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Автор: Ingrid E.C.Verhaart
Год: 2014
Издание:
LAP Lambert Academic Publishing
Страниц: 140
ISBN: 9783659286834
Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder for which at the moment is no cure. It is caused by mutations in the DMD gene, leading to a complete absence of the dystrophin protein. Dystrophin is an important protein for stabilization of muscle cells. This books gives an overview of several strategies aiming to (partly) correct the underlying genetic defect and restoring dystrophin synthesis. Especially attention is given to antisense oligonucleotide mediated exon skipping. Furthermore various therapeutical strategies trying to alleviate the secondary symptoms are discussed.
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