America develops sickle cell therapy

A group of Stanford scientists (USA) have been making great strides in studying treatment for sickle cell disease.

A group of Stanford scientists (USA) have been making great strides in studying treatment for sickle cell disease. Expected human trials will be conducted in early 2018.

According to Engadget, sickle cell disease is a condition in which red blood cells in the blood do not have a round shape but instead have a crescent shaped shape. This will cause blood cells to be inflexible, difficult to circulate and block blood vessels leading to a lack of oxygen in the body parts. Currently there is no specific cure for this sickle cell disease.

The team used CRISPR to repair mutant genes in stem cells taken from patients. The CRISPR technique helps researchers cut out faulty gene fragments and replace them with normal DNA sequences. This will help prevent blood cells from turning into small sickle cells - which tend to clog blood vessels and cause organ damage.

Picture 1 of America develops sickle cell therapy

Sickle cell disease is a condition in which red blood cells in the blood do not have a round shape but instead have a crescent shaped shape.

In their tests, scientists injected repaired stem cells into small mice and they are still alive, thriving even after 16 weeks. This is important because sickle cell cells tend to die after only 10 days, leading to anemia throughout the body.

Despite successful testing, scientists still have to make sure that the gene therapy they are developing is completely safe. They need to find unforeseen immune responses and find ways to ensure never correct incorrect DNA fragments.

This therapy will take some time before it can be used on a large scale. Stanford is currently building the necessary infrastructure to expand this research scale.

With the constant development of medicine, dangerous diseases such as sickle cell disorder will soon become golden.

Update 15 December 2018
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