Gene replacement restores the contractile and passive properties of skeletal muscle in murine models of Duchenne muscular dystrophy
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[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT AUTHOR'S REQUEST.] Duchenne muscular dystrophy (DMD) is a lethal disease caused by the loss of the dystrophin protein. Loss of mobility is a key clinical presentation in DMD. It is believed that deterioration in the mechanical properties (contractile and passive properties) of skeletal muscle contribute to reduction in mobility. These two sets of properties are inseparable aspects of muscle function. To improve mobility of patients, both contractile and passive properties must be restored. Gene therapy holds great promise for treating DMD. Restoration of dystrophin expression using gene replacement strategies has improved histopathology and increased muscle force in mouse models of DMD. However, it is not yet known if gene replacement can also ameliorate defects of the passive properties. To address this concern, I performed comprehensive studies that provided new information on the passive properties changes in skeletal muscles of murine models of DMD, and have also offered new insights on how gene replacement may help improve the passive muscle properties in DMD. Furthermore, these studies provided support to further develop mini- and micro-dystrophin gene therapy to improve loss of mobility in DMD patients.
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