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Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity.

Shi Q, Fahs SA, Wilcox DA, Kuether EL, Morateck PA, Mareno N, Weiler H, Montgomery RR.

Blood. 2008 Oct 1;112(7):2713-21. doi: 10.1182/blood-2008-02-138214. Epub 2008 May 21.


The important role of von Willebrand factor in platelet-derived FVIII gene therapy for murine hemophilia A in the presence of inhibitory antibodies.

Shi Q, Schroeder JA, Kuether EL, Montgomery RR.

J Thromb Haemost. 2015 Jul;13(7):1301-9. doi: 10.1111/jth.13001. Epub 2015 Jun 11.


Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing anti-factor VIII immunity.

Kuether EL, Schroeder JA, Fahs SA, Cooley BC, Chen Y, Montgomery RR, Wilcox DA, Shi Q.

J Thromb Haemost. 2012 Aug;10(8):1570-80. doi: 10.1111/j.1538-7836.2012.04791.x.


The immunogenicity of platelet-derived FVIII in hemophilia A mice with or without preexisting anti-FVIII immunity.

Chen Y, Schroeder JA, Chen J, Luo X, Baumgartner CK, Montgomery RR, Hu J, Shi Q.

Blood. 2016 Mar 10;127(10):1346-54. doi: 10.1182/blood-2015-08-662916. Epub 2015 Dec 14.


Platelet gene therapy corrects the hemophilic phenotype in immunocompromised hemophilia A mice transplanted with genetically manipulated human cord blood stem cells.

Shi Q, Kuether EL, Chen Y, Schroeder JA, Fahs SA, Montgomery RR.

Blood. 2014 Jan 16;123(3):395-403. doi: 10.1182/blood-2013-08-520478. Epub 2013 Nov 22.


In vivo enrichment of genetically manipulated platelets corrects the murine hemophilic phenotype and induces immune tolerance even using a low multiplicity of infection.

Schroeder JA, Chen Y, Fang J, Wilcox DA, Shi Q.

J Thromb Haemost. 2014 Aug;12(8):1283-93. doi: 10.1111/jth.12633. Epub 2014 Jul 17.


Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity.

Doering CB, Gangadharan B, Dukart HZ, Spencer HT.

Mol Ther. 2007 Jun;15(6):1093-9. Epub 2007 Mar 27.


Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A.

Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, DU LM, Desai D, Montgomery RR.

J Thromb Haemost. 2007 Feb;5(2):352-61.


Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies.

Shi Q, Wilcox DA, Fahs SA, Weiler H, Wells CW, Cooley BC, Desai D, Morateck PA, Gorski J, Montgomery RR.

J Clin Invest. 2006 Jul;116(7):1974-82.


Correction of murine hemophilia A by hematopoietic stem cell gene therapy.

Moayeri M, Hawley TS, Hawley RG.

Mol Ther. 2005 Dec;12(6):1034-42. Epub 2005 Oct 12.


Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens.

Ide LM, Gangadharan B, Chiang KY, Doering CB, Spencer HT.

Blood. 2007 Oct 15;110(8):2855-63. Epub 2007 Jun 14.


Functional aspects of factor VIII expression after transplantation of genetically-modified hematopoietic stem cells for hemophilia A.

Ide LM, Iwakoshi NN, Gangadharan B, Jobe S, Moot R, McCarty D, Doering CB, Spencer HT.

J Gene Med. 2010 Apr;12(4):333-44. doi: 10.1002/jgm.1442.


Factor VIII delivered by haematopoietic stem cell-derived B cells corrects the phenotype of haemophilia A mice.

Ramezani A, Zweier-Renn LA, Hawley RG.

Thromb Haemost. 2011 Apr;105(4):676-87. doi: 10.1160/TH10-11-0725. Epub 2011 Jan 25.


Comparison of platelet-derived and plasma factor VIII efficacy using a novel native whole blood thrombin generation assay.

Baumgartner CK, Zhang G, Kuether EL, Weiler H, Shi Q, Montgomery RR.

J Thromb Haemost. 2015 Dec;13(12):2210-9. doi: 10.1111/jth.13169. Epub 2015 Nov 25.


High-level expression of porcine factor VIII from genetically modified bone marrow-derived stem cells.

Gangadharan B, Parker ET, Ide LM, Spencer HT, Doering CB.

Blood. 2006 May 15;107(10):3859-64. Epub 2006 Jan 31.


Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.

Chen Y, Schroeder JA, Kuether EL, Zhang G, Shi Q.

Mol Ther. 2014 Jan;22(1):169-77. doi: 10.1038/mt.2013.197. Epub 2013 Aug 23.


Phenotypic correction of hemophilia A by ectopic expression of activated factor VII in platelets.

Ohmori T, Ishiwata A, Kashiwakura Y, Madoiwa S, Mitomo K, Suzuki H, Hasegawa M, Mimuro J, Sakata Y.

Mol Ther. 2008 Aug;16(8):1359-65. doi: 10.1038/mt.2008.117. Epub 2008 Jun 3.


Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice.

Zhang G, Shi Q, Fahs SA, Kuether EL, Walsh CE, Montgomery RR.

Blood. 2010 Aug 26;116(8):1235-43. doi: 10.1182/blood-2009-11-255612. Epub 2010 May 5.


Development of improved factor VIII molecules and new gene transfer approaches for hemophilia A.

Saenko EL, Ananyeva NM, Moayeri M, Ramezani A, Hawley RG.

Curr Gene Ther. 2003 Feb;3(1):27-41. Review.


Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells.

Moayeri M, Ramezani A, Morgan RA, Hawley TS, Hawley RG.

Mol Ther. 2004 Nov;10(5):892-902. Erratum in: Mol Ther. 2005 Sep;12(3):579.

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