A dystrophic Duchenne mouse model for testing human antisense oligonucleotides

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease generally caused by reading frame disrupting mutations in the DMD gene resulting in loss of functional dystrophin protein. The reading frame can be restored by antisense oligonucleotide (AON)-mediated exon skipping, allowing produc...

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Vydáno v:PLoS One
Hlavní autoři: Veltrop, Marcel, van Vliet, Laura, Hulsker, Margriet, Claassens, Jill, Brouwers, Conny, Breukel, Cor, van der Kaa, Jos, Linssen, Margot M., den Dunnen, Johan T., Verbeek, Sjef, Aartsma-Rus, Annemieke, van Putten, Maaike
Médium: Artigo
Jazyk:Inglês
Vydáno: Public Library of Science 2018
Témata:
Research Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5821388/
https://ncbi.nlm.nih.gov/pubmed/29466448
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0193289
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