Evaluation of gene therapy in thalassemia

Thalassemia is a heterogeneous group of inherited anemias and is aquantitative disorder caused by a mutation that affects the production ofhemoglobin, which is divided into three categories: ۱. major (severe form)۲. middle ۳. minor. Homozygous (major) thalassemia occurs if the geneticdisorder is inherited from both parents, and heterozygous (minor)thalassemia occurs if the genetic disorder is inherited from only one parent.Gene therapy is the release of genetic components into cells to regulate cellfunction and activity and for the cell to respond to externally expressedproteins. Gene therapy is a promising way to treat many diseases includingcancer, vascular disorders, genetic and inherited diseases. It is done in twoways: somatic and germline.Relevant information was searched from databases including the PubMed,Scopus, Scholar google, mag Iran, and the data were analyzed qualitatively.According to the findings, genetics and heredity are very important inthalassemia, and therefore it is one of the best treatments for gene therapy,which is done in both somatic and germline. In the somatic method, allchanges are limited to the patient, but in the germline, changes are alsopassed on into the next generation. Gene therapy is the best way to treatthalassemia, which is the most common and ethical method among somaticmethods.

کلیدواژه ها:

Gene therapy ، germline ، thalassemia