Scientists at Seattle Children’s Research Institute are paving the way to use gene-edited B cells — a type of white blood cell in the immune system — to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first of its kind in clinical trials at Seattle Children’s in as soon as five years.

B cells play a central role in the immune system. When the body is confronted with an infection, B cells turn into plasma cells that release protective antibodies that both fight ongoing infections and prevent future ones. Unlike other cells of the immune system that have relatively short lifespans, plasma B cells can survive and continuously produce antibodies for decades.

Because plasma cells are so efficient at making and secreting proteins like antibodies, they could be the ideal cell type to produce therapeutic proteins.

Dr. David Rawlings, Dr. Richard James and their colleagues in the research institute’s Center for Immunity and Immunotherapies are among a small number of research groups in the United States dedicated to exploring how clinicians might some day use plasma cells to fight diseases in children. In a scientific first, a basic science research team led by Rawlings and James genetically reprogrammed human B cells to act as cell factories capable of delivering sustained, high doses of a therapeutic protein introduced by gene editing.

“Theoretically, the B cells we created could provide a long-term treatment for a wide range of diseases where the body lacks the ability to make a certain protein, like in the case of the bleeding disorder, hemophilia B,” James said. “Such a cell therapy could also have broad applications for autoimmune conditions, where the introduced protein could be used to turn off abnormal immune responses, or to disarm infectious diseases by secreting known protective antibodies.”

Read more in Researchers Ready B Cells for Novel Cell Therapy in On the Pulse.