Targeting platelet GPIbα transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbβ and GPIX

Clemetson, 1982, Characterization of the platelet membrane glycoprotein abnormalities in Bernard–Soulier syndrome and comparison with normal by surface‐labeling techniques and high‐resolution two‐dimensional gel electrophoresis, J Clin Invest, 70, 304, 10.1172/JCI110618 Hagen, 1980, Immunochemical evidence for protein abnormalities in platelets from patients with Glanzmann's thrombasthenia and Bernard–Soulier syndrome, J Clin Invest, 65, 722, 10.1172/JCI109719 Du, 1987, Glycoprotein Ib and glycoprotein IX are fully complexed in the intact platelet membrane, Blood, 69, 1524, 10.1182/blood.V69.5.1524.1524 Modderman, 1992, Glycoproteins V and Ib‐IX form a noncovalent complex in the platelet membrane, J Biol Chem, 267, 364, 10.1016/S0021-9258(18)48503-1 Michelson, 1996, Effects of nitric oxide/EDRF on platelet surface glycoproteins, Am J Physiol, 270, H1640 Murata, 1991, Site‐directed mutagenesis of a soluble recombinant fragment of platelet glycoprotein Ib alpha demonstrating negatively charged residues involved in von Willebrand factor binding, J Biol Chem, 266, 15474, 10.1016/S0021-9258(18)98640-0 Lopez, 1998, Bernard–Soulier syndrome, Blood, 91, 4397, 10.1182/blood.V91.12.4397 Poujol, 2000, Ultrastructural analysis of megakaryocytes in GPV knockout mice, Thromb Haemost, 84, 312, 10.1055/s-0037-1614013 Peters, 1998, Treatment of a patient with Bernard–Soulier syndrome and recurrent nosebleeds with recombinant factor VIIa, Thromb Haemost, 80, 352, 10.1055/s-0037-1615209 Monroe, 2000, The factor VII–platelet interplay: effectiveness of recombinant factor VIIa in the treatment of bleeding in severe thrombocytopathia, Semin Thromb Hemost, 26, 373, 10.1055/s-2000-8455 Almeida, 2003, The use of recombinant factor VIIa in children with inherited platelet function disorders, Br J Haematol, 121, 477, 10.1046/j.1365-2141.2003.04286.x Kaleelrahman, 2004, Use of recombinant factor VIIa in inherited platelet disorders, Br J Haematol, 125, 95, 10.1111/j.1365-2141.2004.04878.x Locatelli, 2003, Hematopoietic stem cell transplantation for the Bernard–Soulier syndrome, Ann Intern Med, 138, 79, 10.7326/0003-4819-138-1-200301070-00028 Vollweiler, 2003, Hematopoietic stem cell gene therapy: progress toward therapeutic targets, Bone Marrow Transplant, 32, 1, 10.1038/sj.bmt.1704081 Wagner, 1996, Analysis of GPIIb/IIIa receptor number by quantification of 7E3 binding to human platelets, Blood, 88, 907, 10.1182/blood.V88.3.907.907 Prandini, 1996, The tissue‐specific transcriptional regulation of the megakaryocytic glycoprotein IIb gene is controlled by interactions between a repressor and positive cis‐acting elements, Blood, 88, 2062, 10.1182/blood.V88.6.2062.bloodjournal8862062 Wilcox, 2003, Gene therapy for platelet disorders: studies with Glanzmann's thrombasthenia, J Thromb Haemost, 1, 2300, 10.1046/j.1538-7836.2003.00476.x Wilcox, 1999, Integrin alphaIIb promoter‐targeted expression of gene products in megakaryocytes derived from retrovirus‐transduced human hematopoietic cells, Proc Natl Acad Sci USA, 96, 9654, 10.1073/pnas.96.17.9654 Wilcox, 2000, Megakaryocyte‐targeted synthesis of the integrin beta(3)‐subunit results in the phenotypic correction of Glanzmann thrombasthenia, Blood, 95, 3645, 10.1182/blood.V95.12.3645.012k51a_3645_3651 Shi, 2003, Expression of human factor VIII under control of the platelet‐specific alphaIIb promoter in megakaryocytic cell line as well as storage together with VWF, Mol Genet Metab, 79, 25, 10.1016/S1096-7192(03)00049-0 Miller, 1990, Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection, Mol Cell Biol, 10, 4239 Lewis, 1994, Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus, J Virol, 68, 510, 10.1128/jvi.68.1.510-516.1994 Bukrinsky, 1993, A nuclear localization signal within HIV‐1 matrix protein that governs infection of non‐dividing cells, Nature, 365, 666, 10.1038/365666a0 Naldini, 1996, In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector, Science, 272, 263, 10.1126/science.272.5259.263 Uchida, 1998, HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells, Proc Natl Acad Sci USA, 95, 11939, 10.1073/pnas.95.20.11939 Reiser, 1999, Transduction of nondividing cells using pseudotyped defective high‐titer HIV type 1 particles, Proc Natl Acad Sci USA, 93, 15266, 10.1073/pnas.93.26.15266 Bai, 2003, Effective transduction and stable transgene expression in human blood cells by a third‐generation lentiviral vector, Gene Ther, 10, 1446, 10.1038/sj.gt.3302026 Moran, 2000, Surface expression of glycoprotein Ib alpha is dependent on glycoprotein Ib beta: evidence from a novel mutation causing Bernard–Soulier syndrome, Blood, 96, 532, 10.1182/blood.V96.2.532.014k33_532_539 Zufferey, 1998, Self‐inactivating lentivirus vector for safe and efficient in vivo gene delivery, J Virol, 72, 9873, 10.1128/JVI.72.12.9873-9880.1998 Zufferey, 1997, Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo, Nat Biotechnol, 15, 871, 10.1038/nbt0997-871 Dull, 1998, A third‐generation lentivirus vector with a conditional packaging system, J Virol, 72, 8463, 10.1128/JVI.72.11.8463-8471.1998 Kenny, 1999, The critical interaction of glycoprotein (GP) IBbeta with GPIX‐a genetic cause of Bernard–Soulier syndrome, Blood, 93, 2968, 10.1182/blood.V93.9.2968.409a15_2968_2975 Greenberg, 1988, Characterization of a new megakaryocytic cell line: the Dami cell, Blood, 72, 1968, 10.1182/blood.V72.6.1968.1968 Lopez, 1987, Cloning of the alpha chain of human platelet glycoprotein Ib: a transmembrane protein with homology to leucine‐rich alpha 2‐glycoprotein, Proc Natl Acad Sci USA, 84, 5615, 10.1073/pnas.84.16.5615 Lopez, 1988, The alpha and beta chains of human platelet glycoprotein Ib are both transmembrane proteins containing a leucine‐rich amino acid sequence, Proc Natl Acad Sci USA, 85, 2135, 10.1073/pnas.85.7.2135 Hickey, 1989, Human platelet glycoprotein IX: an adhesive prototype of leucine‐rich glycoproteins with flank‐center‐flank structures, Proc Natl Acad Sci USA, 86, 6773, 10.1073/pnas.86.17.6773 Lanza, 1993, Cloning and characterization of the gene encoding the human platelet glycoprotein V. A member of the leucine‐rich glycoprotein family cleaved during thrombin‐induced platelet activation, J Biol Chem, 268, 20801, 10.1016/S0021-9258(19)36855-3 Andrews, 1992, Identification of a region in the cytoplasmic domain of the platelet membrane glycoprotein Ib‐IX complex that binds to purified actin‐binding protein, J Biol Chem, 267, 18605, 10.1016/S0021-9258(19)37004-8 Andrews, 1991, Interaction of purified actin‐binding protein with the platelet membrane glycoprotein Ib–IX complex, J Biol Chem, 266, 7144, 10.1016/S0021-9258(20)89622-7 Kenny, 2002, The cysteine knot of platelet glycoprotein Ib beta (GPIb beta) is critical for the interaction of GPIb beta with GPIX, Blood, 99, 4428, 10.1182/blood.V99.12.4428