GENOMIC SAFE HARBORS FOR GENETIC THERAPIES IN HUMAN STEM CELLS AND ENGINEERED NANOPARTICLES TO PROVIDE TARGETED GENETIC THERAPIES

摘要

Genomic safe harbors (GSH) for genetic therapies in human stem cells and engineered nanoparticles to provide targeted genetic therapies are described. The GSH and/or associated nanoparticles can be used to safely and efficiently treat a variety of genetic, infectious, and malignant diseases.