Progesterone Antagonist Therapy in a Pelizaeus-Merzbacher  Mouse Mode

ABSTRACT: Pelizaeus-Merzbacher disease (PMD) is a severe hypomyelinating disease, characterized by ataxia, intellectual disability, epilepsy and premature death. In the majority of cases, PMD is caused by duplication of PLP1 that is expressed in myelinating oligodendrocytes. Despite detailed knowledge of PLP1, there is presently no curative therapy for PMD. We used a Plp1 transgenic PMD mouse model to test the therapeutic effect of Lonaprisan, an antagonist of the nuclear progesterone receptor, in lowering Plp1 mRNA overexpression. We applied placebo-controlled Lonaprisan therapy to PMD mice for 10 weeks and performed the grid slip analysis to assess the clinical phenotype. Additionally, mRNA expression and protein accumulation as well as histological analysis of the central nervous system were perf

ORGANISM(S): Mus musculus

SUBMITTER: Sven Wichert 

PROVIDER: E-GEOD-55315 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress