Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxonin

Giant axonal neuropathy (GAN) is a progressive neurodegenerative disease caused by autosomal recessive mutations in the GAN gene resulting in a loss of a ubiquitously expressed protein, gigaxonin. Gene replacement therapy is a promising strategy for treatment of the disease; however, the effectivene...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Johnson-Kerner, Bethany L., Ahmad, Faizzan S., Diaz, Alejandro Garcia, Greene, John Palmer, Gray, Steven J., Samulski, Richard Jude, Chung, Wendy K., Van Coster, Rudy, Maertens, Paul, Noggle, Scott A., Henderson, Christopher E., Wichterle, Hynek
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2015
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4402342/
https://ncbi.nlm.nih.gov/pubmed/25398950
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu556
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