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Progress in therapeutic antisense applications for neuromuscular disorders

Neuromuscular disorders are a frequent cause of chronic disability in man. They often result from mutations in single genes and are thus, in principle, well suited for gene therapy. However, the tissues involved (muscle and the central nervous system) are post-mitotic, which poses a challenge for mo...

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Autors principals: Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2010
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2987179/
https://ncbi.nlm.nih.gov/pubmed/19809477
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/ejhg.2009.160
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