MicroRNA Profile, Putative Diagnostic Biomarkers and RNA-Based Therapies in the Inherited Lipid Storage Disease Niemann-Pick Type C

Lipids are essential for cellular function and tightly controlled at the transcriptional post-transcriptional levels. Dysregulation of these pathways is associated with vascular diseases, diabetes, cancer, several inherited metabolic disorders. MicroRNAs (miRNAs), in particular, a family gene repressors regulation many genes that encode proteins involved multiple lipid metabolism pathways, thereby influencing their homeostasis. Thus, this class non-coding RNAs (ncRNAs) has emerged as promising therapeutic target treatment lipid-related alterations. Most miRNAs act an intracellular level, but past few years, role intercellular signaling molecules also been uncovered since they can be transported bodily fluids used potential biomarkers In review, we point out current knowledge on miRNA signature lysosomal storage disorder dysfunction, Niemann-Pick type C, discuss use targets RNA-based therapies.