Mitochondrial modulation with leriglitazone as a potential treatment for Rett syndrome.

ABSTRACT:

Background

Rett syndrome is a neuropediatric disease occurring due to mutations in MECP2 and characterized by a regression in the neuronal development following a normal postnatal growth, which results in the loss of acquired capabilities such as speech or purposeful usage of hands. While altered neurotransmission and brain development are the center of its pathophysiology, alterations in mitochondrial performance have been previously outlined, shaping it as an attractive target for the disease treatment.

Methods

We have thoroughly described mitochondrial performance in two Rett models, patients' primary fibroblasts and female Mecp2^tm1.1Bird-/+ mice brain, discriminating between different brain areas. The characterization was made according to their bioenergetics

SUBMITTER: Musokhranova U 

PROVIDER: S-EPMC10601217 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature