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Use of blood outgrowth endothelial cells for gene therapy for hemophilia A.

Lin Y, Chang L, Solovey A, Healey JF, Lollar P, Hebbel RP.

Blood. 2002 Jan 15;99(2):457-62.


Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors.

Matsui H, Shibata M, Brown B, Labelle A, Hegadorn C, Andrews C, Hebbel RP, Galipeau J, Hough C, Lillicrap D.

Stem Cells. 2007 Oct;25(10):2660-9.


Sustained transgene expression by human cord blood derived CD34+ cells transduced with simian immunodeficiency virus agmTYO1-based vectors carrying the human coagulation factor VIII gene in NOD/SCID mice.

Kikuchi J, Mimuro J, Ogata K, Tabata T, Ueda Y, Ishiwata A, Kimura K, Takano K, Madoiwa S, Mizukami H, Hanazono Y, Kume A, Hasegawa M, Ozawa K, Sakata Y.

J Gene Med. 2004 Oct;6(10):1049-60. Erratum in: J Gene Med. 2005 Jun;7(6):836. Kimura, Konzoh [corrected to Kimura, Kouzoh].


The establishment of murine blood outgrowth endothelial cells and observations relevant to gene therapy.

Somani A, Nguyen J, Milbauer LC, Solovey A, Sajja S, Hebbel RP.

Transl Res. 2007 Jul;150(1):30-9.


Sustained expression of human factor VIII in mice using a parvovirus-based vector.

Chao H, Mao L, Bruce AT, Walsh CE.

Blood. 2000 Mar 1;95(5):1594-9.


Sustained expansion and transgene expression of coagulation factor VIII-transduced cord blood-derived endothelial progenitor cells.

Herder C, Tonn T, Oostendorp R, Becker S, Keller U, Peschel C, Grez M, Seifried E.

Arterioscler Thromb Vasc Biol. 2003 Dec;23(12):2266-72.


Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction.

Tiede A, Eder M, von Depka M, Battmer K, Luther S, Kiem HP, Ganser A, Scherr M.

Gene Ther. 2003 Oct;10(22):1917-25.


Bone marrow mesenchymal cells for haemophilia A gene therapy using retroviral vectors with modified long-terminal repeats.

Van Damme A, Chuah MK, Dell'accio F, De Bari C, Luyten F, Collen D, VandenDriessche T.

Haemophilia. 2003 Jan;9(1):94-103. Erratum in: Haemophilia. 2003 May;9(3):345.


Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors.

Chuah MK, Schiedner G, Thorrez L, Brown B, Johnston M, Gillijns V, Hertel S, Van Rooijen N, Lillicrap D, Collen D, VandenDriessche T, Kochanek S.

Blood. 2003 Mar 1;101(5):1734-43.


Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient.

Piacibello W, Bruno S, Sanavio F, Droetto S, Gunetti M, Ailles L, Santoni de Sio F, Viale A, Gammaitoni L, Lombardo A, Naldini L, Aglietta M.

Blood. 2002 Dec 15;100(13):4391-400.


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.


Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice.

Dwarki VJ, Belloni P, Nijjar T, Smith J, Couto L, Rabier M, Clift S, Berns A, Cohen LK.

Proc Natl Acad Sci U S A. 1995 Feb 14;92(4):1023-7.


Storage and regulated secretion of factor VIII in blood outgrowth endothelial cells.

van den Biggelaar M, Bouwens EA, Kootstra NA, Hebbel RP, Voorberg J, Mertens K.

Haematologica. 2009 May;94(5):670-8. doi: 10.3324/haematol.13427.


Phenotype correction of hemophilia A mice with adeno-associated virus vectors carrying the B domain-deleted canine factor VIII gene.

Ishiwata A, Mimuro J, Kashiwakura Y, Niimura M, Takano K, Ohmori T, Madoiwa S, Mizukami H, Okada T, Naka H, Yoshioka A, Ozawa K, Sakata Y.

Thromb Res. 2006;118(5):627-35.


Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system.

Ohlfest JR, Frandsen JL, Fritz S, Lobitz PD, Perkinson SG, Clark KJ, Nelsestuen G, Key NS, McIvor RS, Hackett PB, Largaespada DA.

Blood. 2005 Apr 1;105(7):2691-8.


Therapeutic levels of human factor VIII in mice implanted with encapsulated cells: potential for gene therapy of haemophilia A.

García-Martín C, Chuah MK, Van Damme A, Robinson KE, Vanzieleghem B, Saint-Remy JM, Gallardo D, Ofosu FA, Vandendriessche T, Hortelano G.

J Gene Med. 2002 Mar-Apr;4(2):215-23.


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.


Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIII.

Wilcox DA, Shi Q, Nurden P, Haberichter SL, Rosenberg JB, Johnson BD, Nurden AT, White GC 2nd, Montgomery RR.

J Thromb Haemost. 2003 Dec;1(12):2477-89.


Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector.

Balagué C, Zhou J, Dai Y, Alemany R, Josephs SF, Andreason G, Hariharan M, Sethi E, Prokopenko E, Jan HY, Lou YC, Hubert-Leslie D, Ruiz L, Zhang WW.

Blood. 2000 Feb 1;95(3):820-8.

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