Rett Syndrome
Type
Collection
...MECP2 disorders. The RSRT has a comprehensive, strategic and aggressive plan to use gene editing, MECP2...reactivation, RNA editing, gene replacement therapy, RNA trans-splicing, and protein replacement to reverse the...purposeful hand movements and regression of speech. In contrast to the sustained loss of skills in neurodegenerative...disease severity. The MECP2 protein is a global transcriptional regulator of thousands of genes and studies...studies have suggested roles in transcriptional repression, activity dependent de-repression, chromatin remodeling...DNA and recruit known co-repressors or other transcriptional modifiers suggests the major function of MECP2...missense mutations cluster in the MBD and NID demonstrating the importance of binding to methylated DNA ...