Development of Dysferlin-Deficient Mouse Models Suitable for Non-Invasive Imaging

Thomas Rando, MD, PhD

Stanford University (Palo Alto, CA)

Dr. Rando is Associate Professor in the Department of Neurology at the Stanford University School of Medicine and Chief of Neurology at the Palo Alto VA Medical Center (Palo Alto, CA).

Past Projects

Objective: 
Generate strains of mice on which non-invasive imaging studies of disease progression in dysferlinopathy can be conducted on a living animal

We are working to generate strains of mice in which the degenerative and regenerative phases of muscular dystrophies can be assessed non-invasively, primarily to be used in experimental therapeutic trials. One of the major limitations to the study of therapeutic agents for the treatment of muscular dystrophies such as LGMD2B is the absence of reliable markers of disease activity in the living mouse. We aim to generate strains of mice in which the luciferase gene will be used either to register degeneration (by loss of activity when myofibers die) or regeneration (when muscle progenitors contribute to new myofibers formation), allowing these processes to be monitored by bioluminescence imaging.