تصحيح التعبير الجيني لمورثة البروتين الليبيدي (Proteolipid Protein)‎ باستعمال تقنية تداخل الحمض النووي الريبي (RNA Interference)‎ على السلالات الخلوية للخلايا قليلة التغصنات من نموذج مخبري لمرض بيليزاوس-ميرتزباخر (Pelizaeus-Merzbacher Disease)‎

Abstract EN

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 PelizaeusMerzbacher 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.