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Sarna, J
Miranda, SRP
Schuchman, EH
Hawkes, R
Citation: J. Sarna et al., Patterned cerebellar Purkinje cell death in a transgenic mouse model of Niemann Pick type A/B disease, EUR J NEURO, 13(10), 2001, pp. 1873-1880
Authors:
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Erlich, S
Friedrich, VL
Gatt, S
Schuchman, EH
Citation: Srp. Miranda 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 THER, 7(20), 2000, pp. 1768-1776
Authors:
Marathe, S
Miranda, SRP
Devlin, C
Johns, A
Kuriakose, G
Williams, KJ
Schuchman, EH
Tabas, I
Citation: S. Marathe et al., 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 function, HUM MOL GEN, 9(13), 2000, pp. 1967-1976
Authors:
Miranda, SRP
He, XX
Simonaro, CM
Gatt, S
Dagan, A
Desnick, RJ
Schuchman, EH
Citation: Srp. Miranda et al., Infusion of recombinant human acid sphingomyelinase into Niemann-Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology, FASEB J, 14(13), 2000, pp. 1988-1995
Authors:
Schuchman, EH
Erlich, S
Miranda, SRP
Dinur, T
Dagan, A
Gatt, S
Citation: Eh. Schuchman et al., Fluorescence-based selection of gene-corrected hematopoietic stem and progenitor cells based on acid sphingomyelinase expression, METH ENZYM, 312, 2000, pp. 330-338
Authors:
He, XX
Miranda, SRP
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Dagan, A
Gatt, S
Schuchman, EH
Citation: Xx. He et al., Characterization of human acid sphingomyelinase purified from the media ofoverexpressing Chinese hamster ovary cells, BBA-PROT ST, 1432(2), 1999, pp. 251-264
Authors:
Erlich, S
Miranda, SRP
Visser, JWM
Dagan, A
Gatt, S
Schuchman, EH
Citation: S. Erlich et al., 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(1), 1999, pp. 80-86