Title:B-Cell Based Gene Therapy for Autoimmune Diseases
Volume: 12
Issue: 3
Author(s): David W. Scott, Ai-Hong Zhang and Yan Su
Affiliation:
Keywords:
Tolerance, B cells, gene therapy, immune system, self tolerance, autoimmune disease, B-cell gene therapy, environmental triggers, patient’s tissue, antigen receptors, immature lymphocytes, systemic lupus erythematosis, cancer, immune therapies, hemophilia
Abstract: The essence of the adaptive immune system is self tolerance, which is maintained by various central and peripheral
check points. However, the tolerance mechanisms can be broken in autoimmune disease conditions due to genetic
predisposition and environmental triggers. As a consequence, a patient’s tissue is attacked by his/her own adaptive immune
system. An ideal therapy for autoimmune diseases should include methods to re-establish tolerance to the underpinning
autoantigen(s). During the last 15 years our lab has been dedicated to developing a novel B-cell gene therapy approach
for antigen-specific tolerance induction. This approach has been successfully applied to at least five different animal
models for human autoimmune diseases. In this article, we will discuss many of our successful preclinical studies using
the B-cell gene therapy approach to induce tolerance, as well as similar studies from others. Particular focus will be
given to the tolerance induction mechanisms that have been revealed from these studies.
