Polytherapy with a combination of three repurposed drugs (PXT3003) down-regulates Pmp22 over-expression and improves myelination, axonal and functional parameters in models of CMT1A neuropathy

Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited sensory and motor peripheral neuropathy. It is caused by PMP22 overexpression which leads to defects of peripheral myelination, loss of long axons, and progressive impairment then disability. There is no treatment available des...

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Detaylı Bibliyografya
Yayımlandı:Orphanet J Rare Dis
Asıl Yazarlar: Chumakov, Ilya, Milet, Aude, Cholet, Nathalie, Primas, Gwenaël, Boucard, Aurélie, Pereira, Yannick, Graudens, Esther, Mandel, Jonas, Laffaire, Julien, Foucquier, Julie, Glibert, Fabrice, Bertrand, Viviane, Nave, Klaus-Armin, Sereda, Michael W, Vial, Emmanuel, Guedj, Mickaël, Hajj, Rodolphe, Nabirotchkin, Serguei, Cohen, Daniel
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: BioMed Central 2014
Konular:
Research
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC4279797/
https://ncbi.nlm.nih.gov/pubmed/25491744
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-014-0201-x
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