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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.…mehr

Produktbeschreibung
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.
Autorenporträt
Ingrid Verhaart (Eindhoven, the Netherlands, 19th of March 1986) studied Biomedical Sciences at the University of Leiden. In 2009, she became a PhD student in the group of Prof. dr. G.J.B. van Ommen and dr. A. Aartsma-Rus, resulting in 2014 in the thesis ¿Optimising antisense oligonucleotide-mediated exon skipping for Duchenne muscular dystrophy¿.