Therapeutic approaches for Duchenne muscular dystrophy (Paperback) (ISBN-13: 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.

Author: Ingrid E. C. Verhaart
ISBN-10: 3659286834
ISBN-13: 9783659286834
Publisher: LAP Lambert Academic Publishing
Language: English
Published: 05/07/2014
Pages: 140
Format: Paperback
Weight: 0.47lbs
Size: 9.00h x 6.00w x 0.33d