AI Article Synopsis
- Rett Syndrome is a serious neurological disorder primarily caused by mutations in the Mecp2 gene, which is important for chromatin structure and gene regulation.
- Recent research has focused on the molecular functions of Mecp2 and the development of new animal and cell models to enhance the understanding of Rett syndrome.
- The review highlights innovative treatment strategies that range from traditional medications for symptom management to cutting-edge therapies aimed at potentially curing the disorder.
Article Abstract
Rett Syndrome is a severe neurological disorder mainly due to mutations in the methyl-CpG-binding protein 2 gene ( ). Mecp2 is known to play a role in chromatin organization and transcriptional regulation. In this review, we report the latest advances on the molecular function of Mecp2 and the new animal and cellular models developed to better study Rett syndrome. Finally, we present the latest innovative therapeutic approaches, ranging from classical pharmacology to correct symptoms to more innovative approaches intended to cure the pathology.
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Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5871944 | PMC |
| http://dx.doi.org/10.12688/f1000research.14056.1 | DOI Listing |
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