Victoria Gray opens the closet door and pulled out a suitcase. She began to pack piles of clothes. Ms. Gray said, “My goodness, Did I really bring this all?” Gray has sickle cell disease and is a first patient with a genetic disorder. Doctors in the US have tried to treat her using CRISPR, the powerful gene-editing technique. Today, Gray is finally getting ready to go home to Forest, Mississippi. She has been away from her four children for months in order to undergo experimental treatment. The treatment involves genetically-modified bone marrow cells’ infusions. According to Dr. Haydar Frangoul, Ms. Gray is a pioneer, and they are very excited about it. Victoria says that she feels hopeful for the future.
The disease is an overwhelming genetic disease that affects millions of people across the globe, including around 100,000 in the US. Sickle cell disease is caused by genetic defects. It turns red blood cells into sticky, hard, and sickle-shaped cells that don’t carry oxygen well. It also blocks the bloodstream, causes tortuous pain, and damage organs. Like other sickle cell patients, Gray had to drop her school, quit her job, and stay in hospitals far from home for weeks. Since many patients are unable to survive in their 40s, Victoria is worried about whether she will live to see her grown-up children as she has turned 34. She now has hope as the doctors have infused her billions of bone marrow cells back after editing with CRISPR in July.
Scientists use this technique to modify a gene in cells to produce fetal hemoglobin, a protein that usually stops shortly after birth. It is hoped that the protein produced by gene editing will make sickle cell patients healthy red blood cells. Sickle cell research is part of a series of studies that have moved CRISPR from the laboratory to the clinic. Earlier this year, two companies sponsoring sickle cell research announced that they had used CRISPR in Germany to treat the first patient with a blood disorder called beta-thalassemia. One of the companies in July revealed that the method might be effective for beta-thalassemia at least. The company reported that the patient-edited cells began to function in the bone marrow.