August 05, 2019
At present a trial that aims to use CRISPR, a gene-editing tool, to treat sickle cell is underway in the United States. Unfortunately for 100,000 people in the U.S. alone, sickle cell is their everyday reality. It is an illness caused by a genetic mutation, that results in sickle-shaped red blood cells that don’t carry oxygen effectively and get stuck in the blood vessels. Sickle cell patients often experience debilitating symptoms, and complications of the disorder can be life-shortening.
Researchers from the Sarah Cannon Research Institute will be taking cells from the bone marrow of the patients involved in the study. Once extracted, the cells will be gene-edited using CRISPR, with a view to make them produce a specific protein, fetal hemoglobin. This protein is typically only produced by fetuses and babies for a short time after birth. Fetal hemoglobin transports oxygen in unborn babies, but the genes that produce it are usually switched off shortly after birth.
The hope is that by switching this gene back on in sickle cell patients, they will be able to produce fetal hemoglobin, which will compensate for the defective hemoglobin produced in their bone marrow. If successful, this study will greatly improve the lives of those suffering through the symptoms of sickle cell. At present their only real treatment option is a bone marrow transplant but finding a match can be incredibly difficult. This new treatment would provide a treatment option to all patients, without the need for a matched donor.
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