Friday, February 1, 2019
Sickle-cell Disease Cure May Be on the Horizon
An article from The New York Times discusses that success may have been made in "the first genetic cure of a common genetic disease". Sickle-cell disease affects the blood. It causes red blood cells to have a sickle or crescent shape as opposed to normal cells with a more smooth and circular shape. (NIH) The sickle shape of the cell causes issues with the ability of the cell to move through the circulatory system. It typically affects individuals of African descent. There is one specific gene that causes sickle-cell disease. It was believed that the disease could be cured if they could fix the problem on a genetic level. Stem cells are taken from a patient's bone marrow and then they are genetically modified and put back in to the individuals bloodstream, with hopes that they will form new and healthy blood cells.
I know that sickle-cell disease can be very painful and dangerous. The young man in the article that received the gene therapy treatment had suffered from four strokes by the time he was 18. Many people also die from complications of this disease. The fact that we are capable of making changes to someone on a genetic level is incredible. This could be a huge stepping stone in treatment for many other genetic disorders.