Gene therapy fort thalassemia
Thalassemia is a typical monogenic genetic disease caused by mutation or deletion of the pearl protein gene, which mainly includes α- and β- thalassemia. The rate of carrying the gene of thalassemia accounts for about 1.5% of the world population (80-90 million), and about 68,000 children with thalassemia are born every year, which is one of the genetic diseases with the greatest impact and incidence in China. Currently there is no complete cure for hemoglobinopathies, and although some patients use bone marrow transplantation, it is expensive and extremely difficult to match. In recent years, the advent of gene therapy has brought hope to patients, with the greatest advantages of not requiring a donor, not requiring an allogeneic transplant, and the possibility of a single treatment leading to a permanent "cure", promising to replace the current imperfect treatment options. Gene Therapy is the introduction of exogenous normal genes into target cells to correct or compensate for diseases caused by defective and abnormal genes for therapeutic purposes. With its unique potential to cure genetic diseases once administered for life, gene therapy makes the impossible possible. It has shown great therapeutic potential in a variety of diseases such as cancer, genetic disorders such as thalassemia, sickle cell anemia, hemophilia and congenital melanosis.
