Gene therapy can help patients with anemia

US and French scientists on September 15 said that patients with rare genes of anemia may have hoped to escape the disease without needing blood transfusion thanks to gene therapy.

The study, published in Nature , is a rare success in the field of gene therapy, but scientists and experts say that more work is still needed to confirm this.

Beta thalassemia is a chromosomal recessive disease usually caused by a β-globin gene mutation located on the 11th short wing of the chromosome, causing a decrease or loss of the globin chain synthesis . Beta thalassemia is one of the most common hemoglobin in the world.

Scientists have used gene therapy to regulate responsible faulty genes for conditions of bone marrow stem cells and then transfer them back to patients.

Dr. Marina Cavazzana-Calvo of the University of Paris said that after nearly three years, patients using this therapy had become healthy without having a blood transfusion for an extended period of time.

Gene therapy is a difficult but promising method for medicine, based on the idea that faulty genes cause a number of treatable diseases. Researchers have tried to find effective ways to safely pump regulated genes back into the body.

Viruses are also often used to carry these genes into the body, but this method is highly risky because the viruses themselves are also very dangerous.

Anemia occurs when healthy red blood cells (RBCs) in the body fall too low. This can lead to health problems because RBCs contain hemoglobin, which carries oxygen to the body's tissues.

Anemia can cause various complications, including fatigue and pressure on the body's organs.

Anemia can be caused by many problems, but there are three main causes of anemia, which are excessive destruction of RBCs; bleed; and production of RBCs is not enough.

In addition, there are other causes such as genetic disorders, nutritional problems (such as iron deficiency or vitamin deficiency), infectious diseases, some forms of cancer or pharmaceutical or toxic substances.