A possible strategy for gene therapy in patients with the most severe form of the muscle-wasting condition congenital muscular dystrophy (MCMD) is reported in this week’s Nature (Vol. 413, No. 6853, 20 Sep 2001).
In a mouse model of the disease, Markus A. Ruegg of the University of Basel, Switzerland, and colleagues, remedied the lack of a muscle scaffold protein with a specially engineered miniature form (miniagrin) of another muscle protein (agrin) featuring only that protein’s functional regions.
That the technique restored the rodents’ muscle function suggests that drugs to raise agrin levels might alleviate symptoms of muscular dystrophy. It also demonstrates how small versions of large proteins can be useful for gene therapy, especially as they are "less likely to cause immunological responses," as Ruegg’s team points out.
Markus A. Ruegg
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