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Patients Suffering from Sickle Cell Are Volunteering for Landmark Gene-Editing Study

October 24, 2019 by Samuel Leave a Comment

The doctors in the USA used CRISPR first time to treat the patient having a genetic disorder. CRISPR is a reliable gene-editing technique. For this first time study, Victoria Gray got ready as a volunteer. She went through sickle cell disease treatment by using the CRISPR technique in Sarah Cannon Research Institute in Nashville. Through this way, Gray became the first patient as being involved in the study of this technique. It will take some months or a few years to confirm the positive test results, and for this period, Gray will be under observation. In her childhood only, she was diagnosed with sickle cell disease. The symptoms restricted her to play and enjoy like other children’s.

Sickle cell disease is a genetic defect, and around 100,000 individuals in the U.S. get affected by sickle cell disease. In sickle cell disease, the bone marrow produces defective protein. This protein makes sickle-shaped blood cells sticky and hard. This cell gets stuck in blood vessels, and they do not carry oxygen. This situation results in weakening and sometimes generates life-threatening complications. In this study, doctors used cells of the bone marrow of the patient. These cells are genetically modified with CRISPR to make them produce a necessary protein. This protein usually gets produced by babies and fetuses after their birth for a short period. The doctors have hope that this protein will cover up defective protein that results in sickle cell disease. Also, this will help the patient to live healthily and generally for the rest of life.

Some of the doctors and specialist says that this is a very new technology. Also, there are many unknown things about this test. When treated with a human being, there are some risks, and this test might make patient sicker. However, Gray said that she is aware of all these risk factors. She also added that she knew that this is the initial step, and other patients might see benefits after some months.

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