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Current SCID Gene Transfer Experience
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Gaspar HB, Parsley KL, Howe S, King D, Gilmour KC, et al. (2004) Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364:2181-2187. |
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Schmidt M, Hacein-Bey-Abina S, Wissler M, Carlier F, Lim A, et al. (2005) Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial. Blood 105(7):2699-2706. |
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Retrovirus Integration and Insertional Mutagenesis
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Akagi K, Suzuki T, Stephens RM, Jenkins NA and Copeland NG. (2004) RTCGD: retroviral tagged cancer gene database. Nucl Acid Res 32:Database Issue D523-D527. |
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Davé UP, Jenkins NA and Copeland NG. (2004) Gene therapy insertional mutagenesis insights. Science 303:333. |
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von Kalle C, Fehse B, Layh-Schmitt G, Schmidt M, Kelly P and Baum C. (2004 ) Stem cell clonality and genotoxicity in hematopoietic cells: Gene activation side effects should be avoidable. Semin Hematol 41:303-318. |
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Mitchell RS, Beitzel BF, Schroder ARW, Shinn P, Chen H, et al. (2004) Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2(8):e234. |
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Bone Marrow Transplantation for SCID
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Antoine C, Müller S, Cant A, Cavazzana-Calvo M, Veys P, et al. (2003) Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 196899. Lancet 361:553-560. |
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Barker JN and Wagner JE. (2003) Umbilical cord blood transplantation : Current practice and future innovations. Crit Rev Oncol Hematol 48:35-43. |
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Buckley RH. (2003) Treatment options for genetically determined immunodeficiency. Lancet 361:541. |
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Friedrich W and Müller SM. (2004) Allogeneic stem cell transplantation for treatment of immunodeficiency. Springer Semin Immun 26:109-118. |
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Myers LA, Patel DD, Puck JM and Buckley RH. (2002) Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 99:872-878. |
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Rao K, Amrolia PJ, Jones A, Cale CM, Naik P, et al. (2005) Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen. Blood 105(2):879-885. |
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