Learn How Gene Knock-out Cures Sickle Cell Disease

Did you know that inactivating a solo cell helps in the treatment of sickle cell disease? You might be wondering how! Well, it is done by keeping the blood young everlastingly. As far as the ailment is concerned, it’s caused due to a mutant type of adult hemoglobin, and not because of fetal hemoglobin. The mutant hemoglobin type has elongated sticky chains inside the red blood cells (RBCs). The cells within these chains may lead to the clogging of small blood vessels, depriving the human organs of life-giving oxygen, resulting in pain. In some cases, sickle cell disease may prove fatal.

According to an article published on https://www.huffingtonpost.com, sickle cell is a genetic disorder, a severe disease, affecting people who inherit the sickle cell gene from their parents. A chronic sickness does not let go. Read on to learn more.

Making fetal hemoglobin

When the human body is tricked into producing fetal hemoglobin, the symptoms of sickle cell disease can be alleviated. Do you know why? Well, that is because fetal hemoglobin does not produce sticky chains. In fact, it is made in the body when the fetus develops in a mother’s womb and then six months after the birth of the baby. However, it has more likeness for oxygen compared to adult hemoglobin, essential in letting the growing fetus to take oxygen from the mother’s blood.

RBCs lack the normal look

When one has the sickle disease, the RBCs does not look normal and are not round in shape. If a person is diagnosed with the ailment, the doctor will say so. The cells appear pointed and long, resembling a sharp-edged knife or sickle. When these sharp borders are fixed in place into the blood vessels, they obstruct blood circulation, thus resulting in excruciating pain and discomfort.

Gene therapy

The different methods of genetic knockout or gene therapy block the effects of the BCL11A gene in human beings. Theoretically, the method has many benefits. The specially made RNA lengths, when entered into the bloodstream through injection, may tie with the BCL11A gene, thus inactivating it. Though the process is effective, it is unfeasible and costly on a large scale. You should look for a long-term goal to use some drug to block the BCL11A function. It is a more challenging process, but it can be applied to a larger population.

Other drugs

There are other treatments to spur the production of fetal hemoglobin. The suggestions have been coming from scientists and medical experts to battle sickle disease. One specific medication is hydroxyurea that is largely used by patients plagued with the ailment but has several side effects. It leads to the reduction of the white blood cells (WBCs). The effect of hydroxyurea on the human body is not well understood. How drugs work is a big question even now. In a few patients, the medication works, but in other it proves ineffective. The results are erratic. The gene is silenced resulting in the restraint of fetal hemoglobin.


If you know anyone plagued with sickle disease, consult your doctor right away. Stay happy and healthy.

Author bio –

Kristen Smith is a Professional writer. She has written many articles on Health. You can visit genetic knockout.

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