Current situation and future of gene therapy for rare diseases

Abstract

Rare genetic disorders are rare diseases that have a very low incidence, most of the patients are children, and most of them are caused by genetic defects. Many of these diseases are serious chronic genetic diseases with a small number of patients, low market demand, and high cost of drug research and development, so there is a lack of effective treatment methods, which often threaten life. Gene therapy is an emerging therapeutic approach that uses vectors to introduce genetic material into target cells to treat or prevent rare diseases by correcting or supplementing defective genes. This paper reviews the clinical application of gene therapy for rare diseases such as blood diseases, neurodegenerative diseases and eye diseases. At present, gene therapy still has some technical problems and rare diseases are complicated and cannot be effectively treated. In the future, with further research and overcoming these problems, the application of gene therapy in rare diseases will make continuous breakthroughs and bring good news to mankind.