The methyl CpG binding protein 2 gene (MECP2) produces a protein of the same name, the level of which is critical for normal brain function. Mutations leading to protein under-expression cause Rett ...
MECP2 Duplication Syndrome is a progressive neurological disorder due to a chromosomal anomaly. The chromosomal disorder was first reported in 2005 and involves duplication of the methyl CpG binding ...
The neurodevelopmental disorder MECP2 duplication syndrome (MDS) is caused by duplications spanning the methyl-CpG binding protein 2 gene (MECP2) locus, and researchers have shown that the MDS-like ...
Many cognitive neurodevelopmental disorders are a result of too many or too few copies of certain genes or chromosomes. To date, no treatment options exist for this class of disorders. MECP2 ...
Researchers have identified a potential treatment strategy for Rett syndrome via alternative splicing modulation of the MECP2 gene.
The FDA has awarded U.S. orphan drug designation and rare pediatric disease designation to Huidagene Therapeutics Co. Ltd.’s HG-204 (AAV-hfCas13Y-gMECP2), an RNA editing therapy based on CRISPR/Cas13Y ...
Brantley was born with MECP-2 Duplication Syndrome. Many Americans first learned about this progressive, neurological disorder in 2018, when NBC’s Richard Engel first announced this his son had been ...
The MECP2 Duplication Syndrome is reversible, say researchers. Importantly their study paves the way for treating duplication patients with an antisense oligonucleotide strategy. The methyl CpG ...
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