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Evaluation of micro-dystrophin based and dystrophin-independent AAV gene therapies for Duchenne Muscular Dystrophy
(University of Missouri--Columbia, 2020)
the impact of hinge 1 (H1) and hinge 4 (H4) regions in the function of a micro-dystrophin ([micro]Dys) construct. Two novel micro-dystrophins were engineered by complete deletion of either hinge 1 or hinge 4 and packaged in AAV9. Three separate groups of 3...
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 genes such as those...
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 ...
Four-limb IMU sensors for canine gait analysis
(University of Missouri--Columbia, 2019)
of our method, we conducted synchronized gait tests with the GAIT4Dog, a commercially available system. Our algorithm detected the swing start event in both front and hind limbs with high accuracy with a mean error [plus-minus] standard deviation of 0...
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)
the environment by improving fibrosis, stiffness, and fiber orientation, which significantly improved early muscle regeneration but not late regeneration (2 [greater than] and [less than] 4 months post-transplant) via enhancing muscle stem cells differentiation...