Our previous study using focal intramuscular injection of AAV6-siPTEN to silence PTEN in mice in vivo has demonstrated successful knockdown of PTEN levels in SMA animals, as well as successful targeting of motor neurons in the ventral horn of the spinal cord.16 Here, we initially extended our findings to show that intramuscular injection of AAV6-siPTEN can ameliorate NMJ pathology in SMA mice. Here, PTEN is linked to proximal spinal muscular atrophy.