Dr. Calos's research is focused on development of novel strategies for gene and cell therapy, using regenerative medicine approaches. They are working to improve the clinical condition of patients suffering from genetic disorders like Duchenne muscular dystrophy and limb girdle muscular dystrophy type 2B and degenerative diseases like Parkinson's disease.

To develop a therapy for muscular dystrophy, we are using induced pluripotent stem cells (iPSC) derived from patients, using non-viral reprogramming methods. They carry out precise genome engineering to add therapeutic sequences to the iPSC genome, using homologous recombination and phage integrases to mediate sequence-specific genomic integration. The corrected cells are differentiated into muscle precursors in vitro, then transplanted back to the patient, where they can engraft and produce healthy muscle fibers.

They 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 genetic diseases. They may also provide new possibilities for treatment of other common diseases and conditions, including normal aging.