When targeting the muscle in humans, T-cell responses directed to the capsid antigen were documented in AAT-deficient subjects receiving intra-muscular injection of an AAV1–AAT vector (25, 55), in study on AAV1-α-sarcoglycan in limb-girdle muscular dystrophy subjects (35) and in our own LPL-deficient subjects. Here, SERPINA1 is linked to limb-girdle muscular dystrophy.