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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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| Publicado no: | Brain |
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| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Oxford University Press
2017
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| Assuntos: | |
| Acesso em linha: | 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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