Scientists are developing a system to use gene-edited B cells to treat a wide range of diseases including protein deficiency disorders such as hemophilia, autoimmune diseases, and infectious diseases. Pre-clinical trials into the novel treatment system have thus far been successful, according to the Seattle Children’s Research Institute scientists whose research was presented at the 2019 American Society for Gene and Cell Therapy annual meeting.
Plasma B cells are essential for both short-term and long-term immunity—fighting ongoing infections and preventing future ones. Unlike other, relatively short-lived immune cells, plasma B cells can survive for decades, continuously producing antibodies. Taking note of these factors, some groups of scientists are exploring ways to use plasma cells to fight disease in children.
In the current study, researchers have succeeded in genetically reprogramming human B cells to act as cell factories capable of delivering continual, high doses of a therapeutic protein introduced by gene editing.
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To test the treatment, the team developed mouse models with the same molecular features necessary for the survival of long-lived antibody secreting cells in humans. This allowed them to demonstrate the ability of their reprogrammed B cells to reside in the bone marrow and produce high levels of a therapeutic proteins for a relatively long period.
The model will allow the scientists to continue to test and improve the treatment, focusing on how to best manipulate the B cells so they are suitable for clinical use.
Image: Blue, yellow and red signals show engineered human B cells located in the bone marrow of mice. Image courtesy of Seattle Children's.