The Applications of Gene Editing Technologies Targeting on MECP2 Gene to Treat Rett Syndrome

Rett Syndrome (RTT) is a neurodevelopmental disorder, together with side complications including respiratory disorders, seizures, etc. The incidence of RTT is related to age and sex, especially in female children. The characteristic of RTT is that after a period of normal development, children begin to show symptoms of stunted growth and gradually lose their fine hand movements and language ability. Research and evidence indicate RTT is caused by the mutation of the MECP2 gene and the loss of function of the MECP2 protein. MECP2 protein can collect some related proteins to repress the transcription in order to inhibit RTT. However, RTT cannot be cured with current treatment approaches. Gene editing technologies have been noticed and used in clinical trials. AAV9, CRISPR/Cas9, and miRARE miRNA have been used to treat RTT by targeting the MECP2 gene, correcting MECP2 mutation, and restoring the function of MECP2. These gene editing technologies have been approved to improve behavior disorders and prolong survival in vivo. This review summarizes the relationship between RTT and MECP2 and gene editing technologies used in curing RTT, aiming to stimulate future studies for clinical applications.