Expression of CD95 on mature leukocytes of MRL/lpr mice after transplantation of genetically modified bone marrow stem cells.
Bone marrow transplantation (BMT) is commonly used for the treatment of severe haematological and immunological diseases. For instance, the autoimmune lymphoproliferative syndrome (ALPS) caused by a complete expression defect of CD95 (Fas, APO-1) can be cured by allogeneic BMT. However, since this therapy may not generate satisfactory results when only partially compatible donors are available, we were interested in the development of a potential alternative treatment by using lentiviral gene transfer of a normal copy of CD95 cDNA in hematopoietic stem cells. Here, we show that this approach applied to MRL/lpr mice results in the expression of functional CD95 receptors on the surface of lymphocytes, monocytes, and granulocytes. This suggests that correction of CD95 deficiency can be achieved by gene therapy.
Federzoni
Elena
E
Institute of Pharmacology, University of Bern, Friedbühlstrasse 49, CH-3010 Bern, Switzerland.
Gordon
Grace
G
Müller
Stefan
S
Schmid
Inès
I
Simon
Hans-Uwe
HU
Yousefi
Shida
S
D000818:Animals; D001854:Bone Marrow Cells / Q000166:cytology; D005260:Female; D015870:Gene Expression; D006098:Granulocytes / Q000276:immunology; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000378:metabolism; D016086:Lentivirus / Q000235:genetics; D007962:Leukocytes / Q000276:immunology*; D051379:Mice; D019463:Mice, Inbred MRL lpr; D009000:Monocytes / Q000276:immunology; D013601:T-Lymphocytes / Q000276:immunology; D014161:Transduction, Genetic; D019014:fas Receptor / Q000235:genetics* / Q000378:metabolism
D019014:fas Receptor