Gene therapy targeting oligodendrocytes provides therapeutic benefit in a leukodystrophy model

Pelizaeus-Merzbacher-like disease or hypomyelinating leukodystrophy-2 is an autosomal recessively inherited leukodystrophy with childhood onset resulting from mutations in the gene encoding the gap junction protein connexin 47 (Cx47, encoded by GJC2). Cx47 is expressed specifically in oligodendrocyt...

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Vydáno v:Brain
Hlavní autoři: Georgiou, Elena, Sidiropoulou, Kyriaki, Richter, Jan, Papaneophytou, Christos, Sargiannidou, Irene, Kagiava, Alexia, von Jonquieres, Georg, Christodoulou, Christina, Klugmann, Matthias, Kleopa, Kleopas A.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2017
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Original Articles
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5837386/
https://ncbi.nlm.nih.gov/pubmed/28100454
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/brain/aww351
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