Browsing by Subject "Teràpia genètica"
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Malin restoration as proof of concept for gene therapy for Lafora disease
(Brain Communications 2022, 4(4), fcac168, 2022)Lafora disease is a fatal neurodegenerative childhood dementia caused by loss-of-function mutations in either the laforin or malin gene. The hallmark of the disease is the accumulation of abnormal ...