Inflammation and response to steroid treatment in limb-girdle muscular dystrophy
Allamand et al., "Mutations in the proteolytic enzyme calpain 3 cause limb-girdle muscular dystrophy
type 2A," Cell, vol.
Westra et al., "Limb-girdle muscular dystrophy
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Abbreviations ALT: Atypical lipomatous tumor WDLS: Well-differentiated liposarcoma LGMD: Limb-girdle muscular dystrophy
Genetic defects in the gene that codes for the protein result in two types of muscular dystrophy: Limb-girdle muscular dystrophy
type 2B (LGMD2B) and Miyoshi muscular dystrophy 1 (MMDI).
Instead, scientists in this study "reprogrammed" adult cells from patients with limb-girdle muscular dystrophy
into stem cells and were able to induce them to differentiate into mesoangioblast-like cells.
In a separate study, he and his associates transferred the large limb-girdle muscular dystrophy
type 2D gene via the adeno-associated virus (Ann.
Injections of the alphasarcoglycan gene in the foot muscles of three patients with limb-girdle muscular dystrophy
type 2D produced 4-5 times the amount of the alpha-sarcoglycan protein, compared with saline injections.
Described as the first gene therapy trial in muscular dystrophy demonstrating promising findings, researchers from the University of Florida (UF), Nationwide Children's Hospital in Columbus, Ohio, and The Ohio State University report how they safely transferred a gene to produce a protein necessary for healthy muscle fiber growth into three teenagers with limb-girdle muscular dystrophy
. The findings, which have relevance to genetic disorders beyond muscular dystrophy as well as conditions in which muscles atrophy, were published online recently in the Annals of Neurology.
Washington, April 16 (ANI): Researchers from the University of Florida (UF), Nationwide Children's Hospital in Columbus, Ohio, and The Ohio State University have safely transferred a gene to produce a protein necessary for healthy muscle fibre growth into three teenagers with limb-girdle muscular dystrophy