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Sickle cell disease treatment: Sickle cell disease is a strange disease. The life of patients suffering from this disease becomes like poison. A good news has come amid such circumstances. A ray of hope has arisen from this news. Here we are talking about sickle cell disease. For the first time in the world, a sickle cell patient is being treated with gene therapy. 12 year old Kendrick Cromer is being given gene therapy in America. After months of treatment, he will likely be sickle cell free.
According to a report published in the New York Times, all of Cromer's dreams were shattered due to sickle cell. It was difficult for him to ride a bicycle, go out in the cold, play football… and have fun like normal children. There was terrible pain. Kendrick Cromer is now hopeful that his condition may improve. He will recover and live a normal life.
Last year, two types of gene therapy were approved in the US to treat sickle cell disease. Kendrick is the first commercial patient from a company called Bluebird Bio. Another company, Vertex Therapeutics, has not yet disclosed whether it has started treating any patients.
About 20,000 people like Cromer in the U.S. suffer from sickle cell disease. Sickle cell disease is a genetic disorder of red cells that causes excruciating pain. People are born with this disease. They inherit the mutated gene for this disease from their parents.
How will gene therapy treat sickle cell patients?
Kendrick Cromer began treatment at Children's National Hospital in Washington on Wednesday. Doctors have removed dead cells from his bone marrow, which Bluebird will genetically modify in his laboratory to provide treatment. This will take months. Before beginning the process, Bluebird will need millions of Kendrick's stem cells. If enough stem cells are not produced the first time, the company will try one or two more times. If that's not enough, Kendrick will have to spend a month preparing for another stem cell extraction.
What is sickle cell disease?
Sickle cell disease is a genetic disease. This is a group of red blood cell disorders that affect hemoglobin. Hemoglobin is the protein that transports oxygen throughout the body. Normally, red blood cells are disc-like and pass easily through the veins. In sickle cell disease, a genetic mutation causes red blood cells to become crescent-shaped or sickle-shaped. In sickle cell disease, red blood cells cannot move easily and can block blood flow through the body. This can lead to stroke, infection and many other health problems.
Sickle cell disease is a lifelong disease. Till now bone marrow transplant was the only treatment for this disease. But in December 2023, America approved two types of therapy. In one therapy, an additional gene is added to the body and in the other, an existing gene is modified.