Rubius Therapeutics, Inc.
AMPLIFIABLE RNAS FOR THERAPEUTIC CELL SYSTEMS

Abstract:

This disclosure provides, among other things, amplifiable nucleic acid constructs for expressing a gene of interest in a cell, e.g., an erythroid cell. The amplifiable nucleic acid construct may contain the gene of interest and an RNA-dependent RNA polymerase (RdRP)-responsive 5' UTR, and may optionally further contain an RdRP-responsive 3' UTR. RdRP may also be provided, e.g., on the same construct or a different construct.