Health.. A new genetic treatment that holds hope for Thalassemia patientsلمرضى

Thalassemia is a blood disorder that affects the body's ability to produce red blood cells.

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Medical experts say a new genetic treatment could pave the way for patients with thalassemia to transform their lives.

Dr. Rabee Hanna, a pediatric hematologist and oncologist at the prestigious American hospital "Cleveland Clinic", explained that the innovative treatment can stop or greatly reduce blood transfusions.

Hanna added that it is possible to cure thalassemia patients with genetic therapy, which is a new step that uses the patient's own blood-forming stem cells to produce healthier red blood cells and repair his blood disorder, stressing that thalassemia patients who received genetic or genetic treatment have either canceled or greatly reduced their operations. Blood transfusions needed to deal with their health condition.

Thalassemia is a blood disorder that affects the body's ability to produce red blood cells and hemoglobin, the oxygen-carrying protein in red blood cells.

Patients can develop iron overload and potentially damage the liver, heart, and endocrine system, even with blood transfusions and appropriate iron chelation therapy (removal of excess iron from the blood).

There are two types of thalassemia: “alpha” and “beta.” The patient develops one of them according to the defects that can occur in the protein chains that make up hemoglobin.

Alpha thalassemia patients tend to be silent carriers without symptoms, while beta thalassemia patients show prominent symptoms early after birth.

Moderate and severe thalassemia cases are usually diagnosed through blood tests in early childhood, and couples can have genetic testing that can predict the risk of thalassemia and related blood disorders.

Unlike blood transfusions that take a long time and last a lifetime, gene therapy can be a one-time treatment and offer a possible cure for the disease.

Although bone marrow transplantation is currently the only option available with the possibility of correcting transfusion-dependent genetic deficiency in thalassemia, this process has potential complications such as transplant failure, rejection of transplanted marrow from the host (GvHD), and opportunistic infections, particularly in Patients undergoing transplants from identical non-sibling donors.

In contrast, gene therapy comes from the patient's own cells, so there is no risk of rejection by the body.

Dr. Hanna sees the challenge as how to make this treatment available worldwide, especially in developing countries, where most patients are located.

The European Medicines Agency has granted conditional marketing authorization to a form of genetically modified product to treat beta thalassemia.

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