The Cure of Sickle Cell Disease Is Close: CRISPR Technology

Sickle cell is one incurable disease that affects Africans. The disease has destroyed lives, altered destines, stopped beautiful love stories, and given pain to families. How does sickle cell happen? The red blood cells take an irregular shape or a crescent structure because of low amounts of oxygen. This stops the blood cells from moving through the blood vessels, causing them to clump, and cause problems like stroke, organ damage, and pain.

In 2020, two researchers got the Nobel Prize in Chemistry for inventing the CRISPR-Cas9 technology. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. This technology edits the sufferers’ genes to help them recover from the disease. Clinical trials carried out saw two patients recover from sickle cell. How can this happen? The CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia does the magic.

It edited the genes by removing the gene BCL11A, which is responsible for suppressing fetal hemoglobin production. When this happens, the stem cells can begin to produce fetal hemoglobin in patients with beta thalassemia or sickle cell disease.

The fetal hemoglobin overcomes the effect of the defective hemoglobin that causes sickle cell. What this means is that the patient does not require a donor. The gene-editing has nothing to do with a viral vector-like in electroporation, which has a low risk of off-target gene activation. Electroporation is the fast production of pores into the cells with high voltage.

While Africans await the full use of this technology, we have seen more researchers achieve successful clinical trials on the disease. Today, we have a better way of monitoring the blood cells without the need for biochemical markers or microscopic imaging. This ensures that the disease can be managed successfully.

While the monitoring of sickle cell disease happens when a blood sample is watched under a microscope, it comes with many disadvantages. The process can delay the capture of vital changes and it is time-consuming. Often, this delay can cause permanent cell damage to the sufferer.

However, new monitoring technology has offered new hope for the sufferers, by the use of a microfluidics-based electrical impedance sensor. This sensor makes it easier for tests to be carried out faster and better.

The technology measures the oxygen levels in real-time and mimics what is happening in the sufferer’s blood. Currently, the only cure for this deadly disease is having a bone marrow transplant. The surgery allows the sufferer to receive bone marrow from a healthy, genetically-compatible donor. The problem is that for many adults, these transplants are risky. For the kids, only a few of their relatives can match their blood as donors.

Gene therapies have become the future of treating these patients. We hope in the nearby-future, many Africans can have access to these new treatments as these technologies continue to improve and become widespread. In the mean time, for those yet to have kids, it is important to choose a partner who is compatible with your genotype to avoid issues showing up in the future with your kids.