遺伝子治療―基本から最新動向まで

抄録

The term “gene therapy” refers to the following activities to treat or prevent disease. Gene therapy is defined as the administration of a gene or transgenic cells into a human body (gene addition), the modification of a human gene by targeting a specific nucleotide sequence (gene correction), or the administration of genetically modified cells into a human body (gene modifications, ex vivo). Although gene therapy for human beings started around the 1980s, mainly in the United States, its practical application has not been easy due to many problems such as safety and efficiency of transfection. However, a gene therapy drug for spinal muscular atrophy, an intractable neurological disease, has finally succeeded and is now available in Japan. This review will introduce the basic concept of gene therapy and recently approved gene therapy drugs focusing on nervous system diseases.