Plasma microRNA signature as biomarker for disease progression in frontotemporal dementia and amyotrophic lateral sclerosis

Four miRNAs are differentially expressed in plasma between C9orf72-associated clinical conditions

Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are increasingly recognised as constituting a spectrum of neurodegenerative disease. The C9orf72 intronic hexanucleotide repeat expansion is the most common genetic cause of FTD and ALS, accounting for about 1 in 10 of all cases, respectively.1 Though limited treatment options can provide a marginally decreased rate of disease progression, there is no current treatment that effectively halts neurodegeneration. However, recent advances have contributed to the development of antisense oligonucleotides targeting the C9orf72 repeat expansion with promising preliminary results.2

As we continue to elucidate the genetic basis of disease for FTD and ALS and progress towards therapeutic development, it is important to identify biomarkers to assess disease onset and progression, especially for presymptomatic or early-stage patients in whom potential disease-modifying treatments could prevent occurrence or worsening of disease. Serum-based…

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