Regulation of proteolipid protein (PLP) expression in oligodendrocytes cell lines of animal models for Pelizaeus-Merzbacher Disease (PMD) using RNA interference (RNAi) technology

Abstract

In this study, RNA interference technology was used to inhibit gene expression of the Proteolipid Protein (PLP) on Oligodendrocytes cell lines from Jimpy mutant mice, an animal model for Pelizaeus-Merzbacher Disease (PMD), which is a X-linked sever leukodystrophy. The preliminary findings demonstrate a significant correction of functional aspects in oligodendrocytes, ie effect of dibutyryladenosine 3 ,5 - cyclic monophosphate (dbcAMP) application on both Myelin Basic Protein and Carbonic Anhydrase II expression. Our results suggest a breakthrough in leukodystrophy therapy especially if coupled with effective delivery system in vivo.