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Development of gene and cell therapy fields
Cell, tissue, and gene therapies.
Our research is focused on development of novel strategies for gene and cell therapy, using gene therapy and regenerative medicine approaches. We are working to improve the clinical condition of patients suffering from genetic disorders like Duchenne muscular dystrophy and limb girdle muscular dystrophy types 2A, 2B, and 2D. To develop a stem cell therapy for muscular dystrophy, we are using induced pluripotent stem cells (iPSC) derived from patients, using non-integrating reprogramming methods. We carry out precise genome engineering to edit mutations and add therapeutic sequences to the iPSC genome, using CRISPR/Cas9, homologous recombination, and phage integrases to mediate sequence-specific gene editing and genomic integration. The corrected cells are differentiated into muscle precursors and transplanted back to the patient, where they can engraft and produce healthy muscle fibers. We are also developing methods to add therapeutic genes and correct mutations directly in the target muscle tissue. We have demonstrated delivery of naked plasmid DNA to muscles through the vascular system and by electroporation. We are attempting to perform genetic engineering on muscle stem cells while they are resident in the tissue.We are currently testing these approaches in mouse models of muscular dystrophy. If successful, these types of therapies will provide new options for patients suffering from muscular dystrophy and other genetic diseases. They may also provide new possibilities for treatment of other common diseases and conditions, including normal aging.