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Reparative dysfunction during muscle regeneration in Duchenne muscular dystrophy: therapeutic approaches to modulating the muscle environment and muscle stem cells
(University of Missouri--Columbia, 2019)
[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT REQUEST OF AUTHOR.] Duchenne muscular dystrophy (DMD) is a lethal muscular dystrophy resulting from functional loss of the dystrophin protein, a critical sub-sarcolemmal ...
Delineation of Duchenne muscular dystrophy gene therapy using genetically engineered mice
(University of Missouri--Columbia, 2016)
Duchenne muscular dystrophy (DMD) is a genetically inherited debilitating muscle disorder affecting young boys due to the loss of dystrophin protein in muscle and the heart. Affected individuals lose their mobility and ...
Evaluation of calcium handling and dwarf open reading frame AAV gene therapy in Duchenne muscular dystrophy
(University of Missouri--Columbia, 2022)
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease. Increased concentrations of cytosolic calcium in dystrophic muscle cells has been implicated in DMD pathogenesis. In healthy muscle, calcium from ...
Duchenne muscular dystrophy gene therapy in the canine model
(University of Missouri--Columbia, 2018)
[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT AUTHOR'S REQUEST.] Duchenne muscular dystrophy (DMD) is a devastating muscle wasting disease caused by loss of function mutations in the dystrophin gene, resulting in the ...
Rational design of split gene vectors to expand the packaging capacity of adeno-associated viral vectors
(University of Missouri--Columbia, 2007)
Adeno associated viruses (AAV) have recently been demonstrated as a very promising gene delivery vehicle. But the limited packaging capacity of AAV vectors (4̃.7kb) hinders their application for diseases involving large ...
Development of gene therapy for Duchenne muscular dystrophy heart disease in the MDX mouse model
(University of Missouri--Columbia, 2010)
[ACCESS RESTRICTED TO THE UNIVERSITY OF MISSOURI AT REQUEST OF AUTHOR.] Duchenne muscular dystrophy (DMD) is a fatal genetic muscle disease with no cure. DMD results from mutations in a critical muscle protein called ...
Gene replacement restores the contractile and passive properties of skeletal muscle in murine models of Duchenne muscular dystrophy
(University of Missouri--Columbia, 2012)
[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 ...