CONTINUATION PAGE: STAY WITHIN MARGINS INDICATED 
Principal Investigator/Program Director (Last, first, middle): l 
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Tsuji, S., Choudary, P.V., Martin, B.M., Stubblefield, B.K., Mayor, J.A., Barranger, J.A. and Ginns, E.I. A mutation in the 
human glucocerebrosidase gene in neuronopathic Gaucher’s disease. New Engl. J. Med. 316:570-575, 1987. 
Das, P.K., Murray, G.J., Gal, A.E. and Barranger, J.A. Glucocerebrosidae deficiency and lysosomal storage of glucocerebroside 
induced in cultured macrophage. Exp. Cell Res. 168:463-474, 1987. 
Jonsson, L.M.V., Murray, G.J., Sorrell, S.H., Strijland, A., Aerts, J.F.G.M., Ginns, E.I., Barranger, J.A., Tager, J.M. and 
Schram, A.W. Biosynthesis and maturation of glucocerebrosidase in Gaucher fibroblasts. Eur. J. Biochem. 164:171-179, 
1987. 
Tsuji, S., Martin, B.M. Barranger, J.A., Stubblefield, B., Lamarca, M., Ginns, E.G. Genetics heterogeneity in Type 1 Gaucher 
Disease: Multiple Genotypes in Ashkenazic and non-Ashkenazic individuals. Proc. Natl. Acad. Sci. 85:2349-2352, 1988. 
Choudary, P.V., Tsuji, S., Martin, B.M., Guild, B.C., Mulligan, R.C., Murray, G.J., Barranger, J.A. and Ginns, E.I. The 
molecular biology of Gaucher disease and potential for gene therapy. In: Cold Spring Harbor Symposium on Quantitative 
Biology. Vol. LI, pp. 1047-1052, 1986. 
Aerts, J.M.F.G., Schram, A.W., Strijland, A., van Weely, S., Jonsson, L.M.V., Tager, J.M., Sorrell, S.H., Ginns, E.I., 
Barranger, J.A. and Murray, G.J. Glucocerebrosidase, a lysosomal enzyme that does not undergo oligosaccaharide 
phosphorylation. Biochimica Biophys Acta. , 964:303-308, 1988. 
Barranger, J.A. and Ginns, E.I. Glucosylceramide lipidosis: Gaucher disease. In: The Metabolic Basis of Inherited Disease, 6th 
Editon, pp. 1677-1698, 1989. 
Barranger, J.A., Ohashi, T., Hong, C. M., Tomich, J., Aerts, J.F.G.M., Tager, J. M., Nolta, J. A., Sender, L. S., Weiler, S., I 
and Kohn, D.B. Molecular Pathology and Therapy of Gaucher Disease. Japanese Journal of Inker. Met. Disease 51: 45- 
71, 1989. 
Kohn, D.B., Nolta, J.A., Hong, C.M. and Barranger, J.A. In: Gene Transfer and Gene Therapy, A.L. Beaudet, R. Mulligan, am 
I. M. Verma (eds) pp. 397-408, 1989. 
Nolta, J.A., Sender, L.S., Barranger, J.A., and Kohn, D.B. Expression of human glucocerebrosidase in murine long-term bone 
marrow cultures following retroviral vector-mediated transfer. Blood IS: 787-797, 1990. 
Morimoto, S., Kishimoto, Y., Tomich, J., Weiler, S., Ohashi, T., Barranger, J.A., Kretz, K.A., and O’Brien, J.S. Interaction of 
saposins, acidic lipids and glucosylceramidase. J. Biol. Chem. 265: 1933-1937, 1990. 
Hong, C.M., Ohashi, T., Yu, X.J., Weiler, S. and Barranger, J.A. Sequence of two alleles responsible for Gaucher Disease. 
DNA and Cell Biology 9: 233-241, 1990. 
Mankin, H.J., Doppelt, S., Rosenberg, A.E. and Barranger, J.A. Metabolic bone disease in patients with Gaucher’s disease. 
Metabolic Basis of Bone Disease. L. V. Alvioli and S. M. Krane (eds.), pp. 730-752, 1990. 
Carlson, D.E., Busuttil, R.W., Guidici, T.A., and Barranger, J.A. Orthotopic liver transplantation in the treatment of complication 
of Type 1 Gaucher Disease. Transplantation 49:1192-1194, 1990. 
Aerts, J.M.D.G., Donker-Koopman, W.E., Brul, S., van Weeley, S., Sa Miranda, M.C., Barranger, J.A., Tager, J. M., and 
Schram, A.W. Comparative study on glucocerebrosidase in spleens from patients with Gaucher disease. Biochemical 
Journal 269:93-100, 1990. 
Aerts, J.M.F.G., Sa Miranda, M.C., Brouwer-Kelder, E.M., van Weely, S., Barranger, J.A., and Tager, J.M. Conditions 
affecting the activity of glucocerebrosidase purified from spleens of control subjects and patients with Type 1 Gaucher 
disease. Biochimica Biophys Acta. , 1041:55-63, 1990. 
Sa Miranda, M.C., Aerts, J.M.F.G., Pinto, R., Fontes, A., de Lacerda, L.W., van Weeley, S., Barranger, J.A., Tager, J.M. 
Activity of glucocerebrosidase in extracts of different cell types from Type 1 Gaucher Disease patients. Clin. Genetics 38, 
218-227, 1990. 
Schram, A.W., Aerts, J.F.M.G., van Weeley, S., Barranger, J.A. and Tager, J.M. Glucocerebrosidase, a membrane-associated 
lysosomal enzyme deficient in Gaucher disease. In: Methodological Surveys in Biochemistry and Analysis, (in press) 
Van Weeley, S., Aerts, J.M.F.G., van Leeuwen, M.B., Heikoop, J.C., Donker-Koopman, W.E., Barranger, J.A., Tager, J.M., 
and Schram, A.W. Nature and function of oligosaccharide processing in glucocerebrosidase, A membrane-associated 
lysosomal hydrolase, Eur. J. Biochem. 191:669-677, 1990. 
Shigematsu, H., Morimoto, S., Kishimoto, Y., Weiler, S., Tomich, J., Barranger, J. A., Shinohara, M., Yeager, A., O’Brien, J.S 
Saposins (Sphingolipid Activator Proteins) in the twitcher mutant mouse. J. Neurochem. 55:1659-1662, 1990. 
Ohashi, T., Hong, C.M., Weiler, S., Tomich, J., Aerts, J.M.F.G., Tager, J.M. and Barranger, J.A. Characterization of mutant 
glucocerebrosidases from different alleles. J. Biol. Chem, 266:3661-3667, 1991. 
Van Weeley, S., van Leeuwen, M.B., Jansen, I.D.C., De Bruijn, M.A.C., Brouwer-Kelder, E.M., Schram, A. W., Sa Miranda, 
M.C., Barranger, J.A., Petersen, E.M., Goldblatt, J., Stotz, H., Sandhoff, K., Svennerholm, L., Erikson, A., Tager, 
J. M. and Aerts, J.M.F.G. Clinical phenotype of Gaucher disease in relation to properties of mutant glucocerebrosidase in 
cultured fibroblasts. Biochemica et Biophysica Acta. 1096:301-311, 1991. 
Ohashi, T., Boggs, S., Robbins, P., Bahnson, A., Patrene, K., Wei, F-S., Wei, J-F., Li, J., Lucht, L., Fei, Y., Clark, S., Kimak 
M., He, H., Mowery-Rushton, P. and Barranger, J.A. Efficient transfer and sustained high expression of the human 
glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by 
a retroviral vector. Proc. Natl. Acad. Sci. USA 89:11332-11336, 1992. 
Bahnson, A.B., Boggs, S.S., Ohashi, T., Robbins, P.D., Patrene, K., Li, J., Lucht, L., Mowery-Rushton, P., Nimgaonkar, M., 
Fei, Y. and Barranger, J.A. Transduction and expression of the human glucocerebrosidase gene in the long term murine 
model and in rhesus monkey bone marrow and human CD34+ cells in vitro. Proceedings of the Sixth International 
Symposium on Autologous BMT. (eds) Dicke, K. and Keating, A., 1993. (In press). 
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