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Loss of spastin function results in disease-specific axonal defects in human pluripotent stem cell-based models of hereditary spastic paraplegia

Human neuronal models of hereditary spastic paraplegias (HSP) that recapitulate disease-specific axonal pathology hold the key to understanding why certain axons degenerate in patients and to developing therapies. SPG4, the most common form of HSP, is caused by autosomal dominant mutations in the SP...

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Библиографические подробности
Главные авторы: Denton, Kyle R., Lei, Ling, Grenier, Jeremy, Rodionov, Vladimir, Blackstone, Craig, Li, Xue-Jun
Формат: Artigo
Язык:Inglês
Опубликовано: 2014
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3947148/
https://ncbi.nlm.nih.gov/pubmed/24123785
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/stem.1569
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