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Miranda, S R; He, X; Simonaro, C M et al. (2000) Infusion of recombinant human acid sphingomyelinase into niemann-pick disease mice leads to visceral, but not neurological, correction of the pathophysiology. FASEB J 14:1988-95
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Erlich, S; Miranda, S R; Visser, J W et al. (1999) Fluorescence-based selection of gene-corrected hematopoietic stem and progenitor cells from acid sphingomyelinase-deficient mice: implications for Niemann-Pick disease gene therapy and the development of improved stem cell gene transfer procedures. Blood 93:80-6
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Chen, F W; Davies, J P; Ioannou, Y A (1998) Differential gene expression in apoptosis: identification of ribosomal protein 23K, a cell proliferation inhibitor. Mol Genet Metab 64:271-82
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Schuchman, E H; Miranda, S R (1997) Niemann-Pick disease: mutation update, genotype/phenotype correlations, and prospects for genetic testing. Genet Test 1:13-9
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Davies, J P; Cotter, P D; Ioannou, Y A (1997) Cloning and mapping of human Rab7 and Rab9 cDNA sequences and identification of a Rab9 pseudogene. Genomics 41:131-4
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