公开(公告)号：US20200308603A1

公开(公告)日：2020-10-01

申请号：US16651918

申请日：2018-09-28

Applicant: Intellia Therapeutics, Inc.

Inventor： Morag H. Stewart ,  Markus P. Vallaster ,  Anthony Monti ,  Pooja K. Narendra ,  Quan Ho

IPC: C12N15/88 ,  C12N15/113 ,  C12N5/0789

Abstract: The present disclosure relates to compositions and methods for introducing an mRNA into stem cells, such as HSPCs, and for delivering gene editing components to such cells in vitro. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 complex in HSPCs, and methods and delivery systems for achieving such gene modification in HSPCs.