Blood Reboot

Let’s get these blood cells into shape!

This is Sandra Tsing Loh with the Loh Down on Science

Sickle cell anemia is a genetic condition that makes round red blood cells crescent- shaped. These cells can’t carry oxygen, which causes them to die early, blocking blood vessels. Patients with sickle cell disease suffer TREMENDOUS pain and even organ damage.

The BEST treatment is a bone marrow transplant. Or is it? 

Enter Dr. Julie Kanter from the University of Alabama and team! 

Thirty-five sickle cell patients donated stem cells that form red blood cells. These stem cells received a gene therapy that makes the crescent shaped cells round. Then the genetically altered stem cells were returned to the patients. 

Results? A single treatment made FORTY PERCENT of red blood cells round. Additionally, patients reported that they had no severe pain for one year after treatment! Some had reduced pain for three years!  

There are some side effects — such as abdominal pain and nausea. But the researchers think this treatment could be more successful than transplants!

These scientists are out for blood… BETTER blood, that is! 

Reference: Kanter, J., Walters, M. C., Krishnamurti, L., Mapara, M. Y., Kwiatkowski, J. L., Rifkin-Zenenberg, S., Aygun, B., Kasow, K. A., Francis J.  Pierciey, J., Bonner, M., Miller, A., Zhang, X., Lynch, J., Kim, D., Ribeil, J.-A., Asmal, M., Goyal, S., Thompson, A. A., & Tisdale, J. F. (2021). Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease. New England Journal of Medicine.