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Leptin antagonism improves Rett syndrome phenotype in symptomatic Mecp2-deficient mice
Biorxiv : the Preprint Server for Biology
|February 13, 2023
Summary
Elevated leptin levels contribute to Rett syndrome (RTT) pathogenesis. Blocking leptin function ameliorates RTT symptoms and neuronal deficits, suggesting leptin as a therapeutic target for this neurodevelopmental disorder.
Area of Science:
- Neuroscience
- Genetics
- Endocrinology
Background:
- Rett syndrome (RTT) is a severe neurodevelopmental disorder caused by mutations in the MECP2 gene.
- Elevated leptin levels are observed in RTT patients and models, impacting brain functions altered in the disease.
Conclusions:
- Elevated leptin levels are implicated in the pathogenesis of Rett syndrome.
- Targeting leptin pathways offers a potential novel therapeutic strategy for RTT.
- Leptin antagonism demonstrates efficacy in ameliorating core RTT symptoms and neuronal dysfunction.
