To examine the expression profiles of vault RNAs (vtRNA), a newly identified group of noncoding RNAs, in the cerebrospinal fluid (CSF) and blood of multiple sclerosis (MS) patients in order to assess their value as molecular markers of multiple sclerosis. 

Noncoding RNAs (ncRNA) have been intensely studied in several diseases for their potential applications as diagnostic and prognostic biomarkers and possibly for their therapeutic value.   The noncoding vtRNAs are part of the vault complex and their molecular functions are still obscure. Recent studies reported their involvement with multi-drug resistances and autophagy modulation. MS is an inflammatory neurodegenerative disease, with relapsing remitting (RRMS) best representing the inflammatory component of the disease and  primary progressive MS (PPMS) typifying degenerative aspects of the disease .   

A differential strand-specific RNA sequencing analysis of ncRNAs was completed of 12 CSF samples (n=4 PPMS, n=4 RRMS, and n=4 healthy donors). The ncRNAs with significantly different expression levels, in particular vtRNA 1-1 and vtRNA 2-1, were validated by quantitative PCR (qPCR) in 32 blood samples (n=13 PPMS, n=11 RRMS, and n=8 healthy donors).

We observed a marked difference of the vtRNAs expression levels in blood samples of PPMS and RRMS patients compared to healthy donors.  These results if validated with a larger cohort of samples may  establish vtRNAs as diagnostic biomarkers for MS and in pathophysiology of disease.