Gene Therapy towards a Cure for Sickle Cell Anemia

   So much work has been done in Sickle Cell Disease research over the last decade. Most recently, doctors in the U.S. are utilizing an innovative gene-editing technique called CRISPR to cure patients with Sickle Cell Disease. In Sickle Cell Disease (SCD), a single-genetic blood disorder causes the blood cells to form an abnormal, sickle shape. Researchers are using cells taken from a patients’ own bone marrow that have been genetically modified with the CRISPR technique. This editing technique helps individuals to permanently produce a protein called Fetal Hemoglobin (a hemoglobin that does not make the blood sickle). Fetal Hemoglobin, is typically only made by fetuses and babies for a short period following birth. Ideally, this protein will compensate for the defective sickle gene protein that causes SCD, therefore helping patients to live a normal life. This treatment is very new so there are a lot of unknown risks. The clinical trials (studies) that are being conducted are just a first step, with a lot more work to be done in the future. It will likely take years before determining whether Gene Therapy is a universal cure and if the benefits last a lifetime. But CRISPR has enabled scientists to make very precise changes in the DNA of the sickle gene, which may lead to new ways to prevent as well as treat other diseases. Recently, CRISPR Therapeutics has revealed for the first time that the treatment might be promising for Beta Thalassemia, a genetic blood disorder. Beta Thalassemia patients may require regular transfusions to treat their condition. A patient enrolled in a research study has not required a blood transfusion in more than four months! While the research surrounding CRISPR is just beginning, it is still very exciting for persons living with Sickle Cell Disease as well as other chronic genetic disorders. We will certainly keep an eye on these studies as they progress. For more information, go to www.nhlbi.nih.gov.

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