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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Dettagli Bibliografici
Pubblicato in:Orphanet J Rare Dis
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2014
Soggetti:
Research
Accesso online: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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