  Kiyoshi Tachikawa

IPC: A61K48/00 ,  C12N15/113 ,  C12N15/90

Abstract: This invention encompasses compounds, structures, compositions and methods for therapeutic guide molecules that direct CRISPR gene editing. A guide molecule for directing gene editing can be allele selective, or disease allele selective, and can exhibit reduced off target activity. A guide molecule can be composed of monomers, including UNA monomers, nucleic acid monomers, and modified nucleotides, wherein the compound is targeted to a genomic DNA. The guide molecules of this invention can be used as active ingredients for editing or disrupting a gene in vitro, ex vivo, or in vivo.