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Therapeutic approaches for Duchenne muscular dystrophy

Therapeutic approaches for Duchenne muscular dystrophy

Paperback

Genetics

ISBN10: 3659286834
ISBN13: 9783659286834
Publisher: Lap Lambert Academic Pub
Published: May 7 2014
Pages: 140
Weight: 0.47
Height: 0.33 Width: 6.00 Depth: 9.00
Language: English
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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Genetics