1Miranda SR,Erlich S,et al.Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease[].Gene Therapy.2000
2Stanbury JB,Wyngaarden JB,et al.Matabolic Basis of Inherited Disease[]..1987
3Crocker AC,et al.The defect in Tay-Sachs disease and NiemannPick disease[].Neurochem.1961