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AANS Neurosurgeon | Volume 27, Number 4, 2018


Researchers Probe Gene Therapy for Frontotemporal Dementia

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Gene therapy has shown promise for treating a neurodegenerative disease known as frontotemporal dementia and a related condition called neuronal ceroid lipofuscinosis, according to new findings presented by researchers from the University of Alabama at Birmingham.

Frontotemporal dementia, or FTD, is a devastating disease in which patients have rapid and dramatic changes in behavior, personality and social skills. The age of onset for FTD is relatively young, usually striking patients in their mid- to late 50s. FTD is often caused when an individual has only one copy of the GRN gene, which encodes for a protein called progranulin. Progranulin production is cut in half in individuals with only one copy of GRN.

A mutation resulting in the loss of both copies of the GRN gene leads to an almost complete absence of progranulin, causing a condition called neuronal ceroid lipofuscinosis, which often strikes adults in their 20s. NCL causes blindness and seizures, and can lead to premature death.  

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