U.S. AGENCY APPROVES GENE-EDITING TREATMENT FOR SICKLE CELL DISEASE.

Sickle cell disease has long been identified across the world as a deadly disease which lasts for a lifetime, exposing patients to numerous diseases and infections. This disease has claimed the lives of both children and adults alike, making living exceptionally difficult for its carriers.

What Is Sickle Cell Disease?

Sickle cell disease is one of the most common blood hereditary diseases, which causes the red blood cells to develop a sickle shape because of this shape and other circumstances, the flow of blood through the body is blocked thereby leading to exposure to infections, diseases and pain crises.

What Causes Sickle Cell?

This is caused due to the change that occurs in red blood cells which originally have a disc shape and flexibility enough to move through the blood vessel, to become crescent or sickle-shaped thereby resulting in difficulty or total blockage of the red blood cell movement through the blood vessel. This blockage tends to lead to health deterioration like fatigue, stroke, death of an organ, infections etc.

Furthermore, this disease spread across the world and has led to many deaths and unpleasant living, with treatments that can help a carrier manage the complications and live longer.

The bone marrow transplant became the only cure for some carriers at one point. However the process included in a bone marrow transplant drastically reduced its chances, getting a matching bone marrow became a hassle to this cure and sometimes carriers who have undergone bone marrow transplants will face bleeding or worse, death.

This is because the process of transplant in children requires time for the new cells to grow in the bone marrow which takes up to 3 weeks. At this time, the child is at risk of getting infections and bleeding and if these are severe can ultimately lead to death.

Therefore, medical personnel and establishments worked hard to create another cure for Sickle Cell Disease, thus the therapy called “Casgevy” from Vertex Pharmaceuticals and CRISPR therapeutics that uses gene-editing tool CRISPR became the first medicine to be approved in the U.S.A. This cure won its founders the Nobel Prize in Chemistry 2020.

The sickle cell cure called Casgevy, using CRISPR edits the DNA in a patient’s stem cell to remove the gene that causes the disease. It completely removes the need for a donor.
This treatment is suitable for children from 12 and above.

Also, On Friday, 8th December 2023, the United States Food and Drug Administration (FDA) authorised a second treatment of the sickle cell disease called “Lyfgenia” invented by drugmaker Bluebird Bio. It is a gene therapy and both treatments genetically modify a patient’s stem cells.

Finally, the sickle cell disease seems to have gotten to a season of eradication, although the sad news about this new therapy is that it costs lots of million dollars and this might restrict poor people who have this disease from getting this treatment and also, it doesn’t prevent the gene from passing down to future generations.

Like Our Story ? Donate to Support Us, Click Here

You want to share a story with us? Do you want to advertise with us? Do you need publicity/live coverage for product, service, or event? Contact us on WhatsApp +16477721660 or email Adebaconnector@gmail.com