Amongst genetic disorders hemoglobinopathies are the ones that affect the structure or production of the haemoglobin molecule which is primarily the red protein responsible for transporting oxygen in the blood.
Most commonly occuring hemoglobiopathies are thalassemia and sickle cell disease. In the United States of America alone more than one lakh children are born with sickle cell disease and worldover more than three lakh thirty thousand are born with either sickle cell disease or thalassemia. An experimental gene therapy has been brought in by a latest research which proposes that with treatment, those people who are suffering from these conditions have the potential ability to make functional haemoglobin molecules also reduce the presence of sickled blood cells or ineffective blood cells in thalassemia and prevent associated complications. This is one of the most talked about medical innovations of the year 2021 which is going to be a boon for the sufferers of hemoglobinopathies.
SOURCE: PUBMED
