DRUGS FOR TREATING RETT SYNDROME

摘要

A drug for treating Rett syndrome which comprises an expression vector containing MeCP2 gene as the active ingredient. The term "expression vector" as used herein means either a viral vector or a nonviral vector. A drug for treating Rett syndrome which comprises, as the active ingredient, a substance capable of repairing a mutation site in MeCP2 gene on a chromosome having mutated. A drug for treating Rett syndrome which comprises, as the active ingredient, a substance capable of artificially activating and expressing normal MeCP2 gene serving as an allele on the other X chromosome being nonfunctional due to the chromosome inactivation (X inactivation) mechanism. In these drugs, the gene expression and repair are carried out in the striatum (putamen) in the cranial nerve.