NCIt definition : A preparation of autologous T-lymphocytes that have been engineered to express a chimeric
autoantibody receptor (CAAR) containing the autoantigen muscle, skeletal receptor
tyrosine-protein kinase (MuSK; muscle-specific kinase) that is fused to the co-stimulatory
domain of 4-1BB (CD137) and the T-cell receptor signaling domain of CD3zeta (CD3z),
with potential immunomodulatory activity. Upon administration, the autologous MuSK-CD3z/4-1BB-expressing
CAAR T-cells MuSK-CAART specifically recognize and induce selective toxicity in aberrant
B-cells expressing MuSK autoantibodies. This inhibits the production of MuSK autoantibodies.
MuSK is a transmembrane protein expressed on skeletal muscle cell membrane that plays
an important role in the formation and maintenance of the neuromuscular junction.
MuSK autoantibodies are produced and expressed by aberrant B-cells in MuSK-associated
myasthenia gravis.;