To determine CAR-T cells killing human MM cells in vivo, immunodeficient NOD.Cg-PrkdcscidIl2rgtm1Wjl/SzJ (NSG) mice were injected human MM cell lines MM1.S and OPM2 and subsequently treated with CAR-T cells or PBS (Fig. 6a). The gene discussed is CTSG; the disease is Miyoshi myopathy.