Scientists recently tested a gene-editing method called adenine base editing to fix two severe mutations that cause -thalassemia, a genetic blood disease.
About Thalassemia:
It is an inherited blood disorder.
It affects the body’s ability to produce normal hemoglobin.
Hemoglobin is a protein in red blood cells (RBCs). It allows the RBCs to transport oxygen throughout your body.
In thalassemia, thebody produces fewer healthy hemoglobin proteins, and bone marrow produces fewer healthy RBCs.
The condition of having fewer RBCs is called anemia.
As RBCs serve the vital role of delivering oxygen to tissues in body, not having enough healthy RBCs can deprive your body’s cells of the oxygen they need to make energy and thrive.
Thalassemia is caused by inheriting a gene mutation (change in the normal DNA) from one or both parents.
Traits for thalassemia are more common in people from Mediterranean countries, like Greece and Turkey, and in people from Asia, Africa, and the Middle East.
There are different types of thalassemia. The type someone has depends on which gene mutation they inherit.
Symptoms: Thalassemia can cause mild or severe anemia and other complications over time (such as iron overload).
Symptoms of Anemia Include:
Trouble breathing.
Feeling cold.
Pale skin.
Severe thalassemia may cause death.
Treatments:
Blood transfusions – regular blood transfusions treat and prevent anemia; in severe cases these are needed around once a month.
Chelation therapy – treatment with medicine to remove the excess iron from the body that builds up as a result of having regular blood transfusions.
The only possible cure for thalassemia is a stem cell or bone marrow transplant, but this is not done very often because of the risks involved.
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