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First Country to approve gene therapy for Sickle Cell Anemia

Research Article about the "First Country to approve gene therapy for Sickle Cell Anemia"


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First Country to approve gene therapy for Sickle Cell Anemia.

12 Year-old boy from the USA has recently become the first person with sickle cell disease in the world to start a commercially approved gene therapy that could potentially cure the illness.

Sickle Cell Disease (SCD):

It is a genetic blood condition. It is characterized by defective hemoglobin.

Hemoglobin is the molecule found in red blood cells (RBCs) that transports oxygen to the body's tissues.

Individuals with sickle cell disease possess abnormal hemoglobin molecules known as hemoglobin S, which can cause red blood cells to take on a sickle shape. SCD disrupts the supply of oxygen to the body's tissues.

What is the impact on blood circulation?

Typically, red blood cells have a round shape and are flexible to navigate the blood vessels smoothly.

In sickle cell disease (SCD), RBCs alter their shape to become crescent- or "sickle"-shaped because of a genetic mutation.

These crescent-shaped red blood cells are not flexible and can obstruct the circulation of blood to other parts of the body.

What is the reason behind it?

SCD is caused by a faulty gene known as the sickle cell gene.SCD will only develop in a person if they inherit two genes, one from each parent.

Signs of illness:

At the beginning: Extreme fatigue or irritability caused by anemia, painfully enlarged hands and feet, and jaundice.

Subsequent phase: Intense discomfort, low red blood cell count, harm to organs, and infections.

Medical care:

A stem cell transplant (bone marrow transplant) has the potential to treat sickle cell disease successfully.Nonetheless, there are therapies available that can alleviate symptoms, reduce complications, and extend lifespan.

Exploration is also underway for gene therapy as a potential cure.

The UK was the first country to authorize gene therapy treatment for sickle cell disease (SCD) recently.

Best Regards,

N. Sri Manoj kumar, 

Biomedical Trainer, 

Atheenapandian Private limited. 

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