found that platelets have a disorganized cytoskeleton due to dysfunctional dystrophin
that may result in increased bleeding during surgery.
The drug aims to arrest the progression of the disease by enabling dystrophin
production using other components of the gene, the institute and the drugmaker said Thursday.
"We believe this is the defect because we found that there's about half as much dystrophin
protein in affected pigs as there is in their normal siblings," says Nonneman, who's in the USMARC Reproduction Research Unit.
Much like dystrophin
, human micro-utrophin can support muscle fiber strength and prevent muscle fiber injury throughout the body.
Mutation detection technique is labor intensive, expensive and sophisticated, so alternative, reliable and feasible technique is linkage analysis by using STR in the introns of dystrophin
Three of the participants in the higher dose cohort showed dystrophin
levels exceeding 18 per cent of those found in normal muscle.
Key Words: Dystrophin
, Immunohistochemistry (IHC), Muscular Dystrophies.
Key Words: Ducheme muscular dystrophy, Becker's muscular dystrophy, dystrophin
, frozen sections.
Generation of functional dystrophin
is considered critical for successful treatment of DMD, AVI said.
Next, the group implanted these muscle stem cells from normal mice into mice lacking the gene to make dystrophin
. Within a couple of weeks of the transplant, mice with the stem cell transplant had markedly improved muscle fibers.
Next, the group implanted muscle stem cells from normal mice into mice lacking the gene to make dystrophin
. The mice have the same genetic defect as that implicated in Duchenne muscular dystrophy, Conboy says.
The diagnosis was established from clinical features consistent with DMD or BMD, absent or altered dystrophin
production (1) (as determined by immunofluorescence assay or western blot analysis), and/or a clear X-linked family history of the disease (2).