Broad Therapy For Muscular Dystrophy
Muscular dystrophy is a group of inherited muscular disorders that are characterized by progressive skeletal muscle weakness, defects in muscle proteins, and the death of muscle cells and tissue. Recent studies have shown that a number of genes can prevent muscle damage, even though they do not fix the genetic defect that causes the disease. However, these surrogate gene therapies have had limited applicability across different forms of muscular dystrophy.
High expression of the protein Galgt2, which alters the expression and properties of other proteins expressed in skeletal muscle, lessens the symptoms of muscular dystrophy in models with decreased expression of either dystrophin or laminin. Xu et al examined the effects of Galgt2 overexpression in a mouse model of limb girdle muscular dystrophy 2D. Galgt2 overexpression resulted in lower levels of muscle damage, and galgt2 gene therapy protected muscle fibers from injury. Increasing Galgt2 expression may therefore have therapeutic benefits in a broad range of muscular dystrophies.
Dr. Martin and colleagues "have developed [a] gene therapy approach to overexpress the Galgt2 cDNA. …Future work will entail developing methods to allow systemic delivery of such gene therapy vectors using the human Galgt2 cDNA driven by muscle- or muscle/heart-specific promoters. … [They also plan to] identif[y] drugs that would increase [Galgt2] expression in muscle … to stimulate the therapeutic effects of Galgt2 over-expression."
Xu R, DeVries S, Camboni M, Martin PT: Overexpression of Galgt2 reduces dystrophic pathology in the skeletal muscles of alpha sarcoglycan-deficient mice. Am J Pathol 2009, 174: 2645-2657
American Journal of Pathology
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