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• W2161314897 abstract "Background Bernard-Soulier syndrome (BSS) is an inherited bleeding disorder characterized by macrothrombocytopenia. Platelet transfusion is used for the management of bleeding, but repeated transfusion often results in alloimmunization. We have recently shown phenotypic correction of murine BSS (GPIbαnull) using lethal radiation conditioning followed by hematopoietic lentivirus-mediated gene transfer. Objectives For application of gene therapy to treatment of human patients, it is important to minimize treatment-related side effects. The objective of this study is to model a clinically relevant non-myeloablative hematopoietic stem cell (HSC) transplantation strategy. Methods Using transplantation of bone marrow (BM) HSCs from transgenic mice that express hGPIbα (hGPIbαtg+/+), we sought to (i) determine the percentage of hGPIbαtg+/+ HSCs required for therapeutic benefit, (ii) evaluate the efficacy of non-myeloablative conditioning using busulfan, and (iii) test the ability of anti-thymocyte globulin (ATG) to prevent/reduce undesirable immune responses. Results Transplantation of 10–20% hGPIbαtg+/+ BM HSCs mixed with GPIbαnull BM HSCs into irradiated GPIbαnull mice was sufficient to correct bleeding time (n = 5). Transplantation of hGPIbαtg+/+ BM HSCs into busulfan-conditioned GPIbαnull mice corrected bleeding time in 21 of 27 recipients. Antibody response to hGPIbα and immune-mediated thrombocytopenia was documented in eight of 27 recipients, suggesting immunogenicity of hGPIbα in busulfan-conditioned GPIbαnull mice. However, these antibodies disappeared without treatment within 30 weeks after transplantation. A combination of busulfan plus ATG conditioning successfully prevented antibody development and significantly increased therapeutic engraftment. Conclusion A conditioning regimen of busulfan in combination with ATG could potentially be used in non-myeloablative autologous gene therapy in human BSS." @default.
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• W2161314897 date "2014-10-01" @default.
• W2161314897 modified "2023-10-14" @default.
• W2161314897 title "Non‐myeloablative conditioning with busulfan before hematopoietic stem cell transplantation leads to phenotypic correction of murine Bernard‐Soulier syndrome" @default.
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• W2161314897 doi "https://doi.org/10.1111/jth.12673" @default.
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