Intellia Therapeutics, Inc.
IN VITRO METHOD OF MRNA DELIVERY USING LIPID NANOPARTICLES

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.