Gene therapy is a treatment method that involves the alteration, correction or replacement of diseased genes in order to treat genetic diseases or alleviate their symptoms. Gene therapy in children stands out as a promising approach, especially in the treatment of inherited genetic diseases. This therapy aims to correct the source of the disease by targeting the underlying genetic causes of the disease. Gene therapy is usually applied with three main methods:n**Increasing Gene Expression:**, **Gene Regulation:**, **Gene Silencing:** nGene therapy is used especially in the treatment of the following diseases in children:nInherited Genetic Diseases: Diseases such as cystic fibrosis, Duchenne muscular dystrophy, haemophilia.nMetabolic Disorders: Metabolic diseases such as phenylketonuria.nNeurological Diseases: Motor neurone diseases such as SMA (spinal muscular atrophy).nGene therapy in children has great potential in the treatment of genetic diseases. With the advancement of technology, safer and more effective treatment methods are expected to be developed. However, overcoming the ethical and financial challenges in this field is important for gene therapy applications to reach a wider audience.