Reengineering Chimeric Antigen Receptor T Cells for Targeted Therapy of Autoimmune Disease

Ellebrecht CT, Bhoj VG, Nace A, Choi EJ, Mao X, Cho MJ, Di Zenzo G, Lanzavecchia A, Seykora JT, Cotsarelis G, Milone MC*, Payne AS*. Science. Jun 30 2016 Jul 1;353(6294):14. | doi: 10.1126/science.aaf6756 [Epub ahead of print]


In a Science study with potentially major implications for the future treatment of autoimmunity and related conditions, co-senior author Mike Milone, MD, PhD, and colleagues have found a way to remove the subset of antibody-making cells that cause an autoimmune disease, without harming the rest of the immune system. The autoimmune disease the team studied is called pemphigus vulgaris (PV), a condition in which a patient's own immune cells attack a protein called desmoglein-3 (Dsg3) that normally adheres skin cells. The paper, published as a Science online First Release, demonstrates their new technique by successfully treating this otherwise fatal autoimmune disease in a mouse model, without apparent off-target effects, thus showing that engineered T cells can selectively target the antibody-producing cells that cause autoimmune disease. Blood Bank clinical fellow Dr. Vijay Bhoj is second author of the paper.
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