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Replacing bad (F)actors: hemophilia.
Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):461-7. doi: 10.1182/asheducation-2014.1.461. Epub 2014 Nov 18.
Hematology Am Soc Hematol Educ Program. 2014.
PMID: 25696895
Review.
Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A.
Johnston JM, Denning G, Doering CB, Spencer HT.
Johnston JM, et al.
Gene Ther. 2013 Jun;20(6):607-15. doi: 10.1038/gt.2012.76. Epub 2012 Sep 20.
Gene Ther. 2013.
PMID: 22996197
Free PMC article.
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Lentiviral vector platform for production of bioengineered recombinant coagulation factor VIII.
Spencer HT, Denning G, Gautney RE, Dropulic B, Roy AJ, Baranyi L, Gangadharan B, Parker ET, Lollar P, Doering CB.
Spencer HT, et al.
Mol Ther. 2011 Feb;19(2):302-9. doi: 10.1038/mt.2010.239. Epub 2010 Nov 16.
Mol Ther. 2011.
PMID: 21081907
Free PMC article.
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