7. Erikson A, Groth CG, Mausson J-E, Percy A, Ringden O, SvennerhoLm L: Clinical and 
biochemical outcome of marrow transplantation for Gaucher disease of the Norrbottnian type. 
Acta Paediatr Scand 79:680-685, 1990. 
8. Rappeport JM, Barranger JA, Ginns El: Bone marrow transplantation in Gaucher disease. Birth 
Defects 22:101-109, 1986. 
9. Tsai P, Lipton JM, Sahdev I, Najfeld V, Rankin LR, Slyper AH, Ludman M, Grabowski GA: 
Allogeneic bone marrow transplantation in severe Gaucher disease. Ped Res 31:503-507, 1992. 
10. Barton NW, Furbish FS, Murray GJ, Garfield M, Brady RO: Therapeutic response to 
intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad 
Sci, USA 87:1913-1916, 1990. 
11. Barton NW, Brady RO, Dambrosia JM, DiBceghe AM, et al: Replacement therapy for inherited 
enzyme deficiency - macrophage-targeted glucocerebrosidase for Gaucher disease. N Eng J Med 
324:1464-1470, 1991. 
12. Beutler E, Kay A, Saven A, Garver P, Thurston D, Dawson A Rosenbloom B: Enzyme 
replacement therapy for Gaucher disease. Blood 78:1183-1189, 1991. 
13. Figueroa ML, Rosenbloom BE, Kay AC, Garver P, et al: A less costly regimen of alglucerase to 
treat Gaucher’s disease. N Eng J Med 327:1632-1636, 1992. 
14. Correll PM, Kew Y, Perry LK, Brady RO, Fink JK, Karlsson S: Expression of human gluco- 
cerebrosidase in long-term reconstituted mice following retroviral-mediated gene transfer into 
hematopoietic stem cell. Hum Gene Ther 1:227-287, 1990. 
15. Weinthal J, Nolta JA, Yu XJ, Lilley J, Uribe L., Kohn DB: Expression of human gluco- 
cerebrosidase following retroviral-mediated transduction of murine hematopoietic stem cells. 
Bone Marrow Transplant 8:403-412, 1991. 
16. Correll PM, Colilla S, Dave HPG, Karlsson S: High levels of human glucocerebrosidase activity 
in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem 
cells. Blood 80:331-336, 1992. 
17. Ohasi T, Boggs S, Robbins P, Bahnson A, Patrene K, et al: 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. PNAS 89:11332- 
11336, 1992. 
18. Schuening FG, Kawahara K, Miller AD, To R, Goetile S, Stewart D, Mullally K, Fisher L, 
Graham TC, Appelbaum FR, Hackman R, Osborne WRA, Storb R: Retrovirus-mediated gene 
transduction into long term repopulating marrow cells of dogs. Blood 78:2568-1576, 1991. 
19. Fink JK, Correll PM, Perry LK, Brady RO, Karlsson S: Correction of glucocerebrosidase 
deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from 
patients with Gaucher disease. Proc Natl Acad Sci, USA 87:2334-2338, 1990. 
20. Nolta JA, Yu XJ, Bahner I, Kohn DB: Retroviral mediated transfer of the human 
glucocerebrosidase gene into cultured Gaucher bone marrow. J Clin Invest 90:343-348, 1992. 
21. Nimgaonkar M, Bahnson A, Boggs S, Robbins PD, et al: Transduction of G-CSF primed 
peripheral blood CD34 + cells with the gene for glucocerebrosidase. J of Cellular Biochemistry 
Suppl 17E, 233, 1993 (abstract). 
22. Brenner MK, Rill DR, Moen RC, Krance RA, Mirro Jr. J, Anderson WF, Ihle JN: Gene- 
marking to trace origin of relapse after autologous bone marrow transplantation. Lancet 341:85- 
86, 1993. 
23. Brecher G, Ansel JD, Micklem HS, Tjio JH, Cronkite EP: Special proliferative sites are not 
needed for seeding and proliferation of transfused bone marrow cells in normal syngeneic mice. 
PNAS 79:5085-5087, 1982. 
Recombinant DNA Research, Volume 18 
[679] 
