Wang H, Davison M, Wang K, et al. - A set of microRNAs (miRs) was identified that could be used as candidate biomarkers in clinical trials in Charcot-Marie-Tooth disease type 1A (CMT1A). Some of the miRs may reflect Schwann cell processes that contribute to the disease's pathogenesis.

• Following an initial pilot screen, a larger screen confirmed elevated levels of several muscle-associated miRNAs (miR1, -133a, -133b, and -206, known as myomiRs) as well as a set of miRs highly expressed in Schwann cells of peripheral nerve.

• When compared with other candidate biomarkers for CMT1A (eg, neurofilament light) measured on the same sample set, there is a comparable elevation of several miRs (eg, miR133a, -206, -223) and the ability to distinguish cases from controls.

• The highest correlation was found between neurofilament light levels and miR133a.

• Furthermore, the putative Schwann cell miRs (eg, miR223, -199a, -328, -409, -431) correlate with the recently described transmembrane protease serine 5 protein biomarker, which is most abundant in Schwann cells and also elevated in CMT1A plasma.