The technique partially restored not only functional dystrophin expression, but also
dystrophic muscle function.
Topics such as the mechanisms of cell injury in normal and
dystrophic muscle, compensatory muscle regeneration and hypertrophy, and the effects of various therapies or voluntary exercise on muscle repair, satellite cell activation, muscle growth, bone density and age - related atrophy are examined using a large variety of cellular, molecular and whole - animal in vivo assays of function.
«VBP15 has an additional property of addressing membrane defects in
dystrophic muscle cells,» remarked Kanneboyina Nagaraju, DVM, PhD, the lead author of the study, and principal investigator in the Center for Genetic Medicine at Children's National.
The study «Elastase levels and activity are increased in
dystrophic muscle and impair myoblast cell survival, proliferation and differentiation» is published in the journal Scientific Reports.
This approach has proved successful in disrupting multiple genes simultaneously in neurons and in editing the Dmdmdx mutation in muscle, thereby restoring dystrophin protein expression and partially alleviating
the dystrophic muscles» functional deficiencies.
Read the full article, «Branched fibers from old fast - twitch
dystrophic muscles are the sites of terminal damage in muscular dystrophy,» published ahead of print in the American Journal of Physiology — Cell Physiology.
Not exact matches
Muscle satellite cells from GRMD
dystrophic dogs are not phenotypically distinguishable from wild type satellite cells in ex vivo culture.
Working in
dystrophic mice while searching for a cure for Duchenne muscular dystrophy (DMD), Dr. Huard's laboratory team first identified a unique population of
muscle - derived stem cells with the ability to repair
muscle 8 years ago.
In vivo gene editing in
dystrophic mouse
muscle and
muscle stem cells.
Goudenege S, Lebel C, Huot NB, et al., Myoblasts derived from normal hESCs and
dystrophic hiPSCs efficiently fuse with existing
muscle fibers following transplantation.