Generation Bio Co.
IDENTIFYING AND CHARACTERIZING GENOMIC SAFE HARBORS (GSH) IN HUMANS AND MURINE GENOMES, AND VIRAL AND NON-VIRAL VECTOR COMPOSITIONS FOR TARGETED INTEGRATION AT AN IDENTIFIED GSH LOCI

Abstract:

The technology described herein relates to methods, compositions and in silico screening approaches for identifying and validating genomic safe harbors (GSHs) in mammalian genomes, including human genomes. Another aspects relates to recombinant nucleic acid vectors, including non-viral and viral vectors comprising a portion of the GSH loci, or gRNA sequences specific to a GSH loci, and methods for use of the vectors for insertion of a gene of interest into a GSH loci.