“…This dose is in the range currently used in clinical and pre-clinical treatment of Niemann-Pick disease and various lysosomal storage disorders. 42 , 43 , 44 , 45 , 46 , 47 To further decrease the suggested dose, we could use serotypes that have increased tropism to the liver; AAV-DJ, for example, is an engineered strain created by capsid shuffling that has been shown to be highly specific to the liver. 48 …”