In World’s First, Gene Editing Cures Teen With Sickle Cell Disease

Breakthrough
In World’s First, Gene Editing Cures Teen With Sickle Cell Disease PHOTOGRAPH: Pixabay | Under Public Domain

A new gene editing therapy was successfully used to reverse a sickle cell disease on a teenager. The scientists used the technique on one French teenager which showed positive results. The breakthrough could effectively lead to a treatment for millions of people crippled with the disease.

The unnamed teenager with the disease had a complete remission after gene therapy at the Necker Children’s Hospital in Paris, France. The therapy took 15 months. Most noteworthy, the patient is already off with the medication. Currently, researchers found it too early to say that the therapy fully cures the teen. Still, the case showed that the scientists are on the right path to pioneer the right kind of treatment for the disease. The patient from France started the treatment at the age of 13. The teen’s blood cells now had no further sign of the disease which is a great development for the researchers.

The disease affects a person when one of the proteins that made a type of hemoglobin used to carry oxygen, takes a different form. The hemoglobin which rested on the red blood cells will affect it. The red blood cells will lose elasticity which will deform them into a curved ‘sickle’ shape, hence, given the name of the disease. There will be clumps of cells piling up before it could pass through the blood vessels.

The therapy used a virus that inserted genes to correct the form of protein. This procedure helped the French teenager recover from the crippling disease. The gene editing procedure allowed the virus to be injected to the bone marrow, restoring the elasticity of the patient’s red blood cells.

Gene editing brought a new breakthrough on the crippling sickle cell disease

Dr. Philippe Leboulch, one of the team members involved in the experiment, stated that the patient has no sign of the disease and does not need hospitalization. Additionally, the patient is not experiencing any pain. The patient no longer needs a transfusion which pleased the researchers. However, doctors have to perform the same therapy to many patients before they can be confident to propose the therapy into the mainstream medical field.

People with the sickle cell disease inherited blood disorders. Patients with the disease have abnormal hemoglobin in their red blood cells. The abnormality causes the blood to clog the tiny vessels and organs of the body.

There are around 275,000 infants around the world that are born with the sickle cell disease every year. In the United States alone, there are an approximately 100,000 people who have it. Most of the patients have African ancestry or those who have African-American descent. Basically, the ratio for the disease is about 1 in every 365 African-American children in the U.S. is born with the disease. With the recent success of the therapy, the medical field made a breakthrough in the search for curing the sickle disease.

 

Source: The New England Journal of Medicine

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