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Blood|July 1, 1997
Bone marrow transplantation in acid sphingomyelinase-deficient mice: engraftment and cell migration into the brain as a function of radiation, age, and phenotypeS R Miranda, S Erlich, J W Visser, et al.Molecular Genetics and Metabolism|June 5, 1999
Identification and characterization of the molecular lesion causing mucopolysaccharidosis type I in catsX He, C M Li, C M Simonaro, et al.Human Molecular Genetics|August 15, 2000
Creation of a mouse model for non-neurological (type B) Niemann-Pick disease by stable, low level expression of lysosomal sphingomyelinase in the absence of secretory sphingomyelinase: relationship between brain intra-lysosomal enzyme activity and central nervous system functionS Marathe, S R Miranda, C Devlin, et al.The EMBO Journal|September 1, 1989
Isolation of cDNA clones encoding human acid sphingomyelinase: occurrence of alternatively processed transcriptsL E Quintern, E H Schuchman, O Levran, et al.Human Gene Therapy|August 1, 1995
Fluorescence-based selection of retrovirally transduced cells in the absence of a marker gene: direct selection of transduced type B Niemann-Pick disease cells and evidence for bystander correctionP L Yeyati, V Agmon, C Fillat, et al.The Tohoku Journal of Experimental Medicine|October 1, 1995
Identification and expression of a missense mutation (Y446C) in the acid sphingomyelinase gene from a Japanese patient with type A Niemann-Pick diseaseT Takahashi, M Suchi, W Sato, et al.Journal of Inherited Metabolic Disease|January 24, 2004
Niemann-Pick disease: sixteen-year follow-up of allogeneic bone marrow transplantation in a type B variantS Victor, J B S Coulter, G T N Besley, et al.Osteoarthritis and Cartilage|November 3, 2015
Acid ceramidase treatment enhances the outcome of autologous chondrocyte implantation in a rat osteochondral defect modelM E Frohbergh, J M Guevara, R P Grelsamer, et al.Gene Therapy|May 26, 1999
Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablationC M Simonaro, M E Haskins, J L Abkowitz, et al.Journal of Inherited Metabolic Disease|September 19, 2007
Acid sphingomyelinase-deficient Niemann-Pick disease: novel findings in a Greek childM Fotoulaki, E H Schuchman, C M Simonaro, et al.Pageof 8