How gene therapy for Sickle Cell Anemia Therapeutics works

 

Anemia is defined as a significant decrease in total red blood cells, RBC count, or haemoglobin. It is characterised as the blood's inability to carry an adequate amount of oxygen. Sickle-cell diseases (SCD) are blood illnesses that are frequently passed down down the generations. Sickle-cell anaemia, or SCA, is one of the most common kinds of SCDs. It essentially causes an abnormality in haemoglobin, the protein that transports oxygen throughout the body. Hemoglobin is a protein found in the body's red blood cells. Under some conditions, this results in the formation of a sickle-shaped cell.

According to the "Coherent Market Insights" Global Industry Insights, Trends, Outlook, and Opportunity Analysis of Sickle Cell Anemia Therapeutics Market.

 

Sickle Cell Anemia Therapeutics Market

How gene therapy for Sickle Cell Anemia Therapeutics works

Fetal haemoglobin is a type of haemoglobin found in all babies in the pregnancy and neonates. Because foetal haemoglobin does not sickle, even babies with sickle cell disease produce normal red blood cells. However, shortly after delivery, the body stops producing foetal haemoglobin and begins producing adult haemoglobin, which is prevented by the expression of the BCL11A gene. This is important for sickle-cell patients since the condition is caused by a gene mutation in the adult haemoglobin gene, not the foetal haemoglobin gene. In sickle cell disease, gene therapy works by driving down the expression of the BCL11A gene, which flips the switch back to foetal haemoglobin while also increasing foetal haemoglobin, which does not sickle, and reducing sickling haemoglobin.

 

Mutations in the beta-globin gene cause sickle cell disease, which results in the production of defective haemoglobin, the oxygen-carrying protein in red blood cells. Normal red blood cells are shaped like donuts, but sickle cell disease causes red blood cells to stiffen and take on a spiky, sickle-like appearance due to aberrant haemoglobin. The disease is expected to impact 100,000 persons in the United States, with Black Americans being the most affected. Sickle cell disease can be healed with a donor bone marrow transplant, however it works best for people who have a closely matched sibling donor, which is a small percentage of the population. LentiGlobin is a revolutionary gene therapy that collects blood-forming stem cells from the patient's blood. The stem cells are subsequently infected with harmless lentiviruses that transmit a modified copy of the beta-globin gene. After being reinfused into the patient, the cells settle in the bone marrow and begin producing healthy new red blood cells.