As anticipated, the remarkable outcomes of CD19 and BCMA-directed CAR-T cell therapy in B-cell lymphoma (7, 8) and multiple myeloma (MM) (9, 10), approved by the U.S. Food and Drug Administration (FDA) and China’s National Medical Products Administration (NMPA), have substantially shifted the clinical research emphasis towards the treatment of solid tumors and non-neoplastic disorders (11). The gene discussed is TNFRSF17; the disease is Miyoshi myopathy.