Autologous MuSK-CD3z/4-1BB-expressing Chimeric Autoantibody Receptor T-cells MuSK-CAART

**Semantic type:** Cell|Pharmacologic Substance

**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.

**Synonyms:** - Autologous Anti-MuSK-CD3z/4-1BB-expressing CAART MuSK-CAART - Autologous MuSK CAAR T-cells MuSK-CAART - Autologous MuSK CAAR-expressing T-cells MuSK-CAART - Autologous MuSK-CD3z/4-1BB-expressing CAAR T-cells MuSK-CAART - Autologous MuSK-CD3z/4-1BB-expressing CAART MuSK-CAART - MuSK-CAART