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Stem Cell Gene Therapy Program

The Stem Cell Gene Therapy Program aims to develop efficient and safe gene therapeutics for sickle cell disease and hemoglobinopathies. Insulator elements are a critical component of gene therapy vectors, and both prevent transgene silencing as well as protect genes surrounding the integration sites from the regulatory influences of vector control regions.

We provide the Genomics Core Facility for the Stem Cell Gene Therapy Program Project. In addition to assisting diverse investigators with high-throughput genomics and informatics needs, we have a focus on identification of new insulator elements that contain the CTCF protein.