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Antisense-mediated exon skipping for Duchenne muscular dystrophy. Development of an antisense-mediated exon skipping therapy for Duchenne muscular dystrophy Making sense out of nonsense

Автор:   Annemieke A.
мягкая обложка
120 страниц
2009 год
Издательство:   Книга по Требованию
Цена:
2005 руб
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Описание:Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disease for which there is currently no cure. It provides an overview of potential therapies for Duchenne muscular dystrophy and describes how antisense-mediated modulation of splicing can be used for other therapeutic and research purposes. This thesis focuses on the development of a promising new therapy, so called antisense-mediated exon skipping, that aims to restore the disrupted dystrophin reading frame to allow generation of internally deleted, but partly functional dystrophin proteins, as found in the less severe Becker muscular dystrophy. The thesis describes the DMD gene, the dystrophin protein, diseases associated with mutations in the DMD gene and animal models. The disease is caused by mutations in the DMD gene that completely abolish the function of the protein dystrophin. Finally it gives a detailed overview of the rationale and the development of the exon skipping therapy for Duchenne muscular dystrophy.


Orthomol Immun Junior Сила иммунитета на страже Вашего ребенка Orthomol Immun Junior Сила иммунитета на страже Вашего ребенка

2990 руб
Бесплатная доставка


The Black Swan: The Impact of the Highly Improbable

I later saw the exact same illusion of understanding in business success and the financial markets. History Does Not Crawl, It Jumps Later, upon replaying the wartime events in my memory as I formulated my ideas on the perception of random events, I developed the governing impression that our minds are wonderful explanation machines, capable of making sense out of almost anything, capable of mounting explanations for all manner of phenomena, and generally incapable of accepting the idea of unpredictability. These events were unexplainable, but intelligent people thought they were capable of providing convincing explanations for themБЂ”after the fact. Furthermore, the more intelligent the person, the better sounding the explanation. WhatБЂ™s more worrisome is that all these beliefs and accounts appeared to be logically coherent and devoid of inconsistencies. So I left the place called Lebanon as a teenager, but, since a large number of my relatives and friends remained there, I kept coming back to visit, especially during the hostilities ...»

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