Cells from patients with sickle cell disease have been used to generate mature red blood cells without the genetic mutation that causes the disease, investigators at Johns Hopkins University reported.
This advance may lead to improved treatment of sickle cell disease patients, who frequently need blood transfusions from healthy donors but often reject foreign blood.
The corrected cells were created through genome editing of human induced pluripotent stem cells (iPSCs), which can make any cell in the body and grow indefinitely in the laboratory.
“Our results represent a significant step towards the clinical applications of genome editing using patient-derived iPSCs to generate disease-free cells for cell and gene therapy,” according to the study’s lead researcher, Xiaosong Huang, and his associates.
The edited iPSCs, which were derived from the patients’ blood cells, produced blood cells as “efficiently” as stem cells that had not been edited, according to a written statement from Johns Hopkins. Still, “to become medically useful, the technique … will have to be made even more efficient and scaled up significantly,” study investigator Linzhao Cheng, Ph.D., noted in the statement.
Find the full study in Stem Cells (2015 Feb. 20 [ doi:10.1002/stem.1969]).