Regulation and Manipulation of Cellular and Viral Genes
Editing the Genome
Genome editing technology is advancing at a breathtaking rate. In particular, programmable nucleases based on the CRISPR/Cas9 system are enabling basic and translational research that holds promise for the development of new therapeutics. MCCB researchers are applying this new technology across a number of different platforms to model and treat human diseases, including cancer. In particular, these new tools provide the possibility for gene correction in cases of congenital human disease. Indeed, collaborative efforts between MCCB researchers and other groups on campus are focused on correction of monogenic disorders, such as Huntington’s Disease and Chronic Granulomatous Disease, as well as the eradication of HIV from latent cellular reservoirs. In these cases, nuclease technology is being coupled with viral delivery systems through collaborations with the UMass Gene Therapy Center and RNA Therapeutics Institute to create gene correction therapies or autologous cell-replacement therapies.
Buttressing these nuclease development efforts is the Mutagenesis Core facility, which is housed in MCCB. This core facility provides UMass Chan Medical School research groups with access to the latest technologies available for introducing targeted genome alterations in human cells and various model organisms.
Our PIs that are conducting research in the area of Regulation and Manipulation of Cellular and Viral Genes:
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