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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 …
Contributors RD and BJT wrote the manuscript.
Funding This research was supported by the Intramural Research Program of the National Institutes of Health (National Institute on Aging, project number: 1ZIAAG000935 (PI Bryan J Traynor)).
Competing interests BJT holds US, EU and Canadian patents on the clinical testing and therapeutic intervention for the hexanucleotide repeat expansion of C9orf72. BJT is an editorial board member for JNNP and Neurobiology of Aging and is an associate editor for Brain.
Provenance and peer review Commissioned; internally peer reviewed.
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