Regeneron Pharmaceuticals, Inc.
TRANSCRIPTION MODULATION IN ANIMALS USING CRISPR/CAS SYSTEMS DELIVERED BY LIPID NANOPARTICLES

Abstract:

Lipid nanoparticles comprising CRISPR/Cas synergistic activation mediator system components together in the same lipid nanoparticle and methods of using such lipid nanoparticles to increase expression of target genes in vivo and ex vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo and ex vivo are provided.