We had previously reported that transduction of the TNALP-D10-expressing self-complementary AAV type 8 (scAAV8) vector into Akp2−/− (TNALP-knockout) mice improved the skeletal bone defects in animals and prolonged their survival.12, 13 The AAV vector is suitable for in vivo gene delivery and has been widely used for vector-mediated ERTs for genetic diseases. This evidence concerns the gene ALPL and hereditary disease.