Rett Syndrome
Type
Collection
...and aggressive plan to use gene editing, MECP2 reactivation, RNA editing, gene replacement therapy, RNA ...mutations in the MECP2 gene. Please refer to Addgene's CRISPR Guide for a general introduction to CRISPR...diagnostic criteria have disease-causing mutations in the gene methyl-CpG binding protein 2 ( MECP2 ). MECP2 Rett...caused from loss-of-function mutations in the MECP2 gene. Causative mutations for Rett syndrome typically...global transcriptional regulator of thousands of genes and studies have suggested roles in transcriptional...activity dependent de-repression, chromatin remodeling, gene activation, and more. How loss of MECP2 protein ...suggests the major function of MECP2 is to regulate gene expression either locally or globally. The MECP2...