At the recent annual meeting of the American Society of Hematology, researchers reported that very early clinical trials of a gene therapy for sickle cell anemia (SCA) were successful. In two patients, scientists used a modified virus to insert normal fetal hemoglobin genes into the blood stem cells of SCA patients. The 35- and 25-year-old patients’ symptoms were reversed.
"One year after treatment of our first patient, and six months after treatment of our second patient, both have seen a remarkable improvement in the quality of life due to remarkable reduction in disease symptoms," said Punam Malik, professor in the department of pediatrics at University of Cincinnati and the chief investigator of the study. "This includes near elimination of chronic pain and sickling events and improved anemia,” she added.
Source: Cincinnati Children's