Although LARGE overexpression by transgenic approach exacerbates muscular dystrophy in FKRP knock-down mice [20] and fukutin knockout mice [21] thought to be caused by inhibition of regeneration from satellite cells, we have shown that LARGE expression ameliorates muscular dystrophy in LARGE mutant and POMGnT1 knockout mice when delivered systemically by an adeno-associated viral vector 9 (AAV9) after birth [22]. The gene discussed is FKRP; the disease is muscular dystrophy.